Susan Haigney

palovarotene molecule, molecular structures, retinoids, 3d model, Structural Chemical Formula and Atoms with Color Coding | Image Credit: ©Сергей Шиманович -stock.adobe.com

Quotient Sciences, a contract research, development, and manufacturing organization, announced on February 26, 2026 that it is extending its commercial supply partnership with biopharmaceutical company, Ipsen.

 Under the partnership, Quotient will manufacture Sohonos (palovarotene), an FDA-approved, highly potent molecule for the treatment of fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease that impacts fewer than 1000 people worldwide. The partnership will ensure that a supply of the FOP treatment is secure.

Because of its highly potent nature, palovarotene requires specialized handling and containment. Ipsen has invested in new equipment at Quotient’s Boothwyn, PA facility for the manufacture of the product. This investment includes a pneumatic closed transfer system, for safe material transfer without impacting blend segregation, and a flexible dispensing isolator to enhance operator safety and remove special cleaning requirements.

“This significant investment in advanced containment and manufacturing technology at our Boothwyn facility underscores Quotient Sciences’ unwavering commitment to supporting patients with ultra-rare diseases,”Marlene Leuenberger, VP and General Manager, Quotient Sciences, Philadelphia, said in a press release.

 “By expanding our capabilities in handling highly potent compounds, we are not only ensuring a reliable supply of critical therapies like Sohonos (Palovarotene) for the FOP community, but also reinforcing our promise to deliver flexible, high-quality solutions for our partners with specialized needs especially for highly potent compounds.”

What other targeted medicines is Ipsen developing?

In Europe, Ipsen’s monotherapy, Ojemda (tovorafenib), was given a positive opinion by the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) for conditional marketing authorization.

 Ojemda (tovorafenib) is a treatment for pediatric low-grade glioma (pLGG) with harboring a BRAF fusion or rearrangement or BRAF V600 mutation, who have progressed after one or more prior systemic therapies, in patients 6 months of age and older. According to the company, BRAF altered pLGG causes problems with sight, speech, and neurological problems in children.

“For children diagnosed with pediatric low‑grade glioma, the journey is long and complex, with limited treatment options and no clear standard of care,” said Christelle Huguet, PhD, EVP and head of Research & Development, Ipsen, in a press release.

 “Innovating in pediatric oncology is challenging, and genuine breakthroughs are rare. The evidence supporting tovorafenib, which acts on tumor growth driven by an abnormal BRAF gene, is an important development. The positive CHMP opinion moves us closer to delivering a meaningful targeted therapy for these patients and their families.”

“Treatment options for relapsed/refractory pediatric low-grade glioma have remained limited for decades, which is why a targeted therapy like tovorafenib has the potential to transform how we manage this disease in particular the most common subtype carrying the BRAF-Fusion oncogene,” said Professor Olaf Witt, director, Translational Pediatric Oncology, Hopp Children’s Cancer Center Heidelberg, in the press release.

 “As a treating physician, I am encouraged by the prospect of introducing a new treatment option for this challenging condition in Europe.”

The CHMP opinion was based on data from a pivotal phase 2 FIREFLY-1 study. Tovorafenib was evaluated in 137 children and young adults with relapsed or refractory BRAF-altered pLGG who had received at least one prior systemic therapy. The study showed there as an overall response rate of 71%, and the median time to response was 5.4 months with an 18-month median duration of response. Tovorafenib was well tolerated, according to the study, with grade 1 or 2 treatment-related adverse events.

Quotient Sciences Extends Commercial Manufacturing Partnership for Ultra-Rare Disease Treatment. Press Release. Quotient Sciences. February 26, 2026. 

https://www.quotientsciences.com/news/quotient-sciences-extends-commercial-manufacturing-partnership-ultra-rare-disease-treatment

Ipsen receives positive CHMP opinion for Ojemda® for the treatment as monotherapy of children with relapsed or refractory BRAF-altered pediatric low-grade glioma. Press Release. Ipsen. February 27, 2026. 

https://www.ipsen.com/press-release/ipsen-receives-positive-chmp-opinion-for-ojemda-for-the-treatment-as-monotherapy-of-children-with-relapsed-or-refractory-braf-altered-pediatric-low-grade-glioma-3246394/

Articles

Quotient Sciences extended its commercial supply partnership with Ipsen to manufacture palovarotene (Sohonos), an FDA-approved, highly potent therapy for fibrodysplasia ossificans progressiva, reinforcing supply security for an ultra-rare population. Ipsen funded containment and handling upgrades at Quotient’s Boothwyn, Pennsylvania site, including closed transfer and isolator technologies. Separately, Ipsen advanced its targeted oncology portfolio in Europe: tovorafenib (Ojemda) received a positive CHMP opinion for conditional approval in relapsed/refractory BRAF-altered pediatric low-grade glioma, supported by FIREFLY-1 efficacy and tolerability.

The companies have extended a commercial supply partnership for the manufacture of Sohonos (palovarotene), which is a treatment for the ultra-rare disease, fibrodysplasia ossificans progressiva.

Quotient Sciences Extends Partnership with Ipsen for Manufacture of Ultra-Rare Disease Treatment

The Importance of Drug Development for Rare Diseases

FDA defines rare diseases as “a disease or condition that affects less than 200,000 people in the United States”. 

 The development of treatments for these diseases has unique challenges that can lead to a lack of options for patients. 

spoke with Elisabeth Gardiner, PhD, chief scientific officer of Tevard Biosciences, about these challenges and what makes treatments for these conditions so important.

Because rare disease treatments target a small number of people, there can be little return on investment for pharmaceutical companies. This can be set off by incentives given by regulators and governments,

 but it’s still can be a gamble for some companies. Gardinar suggests that a therapeutic approach that can be applied to multiple rare diseases may create the opportunities companies are looking for that can also help additional people.

“The ironic thing is that when drug hunters think about looking at targets for new drug discovery projects, they really look to rare diseases to get hints for targets and for this deep underlying biology that might be really interesting and exciting,” Gardiner says. “An example of this is PCSK9 inhibitors. They were originally found by looking at familial hypercholesterolemia that was actually quite rare. Homozygous familial hypercholesterolemia was discovered in the 1930s. This observation has yielded not only Nobel Prizes, but some of the most successful drugs that we have. Really understanding the work that was decoded by Brown and Goldstein, you know, that work earned them the Nobel Prize. That was on LDL [low-density lipoprotein] receptor mutations, and essentially, these genetic insights led to the development of PCSK9 inhibitors that can dramatically lower LDL. And in fact, there's even a genetic, medicine now, being brought forward by Verve. It's a CRISPR-based editor. It essentially, it can address familial hypercholesterolemia, but the way it's working in the clinic, it may be applicable to anybody with hypercholesterolemia. It's incredibly exciting.”

https://www.fda.gov/patients/rare-diseases-fda

FDA. Designating an Orphan Drug Product: Drugs and Biological Products. FDA.gov. August 12, 2025. 

https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products

Elisabeth Gardiner, PhD, Chief Scientific Officer, Tevard Biosciences

Elisabeth is chief scientific officer at Tevard Biosciences, joining the leadership team in 2025 to spearhead the company’s pioneering tRNA-based gene therapies for rare diseases with high unmet need. With more than 25 years in the biopharmaceutical industry, she has directed and managed drug discovery and development efforts yielding 11 IND filings, four Phase I/II trial candidates, and one Phase III candidate. Prior to Tevard, Elisabeth held senior R&D leadership roles at Tactile Therapeutics, Alterome Therapeutics, Aravive, and Kinnate Biopharma, where she directed multidisciplinary teams and advanced programs from discovery to clinical-stage development across neurology, oncology, and rare disease. Elisabeth gained experience in tRNA biology while working at aTyr Pharma and at Scripps Research Institute in the lab of Paul Schimmel. Elisabeth’s commitment to the ethical development of effective and accessible medicines is her key focus in life. In addition to her professional work, Dr. Gardiner acts as a patient advocate in the rare disease and oncology space. Elisabeth earned her Ph.D. from the University of Wisconsin-Madison and holds a B.S. and an M.S. from Texas A&M University.

Editor's note: This transcript is a lightly edited rendering of the original audio/video content. It may contain errors, informal language, or omissions as spoken in the original recording.

Hi, I am Elisabeth Gardner. I'm CSO of Tevard Biosciences. There is a significant unmet medical need to address rare diseases. Given the challenges in developing treatments, especially disease-modifying treatments for rare disease, it's critical to be able to have an approach that can efficiently target multiple rare diseases.

I think that part of the problem is, you know, when you're going after a single rare disease, you don't actually get as much, bandwidth, much addressable population. So, if you have a therapeutic approach that can actually address multiple rare diseases, potentially rare diseases with the same sort of underlying genetic mechanism, then you really have an excellent opportunity to help a lot more people.

And I think rare diseases are very poorly named. It's only rare until it's your loved one that has the rare disease, and then all of a sudden you realize that you're part of this club that you really didn't want to be part of, and you're suddenly part of a really large community of people that are desperate for answers and desperate for cures.

The ironic thing is that when drug hunters think about looking at targets for new drug discovery projects, they really look to rare diseases to get hints for targets and for this deep underlying biology that might be really interesting and exciting.

An example of this is PCSK9 inhibitors. They were originally found by looking at familial hypercholesterolemia that was actually quite rare. Homozygous familial hypercholesterolemia was discovered in the 1930s.

This observation has yielded not only Nobel Prizes, but some of the most successful drugs that we have. Really understanding the work that was decoded by Brown and Goldstein, you know, that work earned them the Nobel Prize.

That was on LDL receptor mutations, and essentially, these genetic insights led to the development of PCSK9 inhibitors that can dramatically lower LDL.

And in fact, there's even a genetic, medicine now, being brought forward by Verve, Verve one oh one. It's a CRISPR-based editor. It essentially, it can address familial hypercholesterolemia, but the way it's working in the clinic, it may be applicable to anybody with hypercholesterolemia. It's incredibly exciting.

I think thinking about rare diseases, they provide insights for the larger population, but, I mean, there's just a huge unmet need. There's so many rare diseases out there.

There are three main challenges that I think everyone working on rare disease encounters. One is the lack of reliable models, either animal models or cellular models. Two is the complex biology of the disease, and three is the lack of funding. So many rare diseases do not have a cellular animal model that can permit drug screening or testing to be done in a way that's actually translational to the human condition.

It's tough, actually, to know if one cell line that is available or one mouse model that you might have access to is going to be representative of the human pathology that you're trying to treat.

It's a really black box. I mean, it's a lot of guessing. It's expensive and time-consuming to actually develop the models that are most relevant, and money and time are often in very short supply when you're trying to be innovative, and certainly, the patients can't wait that long.

One of the reasons why rare disease, medications take so long to get to the clinic is because we have very specific guidelines about how we bring drugs to the clinic, and part of that is, you know, we need a cellular model, we need an animal model, we need to do the toxicity testing, and then we can put it in humans.

I think all of that is, is really important, but when you talk about rare disease, you really need to think out of the box. I love actually what the FDA is doing right now. They're really trying to streamline the process for rare disease. They're well aware that the animal models may not be there.

They're focused on cell model development, and in fact, there's a whole coalition that's kind of working on not just thinking about how to move rare disease medications forward, but really thinking about how to build the cellular models, how to build the pediatric models, that, that might be key for, for getting things, into the clinic without having to build the animal models. And so, changing that timeframe, you know, changing that delay.

So, for rare disease research and development, the lack of funding is really a tricky thing. I think often this comes because of the complex biology of the disease, so investors may be loath to jump into investing in something that has such complex biology.

They look at it, and they think: "That's gonna take a long time for me to get return on my investment." And I think that's logical.

But when, when you think about it, you know, genetic diseases are not just one thing. It may be just one gene that is the causative gene that's making the pathology relevant to the patient, but often you're not dealing with a simple situation.

So that one genetic malformation may cause a whole cascade of biological problems, and so you're dealing with a potentially very fragile patient population.

And so that's another reason why people may have difficulty, actually getting into rare disease biology and thinking about it from a drug discovery perspective.

Often, patients are on another type of drug to manage symptomology, and this can actually mask the benefit of a potential new medication. It may actually exacerbate the condition, too.

So, for example, you may be on a genetic therapy that's very helpful to cure your disease by modifying the gene, but if that genetic therapy is causing liver damage, that could kill you. And so, you may have a patient that ultimately could be helped, but ends up having an exacerbation that leads to a lifetime of illness, unrelated to the original genetic cause.

So, I think that there's a lot of problems, you know, this sort of, this thought process that I have, you know, this one mutation that's caused me to have epilepsy.

But all of my organ systems are now disturbed because my epilepsy has actually damaged my brain, and so that could be GI issues, that could be verbal issues, that could be issues responding to other medication because brain chemicals are changed by the constant inflammation.

There's a lot of reasons why the complex biology of the disease just makes rare diseases very tricky, and that gets to the lack of funding. So, it really takes a lot of money and highly trained people to make a rare disease treatment work well.

One example, quite recent, that was highly successful was the Baby KJ story. This was highly publicized. It was truly a tremendous scientific effort for one baby, you know, with one rare disease. And stories like this catch the attention and, you know, really fill people with a lot of hope and excitement.

But the reality is that this took a tremendous amount of money and a tremendous amount of effort, and I would say altruism, on the part of a large number of people.

If you look at the paper, there's a lot of names, you know, on the author list, but everybody behind that, you know, this kid's parents, all the caregivers, all the nurses, therapists, rehab folks, everybody who worked with this kid, and, I mean, he didn't spend one night at the hospital.

He spent a long time at the hospital trying to make sure that the therapy was working and actually working through any, potential ramifications of the genetic, medication that he got.

I was so impressed when I read this because what a tour de force, right?

But a success story like this really hammers the point home that this is big business. I mean, it requires a lot of money to work efficiently. They took so little time to get that done, and honestly, the FDA worked with them in a tremendously efficient way.

But when you're an investor, you think, "Okay, that's just one patient. How am I going to get return if I throw millions of dollars at this? Will I be getting millions of dollars back? Will I be getting billions of dollars back? Is there that big a market?"

I think that the way that we can think about this is, you know, a lot of times you think, "Well, I can charge three million dollars for a single treatment," but when you're the patient, or the patient's family, and, or an insurer, and that's your option, you may not be very interested in that. I mean, that may be out of reach.

And so, I think that we need to be able to think long term and think about expanding out from the original patient population into the broader population, similar to the PCSK9 story. You know, from an investor standpoint, understanding how much return on investment you're going to get is a really sensible question. But I think it's important to be creative and having a real understanding about the complex biology so that you can expand and show the investors that we'll treat these 10 patients or maybe these 100 patients, but this potential therapy can be broad spectrum.

You know, we can really look across a lot of disease states if we can actually tackle this one disease state.

Videos

Elisabeth Gardiner, PhD, CSO of Tevard Biosciences, explains some of the challenges associated with the development of treatments for rare diseases.  

scientist with equipment and science experiments, laboratory glassware containing chemical liquid for research for pharmaceutical or analyzing a sample into test tube in laboratory | Image Credit: ©CasanoWa Stutio -stock.adobe.com

Drug discovery can involve extensive research to identify new products for pharmaceutical companies to invest in. After discovery, the development of these products involves decisions on delivery options, materials selection, and quality operations to bring a new drug to market. Contract research organizations (CROs) and contract research, development, and manufacturing organizations (CRDMOs) assist sponsor pharmaceutical companies in performing the varied tasks involved in research and development.

 spoke with Tuneer Ghosh, executive vice president and head of CMC Services at Sai Life Sciences; Lidia Garcia Martin, MSAT & New Productions head, at Recipharm; and Marcio Temtem, vice president for Strategic Business Management at Hovione, to find out more about outsourcing of research and formulation activities.

Which Overall 2025 Trends Impacted the Pharma Industry the Most?

 Throughout 2025, the pharmaceutical landscape was reshaped by the accelerating demand for targeted and increasingly complex therapies. Growth in oncology, immunology, and precision-medicine pipelines intensified the focus on molecules with higher potency and narrower therapeutic windows. These compounds offer notable clinical promise but demand far more sophistication in handling, containment, and process control.

As sponsors advanced more niche and personalized modalities, manufacturers were pushed to adopt holistic approaches to safety and quality. For example, integrating equipment design, facility layout, cleaning strategies, and procedural controls to manage occupational exposure and maintain compliance.

The industry also saw expanded investment in scalable containment solutions, digitalized process oversight, and the technical capabilities required to progress highly active compounds through development. Successful drug development depends on managing potent materials safely, efficiently, and with strong technical discipline.

 One of the trends that we see today is the increased complexity of small molecules. They are not so small anymore. These days, they have reached a size and complexity that require multiple chemical steps and very often high potency. On the formulation side, poor solubility and consequently poor bioavailability became a major trend. At Hovione, the platform of amorphous solid dispersions by spray drying has been gaining relevance in addressing these challenges, with multiple products approved each year.

Another trend is the “faster pace” at which new molecules are being added to development pipelines. AI is booming across the industry, and with this new way of synthesizing molecules also comes complexity and the need to debottleneck the chemistry, manufacturing, and controls section, achieving clinical supply and processes scale-up in much tighter timelines.

The third trend is the growing concern about the regionalization of supply chains. In our case, we look at this as an opportunity. Hovione has sites in three continents: in Asia (China, Macau), in Europe (Portugal, Ireland), and the United States. All these sites are regularly inspected by the medical authorities, including FDA, and we believe this is an opportunity for Hovione to continue serving our clients globally.

What Standout Trends Specifically Impacted Drug Formulation in 2025?

 In 2025, drug formulation was shaped by the rise of complex modalities, broader use of AI-enabled tools, increased focus on patient-centric dosage forms, and advances in drug delivery technologies. These trends increased formulation complexity and regulatory expectations. As a result, clients increasingly gravitated toward CRDMOs that offer integrated services across formulation, analytical development, scale-up, and regulatory readiness under one roof. The growth of peptides, PROTACs [proteolysis targeting chimeras], and gene therapies, among others, also accelerated the shift away from fragmented outsourcing toward full-service CRDMO models.

How Early Do CRDMOs Typically Get Involved in Orphan or Rare-Disease Drug Development?

 At Sai Life Sciences, we are often engaged at very early stages of orphan and rare-disease programs, starting with preclinical formulation screening and extending through first-in-human studies. Early involvement allows integrated teams to assess feasibility, define formulation strategy, support small-batch manufacturing, and align development decisions with downstream regulatory requirements. More broadly, this reflects a growing trend where customers and clients involve CRDMOs earlier in orphan programs to manage compressed timelines, small patient populations, and complex formulation challenges more effectively.

How Important Are CRDMOs in the Formulation and Regulatory Approval of Orphan Drugs?

 CRDMOs play a critical role in orphan drug development, where flexibility, speed, and specialized expertise are essential. They support formulation and delivery challenges, including low-dose, pediatric-friendly, and alternative dosage forms, while contributing to regulatory strategy. Integrated formulation, analytical, and regulatory teams should work together to generate robust data packages, conduct stability and comparability studies, and align development with accelerated or orphan approval pathways. This integrated model helps clients advance programs efficiently toward regulatory approval.

What Do You Anticipate for Outsourced Formulation Services in 2026 and Beyond?

 Outsourced formulation services are expected to evolve toward deeper integration, greater use of AI-driven development tools, and increased demand for specialized delivery technologies. Growth in personalized and precision medicine will drive more tailored formulation approaches, particularly for complex and low-volume programs. Clients are also likely to favor long-term partnerships with integrated CRDMOs who can support formulation, analytics, manufacturing, and regulatory needs across the development lifecycle, moving beyond transactional outsourcing models.

Which Trends Do You See Driving Innovation and Drug Development in 2026?

 Some of the trends I mentioned before, such as increased complexity and acceleration, are being managed by Hovione, with a focus on developing new hardware and software.

On the hardware side, we recently signed a partnership with GEA, one of the leading suppliers of continuous tableting rigs for the pharma industry. As part of this partnership, we have launched a new solution for the continuous manufacturing of tablets—the new ConsiGma CDC Flex.

 Such a disruptive offer enables having three modes of operation in one single instrument. There’s an opportunity to run the press in batch tableting or running the equipment in continuous mode at modes that would typically require distinct units, for very low or commercial throughputs. The unit has been designed with this modularity, expecting this flexibility to accelerate development while minimizing API use and learner scale-up.

Another hardware example is our partnership with Microinnova to develop modular continuous-flow setups.

 With the use of Module Type Packages, enabling “plug-and-produce” in a process orchestration that builds versatility and flexibility on the shop floor. Chemists start from the early stages of development, working and establishing, in a “Lego fashion,” the setup that will later be used in commercial manufacturing.

On the software side, we have also been exploring new ways of doing sustainable chemistry, with micellar chemistry surfacing as one of the neatest additions to our toolbox. This proprietary process technology enables the use of less energy-intensive conditions, lower levels of precious-metal-containing catalysts, and reduced use of organic solvents in the chemical synthesis of APIs.

Another example is modeling, where Hovione has spent the past 10–15 years developing tools (databases, models, proprietary lab setups, etc.) that enable formulation development, scale-up, and process transfer with minimal API use and in a short time frame, particularly in the areas of solubility enhancement through amorphous solid dispersions.

 A key trend we can expect to see drive innovation in 2026 is the surge in high-potency APIs for high-potency drug development, as more therapies rely on highly active compounds for targeted efficacy. This shift places a premium on specialist capabilities to ensure both operator safety and predictable product quality, particularly advanced containment, automation, and data-driven process control.

The next wave of progress will likely stem from robotics and closed-system technologies that reduce manual handling and mitigate exposure risks, alongside digitalized monitoring that strengthens science-based decision-making and enhances risk management. As developers seek to move potent candidates from early development toward commercial readiness, technical transfer expertise and integrated containment strategies will become differentiators.

Hovione. Hovione and GEA Set New Standards for Continuous Tableting. Press Release. Dec. 2, 2025. 

https://www.hovione.com/press-room/press-release/hovione-and-gea-set-new-standards-continuous-tableting

Hovione. Hovione and Microinnova Partner to Advance Modular Flow Chemistry for Multi-Purpose Manufacturing. Press Release. Sept. 24, 2025. 

https://www.hovione.com/press-room/press-release/hovione-and-microinnova-partner-advance-modular-flow-chemistry-multi-purpose-manufacturing

Susan Haigney is lead editor of PharmTech.

Industry experts discuss the benefits of utilizing contract development and contract research organizations in drug discovery and development. 

Outsourcing Formulation Activities May Advance Drug Development

Insights on New Modalities, Digital Integration, and Manufacturing Innovation

, Andrew Mitchell, associate vice president of Business Development at BioVectra, provides a comprehensive overview of the evolving contract development and manufacturing organization (CDMO) landscape. According to Mitchell, the most significant industry trend that emerged in 2025 was the “boom” of new therapeutic modalities, specifically antibody-drug conjugates (ADCs), RNA, and cell and gene therapies. However, the industry also faced geopolitical turmoil that affected vaccine sentiment and funding cuts that forced small biotech companies to consolidate their pipelines. Despite these challenges, the market saw significant M&A activity.

Digital transformation is increasingly central to manufacturing efficiency. While tools like laboratory information management systems, electronic batch records, and project management software are already established, AI is now emerging in manufacturing and data analysis. A major barrier to AI adoption remains the high cost of proprietary licenses, as companies cannot use public, open-data models for sensitive pharmaceutical work.

The rapid pace of digitalization has created a skills gap, necessitating that staff with traditional chemistry or biology degrees upskill in the IT sector . BioVectra addresses this by collaborating with local universities and training-based companies to provide continuous education, while also using pay and benefits to retain talent.

To improve competitiveness, CDMOs are focused on reducing cycle times to lower costs for clients. Technical innovations include engineering 

 expression systems for extracellular secretion in microbial fermentation, which reduces capital and operational expenses while avoiding hazardous chemicals. Additionally, the use of flow reactors in chemistry ensures cleaner reactions and fewer impurities.

Looking ahead to 2026, Mitchell predicts continued growth in precision oncology and RNA therapeutics. He highlights the increasing importance of drug delivery systems, specifically the development of new lipids to improve the stability of lipid nanoparticles. Finally, there is a trend toward companies seeking CDMO partners that provide a pathway from early-stage development to commercial production.

Andrew Mitchell is associate vice president of Business Development at BioVectra.

Hi. My name is Andrew Mitchell. I'm the associate vice president of business development. Business development in BioVectra is the client facing section, the commercial section. And our company is based in Atlantic, Canada. We have four different sites which are using different modalities to manufacture drug substance, primarily drug substance, but also some drug products for the pharmaceutical industry.

Which trends did you see or experience in 2025 that you believe impacted the pharmaceutical industry the most? How and why?

So, I think the biggest trend that we continue to see is the emergence of new therapeutic modalities, specifically at this point in time, ADCs, which are which are really having a boom in the industry, radio ligament therapies, RNA and cell and gene therapies, and also the bio multi specific antibodies as well.

We also see, I mean, it's quite obvious, there's been a lot of geographic political turmoil which has affected particularly the sentiment about vaccines. The industry itself. There have been cuts in funding, which has restricted many smaller biotech pipelines, and generally, there has been a move to consolidate pipelines to maybe one or two maximum in the small biotechs in the clinical trials, which has narrowed the opportunities for people like ourselves in the CDMO field. However, on top of that, there has been a very buoyant pharma acquisition market in 2025 again, continued consolidation in the CDMO field, like ourselves, we were acquired by Agilent in late 2024 and have been going through an integration process in 2025, but there's also been vertical integration by CDMOs, and a noticeable activity by private equity in acquiring CDMO assets as well.

What role do artificial intelligence and digital technologies play in accelerating drug discovery and improving manufacturing efficiency, and what are some unresolved barriers to full adoption?

So, I mean, digital technologies have been moving in for quite some time. I mean, we've got LIMS systems. So, you know, laboratory systems that are that are helping to streamline the progress of samples and testing throughout the system. We now have the use of electronic batch records, which are becoming increasingly more popular in the industry, and also project management software, whether that be monday.com or smart sheets or whichever system that is available to different companies.

And there is now a lot of use starting to emerge of artificial intelligence in manufacturing, analysis of quality data and data in general, but one of the big barriers to this is the cost of the license. You clearly can't use a public data open data AI model. You have to have an AI agent which is specific to your company. And that's a very large cost at the beginning. But it does seem that everybody at the moment is looking at AI in different ways, in terms of manufacturing, and I think that's something that's going to be increasing in the future, and hopefully will bring not just efficiencies, but also productivity as well, into the industry.

How can organizations bridge the widening skills gap created by the rapid digitalization of pharmaceutical manufacturing, and what best practices are emerging for workforce development?

In our industry, it's very common that we have staff with degrees in either chemistry, biology, or engineering or combination of those. But as mentioned in previous discussion,

the use of digitalization is requiring people to also upskill in the IT sector as well, and that works hand in hand with their existing experience. So to do that, we've been working with a number of local universities and also training-based companies to provide our business with the workforce that is trained and efficient to enable us to not only keep talent, but also we have to work to train it constantly and update it, but also incentivize them through pay and other benefits to help them to remain with us.

Which recent innovations in product development or process optimization have had the broadest impact on cost, quality, or speed-to-market in manufacturing operations?

Speaking, specifically from a CDMO point of view, we're all about improving the cycle times of the processes that we operate on behalf of our clients and are continually looking at a process of chipping away to make small improvements.

Improvements, and the many small improvements, add up to a large gain in terms of cycle time reduction for the client, which is always good. They get less time than our client means lower cost, and lower cost basically also means for them, you know, better margins on their drug product, better competitiveness as well. One of the major opportunities we have is in the manufacture of biologics through microbial fermentation. In addition to bringing in technologies which improve the flow in line, sterilizers as one example, we've also been looking at engineering the expression systems, specifically 

, so that we can actually perform extra cellular secretion, and this allows us to cut costs in terms of CapEx, OPEX cycle times, and the use of hazardous and not very nice chemicals when doing the repro refolding of proteins. So, we're always looking at this, these types of improvements, I would say, on our chemistry business, and also in our highly potent business, we're looking at the use of flow reactors to ensure that we get cleaner reactions fewer impurities. Are able to handle a much wider range of reactions and basically then adding that and rounding all of those things off with the use of real-time release and analytical technology.

Which trends do you see driving innovation and drug development in 2026? How and why?

We continue to see the growth in the new modalities. I don't think that we will ever believe that small molecules will go away or mammalian products will go away, but we do believe that there is still a lot more to go. In terms of RNA therapeutics, the cell and gene therapies, they seem to be making headway. They have some hiccups along their path, but seem to be moving in the right direction. We see a lot of work being done on precision oncology, and that we're involved very much in the ADC part of that, the payload part, not so many new payloads. Some are still coming through, but a selection of payloads which can be added to new antibodies to for targeting.

One other part that we see is delivery systems, and this is becoming quite important. People realize that perhaps the lipid systems, just the standard lipid nano particle could be improved, and they're doing a lot in that field to try and find new types of lipids which might improve delivery and stability of the drug product to into the body. And finally, we see a slight change in that companies who perhaps are more constrained in their early stage are looking for companies such as ours that have capabilities in both early stage and a pathway to commercial production. And I think these are the trends which are going to be seen and will continue in 2026.

PharmTech spoke with Andrew Mitchell, associate vice president of Business Development at BioVectra, about which 2025 trends will continue to impact the pharma industry and how CDMOs can offer solutions.

Ask the Expert: Working with Regulators to Develop Orphan Drugs

 video series. This series is an extension of the 

 Group’s long-running print column in which industry experts answer common quality and regulatory questions from the industry. This video series expands on topic areas to include development and manufacturing concerns to help those working in the industry better understand bio/pharmaceutical requirements. Have a question you would like answered? Send it to 

, and it may appear in a future episode or print column.

In this episode of the Ask the Expert video series, Susan J. Schniepp, Distinguished Fellow, Regulatory Compliance Associates, a Nelson Labs Company, and Siegfried Schmitt, Vice President, Parexel, answer the question:

“We are a medium sized company and are looking to develop a new drug and are thinking of applying for orphan drug status. Do you have any suggestions for working regulators?”

For medium-sized pharmaceutical companies considering developing a drug for orphan drug designation, the transition from traditional development to rare disease therapy requires a shift in regulatory, clinical, and operational planning. An orphan drug is defined by the size of the population it treats; in the United States, this generally refers to conditions affecting 200,000 or fewer people,

 while the European Union uses a threshold of approximately five in 10,000.

What are the regulatory and financial incentives

According to Schniepp and Schmitt, the orphan drug designation route offers government-backed incentives designed to offset the limited blockbuster potential of these products. These include financial support that includes government funding, grants, and tax credits. In the US, fees under the Prescription Drug User Fee Act may be waived for orphan drug developers. Regulators also offer more support for applications and faster approval timelines.

What challenges does the Orphan Drug pathway bring?

Orphan drugs must meet the same rigorous quality and compliance standards as traditional drug approvals , Schmitt and Schniepp emphasize. An accelerated approval process often means manufacturers must be more agileto apply high-level quality systems within a shorter timeframe.

Orphan drugs may also introduce specific manufacturing complexities. Producing smaller batches for clinical trials or commercial use is often more difficult than large-scale production.

Smaller companies without internal facilities may need to utilize contract development and manufacturing organizations (CDMOs). However, CDMOs may find it harder to integrate low-volume runs into their schedules compared to large-scale large tank production.

Because the patient population is small and globally dispersed, distributing small batches of product to far-reaching locations may also be a logistical hurdle.

Patient enrollment for rare-disease treatment development may be difficult, especially if multiple companies are competing for the same eligible participants. Medium-sized firms lacking regulatory experienceshould seek external expertise, according to Schmitt, and work in close collaboration with regulatory authorities. While these therapies may be commercial risks, they may lead to the discovery of broader indications for other diseases, Schniepp suggests, making them even more worthwhile to pursue.

FDA. Office of Orphan Products Development. FDA.gov. Oct. 1, 2024 

https://www.fda.gov/about-fda/office-chief-medical-officer/office-orphan-products-development

EMA. Orphan Designation. Overview. Ema.europa.eu. 

https://www.ema.europa.eu/en/human-regulatory-overview/orphan-designation-overview

Susan J. Schniepp is distinguished fellow at Regulatory Compliance Associates and a member of PharmTech’s Editorial Advisory Board.

Siegfried Schmitt, PhD, is vice president, Technical at Parexel and a member of PharmTech’s Editorial Advisory Board.

Hi, everyone. Welcome back to Ask the Expert. I'm Sue Schniepp, and I'm with Nelson Labs, and I'm with my colleague, Siegfried Schmitt, with Parexel, and we're here to talk about, I think Siegfried has a question on orphan drugs. So, let's go for it, Siegfried.

Thank you, Sue. Yes, thanks for the introductions. In fact, yes, the question that came in is:

"We are a medium-sized company, and we are developing a new drug, and we are currently thinking of perhaps applying for orphan drug status with the regulators. Would you have any suggestions, comments, recommendations in that context?"

So, Sue, maybe you would like to start off with this.

Well, that's interesting, 'cause sometimes companies do choose the orphan drug route. Now, there's some advantages to choosing that. So first, we should clarify, an orphan drug is a drug that's treating a condition in people. In the United States, I think it's two hundred thousand or less people are affected by the condition, and I think it's a little different in the European Union.

It's, like, five in a thousand or something, or do you know, Siegfried, what that...?

I don't know the precise figure, but I know that there are slight differences between EU, US, and I believe also in other jurisdictions. You have not exactly the same threshold. Right.

But I believe in most cases, when you're looking at an orphan drug designation, you're probably looking at a rather small population that is affected by the particular disease for which you're developing your drug. Right.

So having clarified that, then the thing that always comes to mind with me is there's a couple of advantages. So, if you go the orphan drug route, you do get assistance from governments, because they realize that if you develop the orphan drug, you probably will not make a lot of money on it, right?

It's not gonna be your next blockbuster because of how few people you're going to really affect with the drug. Once they get out on the market, they're high-priced too, but they are reimbursed typically by insurance. But, you still need to meet all the standards of quality when you're developing an orphan drug.

The thing that I think you need to work the closest with the regulators on are on your clinical trials, because it can be difficult to get patients enrolled in your clinical trial, and that's what the success of your drug is based on.

You know that better than I do, Siegfried.

Well, you're right, Sue. You need to think about where you can find eligible patients, and of course, not every eligible patient will also want to take part in a trial. And interestingly, just because you may be developing a drug for a rare disease, doesn't mean that you're probably the only company doing that.

There are often many competing companies developing drugs for a similar rare disease. So, it's not that you're out there on your own, and you are without competition.

As you quite rightly say, you need to work closely with the regulators, and the regulators offer a lot of support there. And what is also very important is you have to have the same quality and compliance standards as if you go the normal drug approval route.

Yes, you have advantages time-wise. You don't have to wait as long for approval as you have to do with the normal approval pathway, but because things happen faster, you also have to be more agile. You have to work harder and faster to have the same standards, the same levels of quality, and just apply them in a shorter timeframe.

So, perhaps this is something that some companies may not fully understand, particularly when we look at the type of companies that often develop these rare disease drugs.

Very often, these are fantastic scientists, spin-offs from universities, perhaps, and as this particular person who put the question forward said, they're a medium-sized company, and that means they may not have as many people with the right experience in regulatory affairs, in bringing drugs to market, et cetera. So perhaps you need more external help there. What do you think, Sue?

I think so. I think it is a desirable route to go, too, though, because you do get some advantages. I know even in the EU, right, as a developer, so you do get funding, right? And you do get some tax credits, at least in the US. I don't know if you do in the EU.

I think both. We have a Prescription Users Drug Act here, where you pay a fee, and those fees are waived for people who are developing orphan drugs and as you said, grants or help from other sources.

So, I think it's kind of, it's not where I've actually played in the game, but I understand the attraction of it. If you are doing that, oftentimes, too, and I'd like to hear your comments on this, once the orphan drug is developed, it can go on to prompt, develop [other drugs] I think the statins came out of the development of an orphan drug; although, I'd have to check that.

But it started with an orphan drug that they somehow found at the time. I can't remember what it was for. I'm gonna have to look up my history, but they found at the time it lowered the cholesterol of the patients that were getting the orphan drug, and that turned into the statin that we now prescribe readily to patients with cholesterol. Am I remembering that right, Siegfried?

I don't know this particular case, but it's quite possible, because very often you'll find, that a particular drug that you developed for one indication is very helpful with another indication you didn't even know about, you didn't think about. So that doesn't have to come from an orphan drug designation, but it could indeed.

So, I think what companies need to weigh up very carefully is, yes, you have lower fees, you have expedited pathways, you have, much more support for your applications if you go the orphan drug route. But on the other hand, it comes with obligations, as I said, to do things faster, but to the same high standards.

Whether this is, the best possible way, again, is something you need to discuss on a regulatory perspective, but also from a commercial perspective indeed. So, it all has to be balanced out to be right.

Yeah, and your commercial perspective, I like in terms of, sometimes you don't have access. This is where it gets a little bit tricky, too. You need to make sure that you have a contract manufacturer if you're doing that route. You don't have access toa manufacturing facility. You're a smaller company, right, without that, so you're gonna have it contracted. You need to make sure that you have space on their line, line time. It's harder for a contract manufacturer to produce a smaller batch clinical trial material, than it is to produce, you know, a large batch.

At least from my experience, I would say it's easier to do, you know, a big tank and thousands of vials than a smaller clinical trial.

Indeed, and also, once you commercialize your product, again, we know it is a small population that you are servicing here, and, so the batch size, again, with a CDMO will be relatively small.

But it's also the distribution of the product, because it's very likely that your population is distributed, spread out, maybe all over the world, and it can be more challenging to distribute smaller size product batches, than it would be if you have a large commercial product.

Yep, exactly. Well, I don't really have any other advice for people other than, you know, it could be a viable pathway. Just make sure you get the right help and assistance, and work closely with your authorities.

That's exactly it. Seek help, seek expertise. If you don't have it inside, there are plenty outside, and also the regulators.

Thanks, Siegfried. I look forward to our next conversation.

In this episode of Ask the Expert, Susan J. Schniepp, Nelson Labs, and Siegfried Schmitt, Parexel, discuss the benefits of orphan drug development and how a mid-sized company can work with regulators through the pathway to approval. 

Women Leading Biotech: Precision Medicine

 about her experiences as a CEO of a biotech company. Medzown combines data analytics, expertise, and personalized navigation to connect patients with treatments.

In this interview, Dr. Carter provides a strategic roadmap for innovation and leadership within the life sciences sector. Having pioneered the field of molecular oncology decision support through her first company, N-of-One, Carter offers insights on the importance of precision medicine and data-driven evolution.

Carter emphasizes that women are currently serving as primary “thought leaders” in the advancement of precision medicine . In the context of pharmaceutical development, she points to women leading phase 1 programs and clinical trials at top medical centers, as well as those developing new models for drug development .

While the industry currently benefits from several remarkable women in executive roles, Carter notes that there remains significant room for growth in leadership diversity. She identifies the need for increasing opportunities for women to take on executive responsibilities in biotech and pharma. A critical component of this growth is the availability of mentorship and coaching to help professionals navigate the “complex systems” that exist within both small and large organizations. Carter asserts that veteran leaders have a responsibility to mentor younger women, helping them understand the challenges of the field and how to grow into leadership positions.

Reflecting on her experience as a founder and CEO, Carter underscores that the life sciences industry requires constant evolution and learning. She advocates for an open mindset where leaders listen to other innovators and observe industry trends to determine what is working and what is not. Carter suggests that leaders must bring external industry challenges back to their organizations to evolve their solutions head-on.

By focusing on continuous improvement and testing ideas against real-world data, companies can better meet the shifting demands of precision medicine and drug development. This philosophy of constant testing and listening ensures that manufacturing entities remain agile and responsive to the broader healthcare landscape.

Dr. Jennifer Carter Levin, MD, founder and CEO, Medzown

Jennifer Levin Carter, MD, MPH, MBA is Founder & CEO of Medzown. Dr. Carter is a globally recognized leader in precision medicine, with a proven track record of building transformative companies at the intersection of data, technology, and patient care. She was the Founder and President of N-of-One, Inc., where she served as CEO from 2008–2012 and later as President and Chief Medical Officer until its acquisition by Qiagen (Market Cap $8B) in 2019. At N-of-One, Dr. Carter led the development of award-winning solutions that continue to deliver novel treatment strategies to hundreds of thousands of cancer patients worldwide.

In 2018, Dr. Carter founded a novel clinical trials company focused on reimagining trial design and access. She served as CEO until its pre-launch acquisition by Integral Health (now Valo Health) in March 2019. These experiences cemented her reputation as an innovator in precision medicine, clinical trial delivery, and patient-centered care models.

Following these ventures, Dr. Carter became a Managing Partner and Venture Partner at Sandbox Industries and Blue Venture Fund, a unique collaboration between BCBS companies, BCBSA, and Sandbox. It was in this role—working closely with BCBS executives—that she gained a deep understanding of the challenges associated with the rising costs of cell and gene therapy. This insight inspired her to launch Medzown, where she continues to address those challenges by building scalable, technology-enabled solutions to improve patient access to advanced therapies.

Dr. Carter is also a seasoned healthcare executive, investor, and board member with expertise spanning healthcare IT and services, digital health and machine learning, genomics, drug development, and the creation of next-generation platforms to improve therapeutic access. She currently serves on the Board of Directors of Synteny.ai, TempraMed, Inc., and Target Cancer Foundation, and is a strategic advisor to numerous venture-backed healthcare and life sciences companies.

Dr. Jennifer Levin Carter, CEO and founder of Medzown, shares her experience as a CEO in the biotech industry and gives insights on how women can succeed in the pharmaceutical industry.