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Dosage Forms: Roundtable on Oral Solid Dosage Trends

Drug Solutions Podcast: 

Pharmaceutical packaging must advance to adapt to new, complex modalities

Out-of-the-Box Packaging Advancements

From Theory to Reality: Automating Process Development

Drug Digest: 

The Importance of Appropriate Supplier Oversight

Ask the Expert

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Process development is a key factor in the development and manufacture of biologics. Processes must be developed and validated so that product specifications can be met to create a safe and effective biologic drug. Contract development and manufacturing organizations (CDMOs) offer sponsor companies the expertise to develop these processes, which then must be validated. Process validation is a necessity to be sure the manufacturing process produces the desired, safe product.

For cell culture processes, process validation must be performed before good manufacturing practice (GMP) batches can be produced for commercial production, according to Daniel Giroux, vice-president of Biologics Development at Abzena. Formal process characterization studies must then be performed before the process validation so that one can understand the impact of variations of product parameters from their targets. Small-scale studies are performed on a validated scale-down model, and the information obtained is then applied to large-scale production.

Characterization and Qualification of Processes

Bio/Pharma Outsourcing Innovation eBook 2025

“You need to understand what are allowable ranges of process parameters and what will have an impact on your process or on the product quality, and those [parameters] that won’t [have an impact]. Then you define your process envelope …the combination of all these parameters, such as agitation, temperature, feed timing, feed amounts, and so on. [These are determined with] design-of-experiment approaches, because you have to measure [the variations’] impacts, both individually, as they change, but also when they change in combination,” says Giroux.

After process characterization and studies are performed and acceptable ranges established, batch records can be drafted, and validation protocols can be created, says Giroux. “And then you have to run multiple at-scale batches, so you know if your commercial process is going to be at the 2000-L scale, then your validation protocol will dictate how many batches you want to run. A typical number is three. It doesn’t have to be three. It could be more. Possibly could be less, rarely,” Giroux explains. “And then you execute your production batches, and you analyze heavily the impact on the product that’s coming out. You do more analysis than you normally would, something like this, and then you basically show that you met the acceptance criteria that you set up in your validation protocol.”

An essential part of process characterization is the establishment of critical process parameters (CPPs), according to Bob Schrock, PhD, senior director, Global Head of Process Development, at Lonza. Proposed critical process parameters (PCPPs) are obtained after process characterization studies are carried out.

“Once we do all the process characterization studies—and there can be literally thousands and thousands of samples, and [you have] done 12 or 15 different process characterization studies—the results are discussed with PD (process development) and MSAT (manufacturing, science, and technology) and from these, the actual CPPs can be determined,” says Schrock. “These CPPs are integrated into the process, and the validation of the process is then basically what we call the PPQ [process performance qualification] runs, the PPQ runs that are done just prior to commercialization. For [much of the] processes, the cell culture portion is only part of the process, for example, [with] viral vector manufacturing. There’s, of course, purification steps downstream from the [cell] culture portion.”

According to Schrock, PPQ runs show that a process is validated and robust and the number of runs needed depends on the variability of the process. “For a viral vector program that uses a large and well-characterized cell bank, you can do the minimum of three runs if they run perfectly. But for something [such as] autologous cell therapy, where you are starting with different cells from patients or healthy donors, you may need to do more,” says Schrock. “And then, of course, the question of using healthy donors to validate a process that is intended to start with patient samples and patient leukopaks packs, that’s always a deep and spirited discussion where you need to get creative on how you justify that. But at the end of your PPQ runs, you write a huge report that’s part of your BLA [biologics license application] submission, and it’s a cornerstone of your BLA. [It] proves that your process is validated.”

Pharmaceutical Technology®/Pharmaceutical Technology® Europe/BioPharm International

®
eBook: Bio/Pharma Outsourcing Innovation 2025
February 2025
Pages: 22–24

When referring to this article, please cite it as Haigney, S. Characterization and Qualification of Processes. 

Pharmaceutical Technology Bio/Pharma Outsourcing Innovation

Articles

Contract manufacturers have special expertise in the characterization and validation of biopharmaceutical manufacturing processes.

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

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Cosette Pharmaceuticals, of Bridgewater, NJ, announced on Feb. 20, 2025 that it has an agreement in place to buy all remaining shares of Australia-based Mayne Pharma Group (1). The current price per share is AUD$7.40, which equates to approximately US$4.69; when the transaction is completed, the total consideration will be an approximate US$430 million.

In a press release, Cosette said that the acquisition will strengthen its dermatology and women’s health businesses, furthering its pharmaceutical footprint in the United States while also asserting a presence in international markets (1). Chiefly, Cosette said it wants to expand access to women’s health therapies. The company said that upon the close of the deal, it will be able to market 12 patent-protected products centering on women’s health and dermatology—some well-known brand names Cosette mentioned in the press release include Annovera, Bijuva, Imvexxy, Intrarosa, Nextstellis, Rhofade, and Vyleesi—plus have numerous programs in clinical development.

“This acquisition marks a transformational step for Cosette, adding patented, high-growth products to solidify our leadership in women’s health in the US and expanding our reach globally,” Apurva Saraf, president and CEO of Cosette Pharmaceuticals, said in the press release (1). “By combining Cosette’s strong portfolio with Mayne Pharma’s proven commercial expertise, the combined company will be well positioned to further invest in innovation, portfolio expansion and better serve our patients. We look forward to a timely closing and welcoming Mayne Pharma to the Cosette family.”

Cosette reports a staff of more than 350 employees, while Mayne Pharma employs more than 480, suggesting that the workforce of the combined company will more than double (1). Two state-of-the-art, FDA-approved manufacturing sites will be operational as a result of the merger, in Salisbury, South Australia, and in Lincolnton, NC, in the US.

“Joining the Cosette team amplifies our shared mission to improve lives through innovative and accessible women’s health and dermatology medicines,” said Shawn Patrick O’Brien, Mayne Pharma CEO, in the Cosette press release. “This transaction represents a unique opportunity for our employees, customers, and patients. Together, we will extend the reach of important therapies, drive innovation, and improve patient access.”

 was announced in May 2024, when the United Kingdom-based retail and commercial bank Santander UK said it was providing a £30 million (approximately US$38 million) loan to UK-based Ennogen Healthcare International, which focuses on not only dermatology but also respiratory and pain relief therapies, especially among those with chronic conditions (2).

 Metrics Contract Services, which had been its full-service specialty contract development and manufacturing organization, to Catalent in August 2022 for $475 million (3).

In this latest transaction, the boards of directors of both Cosette and Mayne Pharma have approved Cosette’s share purchase agreement, and Mayne Pharma has directed its shareholders to vote in favor of the merger, according to the press release (1). The acquisition is expected to close in the second quarter of 2025.

1. Cosette Pharmaceuticals. Cosette Pharmaceuticals Announces Acquisition of Mayne Pharma, Transforming Company into a Leader in Women’s Health and Dermatology. Press Release. Feb. 21, 2025.
2. Santander. Santander Provides Pharma Manufacturer Ennogen with £30 Million Credit Facility for Growth. Press Release. May 2, 2024.
3. Catalent. Catalent to Acquire Metrics Contract Services for $475 Million to Expand High-Potent Capabilities and Oral Development and Manufacturing Capacity. Press Release. Aug. 9, 2022.

While the boards of directors of both companies have approved the transaction, it is not expected to close until the second quarter of 2025, as Mayne Pharma’s shareholders must still vote in favor of the acquisition.

Cosette Pharmaceuticals Adds to Dermatology and Women’s Health Portfolio with Mayne Pharma Purchase

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Suppliers of raw materials, lab equipment, manufacturing equipment, and other elements that go into the development and manufacture of bio/pharmaceuticals play an important role in the ecosystem that is sustainable manufacturing. Sponsor companies and contract development and manufacturing companies must ensure that the suppliers they use are following sustainability guidelines to truly produce ‘green’ products.

Stefan Lutz, PhD, SVP of Research at Codexis, a provider of enzymes and enzyme technology platforms, points to organic solvents from petrochemical sources as a “dominating factor” in global warming potential (GWP). “Therefore, technologies that replace organic solvents with aqueous-based processes or enable the design of routes to have fewer organic solvent-using steps are highly desirable in terms of improving this key GWP sustainability metric,” says Lutz. “As an added benefit, enzymatic processes often enable more selective synthesis of target molecules, potentially improving yields and simplifying downstream processing.” For the manufacture of small-molecule drugs, Lutz also points to the use of biocatalysts instead of stoichiometric reagents and rare or precious metal catalysts as potential solutions to for economic or hazardous waste production issues.For large-molecule drugs, such as therapeutic RNA oligonucleotides, Lutz calls out the use of organic solvents as an environmental factor.

“Approximately 3000 kg of acetonitrile is used per kg of RNA produced giving rise to a very large carbon footprint. Enabling higher coupling efficiencies, or fragment-based syntheses, will give higher purity, full-length products that could require less or even no further purification, which is a significant contributor to overall solvent use.”

Codexis has focused on sustainability since the company was founded in 2002, and its enzymatic solutions for API synthesis, asserts Lutz, create higher yields with reduced energy use and less waste. Oligonucleotide manufacturing, for example, uses traditional chemical processes, according to Lutz, that create hazardous reagents and chemical waste and are energy intensive. “By contrast, biocatalytic processes using enzymes generally require milder conditions, utilize renewable resources, and generate less hazardous waste,” he explains.

“Codexis is currently launching its first-in-class Enzyme Catalyzed Oligonucleotide (ECO) Synthesis manufacturing platform to provide a highly versatile and effective tool box for production of therapeutic RNA oligonucleotides. The ECO Synthesis platform addresses critical sustainability challenges of traditional manufacturing methods by eliminating the reliance on hazardous chemicals and significantly decreasing the use of acetonitrile,” Lutz says.

Ecolab—which provides contamination control strategies and solutions, water management programs, and resins for downstream purification—uses greener chemistry principles, material and energy sourcing, and packaging initiatives, according to Christine Yore, vice president, Enterprise Corporate Accounts, Ecolab Global Life Sciences. The sustainable manufacture of biopharmaceuticals is challenged by high water consumption, refrigeration energy demands, and chemical and plastic waste generation, according to Yore. Closed-loops systems and water recycling are key to combat these challenges. The company’s Water for Climate program offers a way for companies to optimize water use across operations, therefore reducing water consumption and minimizing the impact on the environment. “We are also seeing that Digital technology is also playing a key role in sustainability initiatives,” says Yore. “Our Ecolab 3D technology provides key insights to our customers by tracking and monitoring water quality and water flow.” Yore also points to the company’s ion-exchange resin and jetting technologies and its cleaning, disinfection, and decontamination products that help improve operations while reducing environmental impacts. Ecolab has committed to sourcing enough clean electricity via a wind farm to power all of its European operations by 2030. Additionally, in 2023, responsible packaging practices at its facilities avoided 14.5 million pounds of virgin plastic use.”

Verdot, which supplies purification systems for the manufacture of biopharmaceuticals and gene therapies, has been proactively anticipating the sustainability requirements of the companies that use their systems by embedding sustainability into their operations, according to Charles Ruban, president and CEO, Verdot. As part of its sustainability strategy, the company launched carbon compensation initiatives in 2025.

“We have achieved one of the lowest carbon footprints, averaging 100 tons equivalent carbon of equivalent carbon per billion euro in revenue,” says Ruban. “And we’ve done that through eco design, you know, really optimizing the material usage of our products and really selecting appropriately the sustainable supplier that we wanted to work with. … [we are] designing energy efficient equipment that [optimize] material use, and we’re partnering with suppliers committed to systemic sustainability, so our carbon neutral initiative reflects the commitment of overcoming those models while enabling compliance to globally reduce their environmental footprint.”

®
eBook: Bio/Pharma Outsourcing Innovation 2025
February 2025
Pages: 20

When referring to this article, please cite it as Haigney, S. Supplying Sustainably. 

Sponsor companies and contract development and manufacturing companies must ensure that the suppliers they use are following sustainability guidelines to truly produce ‘green’ products.

Supplying Sustainably

Circular economy concept. Energy consumption and CO

 emissions are increasing. Sharing,reusing,repairing,renovating and recycling existing materials and products as much possible. | Image Credit: ©Miha Creative- stock.adobe.com

The manufacture of pharmaceuticals involves active chemicals and other materials that may impact the environment. The latter half of the 20th century saw a surge in a movement to reduce climate change that continued into the 2000s. In 2015, the Paris Agreement put forth principles to keep global warming under 2 oC by 2050 (1). In 2019, the European Union introduced the European Green deal that committed Europe to become climate neutral by 2050 (1). In 2014, according to IQVIA, the healthcare industry contributed to 4.4% of the world’s carbon footprint (1). In response to these and other initiatives, the pharmaceutical industry has been working toward more sustainable manufacturing practices.

Working Together to Improve the Environment

The push toward sustainability in pharmaceutical manufacturing also is seen in the increased requirements put on contract development and manufacturing organizations (CDMOs) by sponsor companies to follow sustainability practices, as contract organizations play a significant role in the development and manufacture of drugs.

“As significant third-party suppliers, CDMOs play a major contributing role in their sponsors’ overall emissions,” says Niall Harkin, executive director of Almac Group. “These requirements are a factor in negotiating partnerships as sponsors increasingly request evidence of sustainable or green manufacturing, and documenting these is now the norm on any new RFPs [request for proposals].”

“When we’re negotiating and contracting with our partners, they’re wanting to work with companies who do have ESG [environmental, social, and governance] sustainability policies in place,” says Andy Burns, vice president, MDI (Metered Dosed Inhaler) Business Development at Kindeva. Pharma companies that are more established have entrenched sustainability procedures, according to Burns, and those companies often require contract manufacturing and development organizations to follow those procedures before signing a contract.

According to Rajendra Kumar Sahu, chief executive officer of Navin Molecular, standards required by sponsor companies may include EcoVadis certification and a minimum requirement of a bronze medal at the CDMO’s last assessment. EcoVadis, founded in 2007, provides business sustainability ratings of more than 150,000 companies (2).

Sponsors may also require science-based targets to reduce carbon emissions and the reporting of scope 1 and 2 carbon emissions. “Such importance is placed on these [criteria]; customers are beginning to refuse to work with suppliers that do not meet at least one of these standards, and there are a number of companies making it clear that suppliers will be expected to be able to meet all three requirements/standards in the near future,” stresses Sahu.

“We used to see only Big Pharma or large generic drug companies having specific sustainability requirements. But now a lot of small to mid-sized biotech companies are talking to us and evaluating us on our sustainability related practices,” says Saharsh Davuluri, vice-chairman and managing director, Neuland Laboratories. “One of our largest generic customers has required us to apply for the SBTi [Science Based Targets initiative] validation, which is something that we are seeing other companies follow as well. On the innovator side, we’re seeing some of the large innovators enquire about our EcoVadis rating, beyond the standard frameworks of assessments that are available.”

®
eBook: Bio/Pharma Outsourcing Innovation 2025
February 2025
Pages: 12–20

When referring to this article, please cite it as Haigney, S. Working Together to Improve the Environment. 

Contractors are working with sponsor companies to establish and maintain sustainable manufacturing practices in pharma.

Feliza Mirasol is the science editor for 

Next-generation biotherapeutics pose challenges to the development of manufacturing processes and analytical characterization because of their complexity and, in some cases, novelty. From cell and gene therapies (CGTs) and new vaccines to antibody-drug conjugates (ADCs), successful development and commercialization of these next-generation therapies may hinge on partnerships with service providers that offer specific focus, expertise, and technological experience. At certain points in the discovery and development process, developers—especially smaller firms—come to realize the unmet needs that prompt them to seek an outsourcing provider and/or partnership.The first order of business would be to bring in a partner with the correct expertise and technology to tackle the challenges of bringing a complex biotherapeutic to market. 

Outsourcing Next-Gen Therapies Relies on Expertise and Technology

Bringing next-generation therapies to market brings up multiple challenges, according to Monika Paule, CEO, Caszyme. These challenges range from scientific and technical hurdles to regulatory and societal barriers. “One of the major obstacles is the social perception and acceptance of novel technologies such as CRISPR [clustered regularly interspaced short palindromic repeats]. Addressing public concerns, providing accurate information, and engaging in meaningful dialogue about the benefits, risks, and ethical considerations of these technologies are crucial,” Paule says.

Ali Pashazadeh, CEO, Treehill Partners, a transaction and drug development advisory firm focused on the biotech industry, points to the fact that the biopharmaceutical industry is currently experiencing its fourth year of financial drought, which has been one of the biggest challenges lately to bringing next-generation therapies to market. “Whilst next-generation technologies are incredibly exciting in their potential, these really require, for the first time, excellence in drug development to be combined with excellence in manufacturing,” Pashazadeh says.

Speaking specifically on nucleic acid-based therapies, Bernard Sagaert, CEO of etherna, emphasizes that the challenges are still big, even after the success of the COVID-19 vaccines. The challenges on the RNA front are tied in with RNA quality, where sometimes an inferior quality of RNA is used, which always leads to project failure in discovery or preclinical stages, cost of goods, or duration of expression, he explains. “On the delivery side, targeted expression and off-target expression reduction, balancing immune expression (regulate up or down, or be immune-neutral), and stability are the main challenges. All of these challenges can be different based on the therapeutic area where the products need to be used, whether it is in prophylactic or therapeutic vaccines, autoimmune diseases, gene editing, oncology, protein replacement, etc.,” Sagaert says.

Meanwhile, other challenges include technical and regulatory complexities, such as optimizing gene-editing precision and efficiency, ensuring scalable and cost-effective manufacturing, navigating complex regulatory requirements, and addressing safety and ethical considerations. “Overcoming these hurdles requires broad scientific expertise, strong analytical capabilities, and strategic collaborations among key ‘players’ to accelerate development while minimizing risks,” says Paule.

For the most part, it is the complexity of manufacturing involved in next-generation biotherapies that drives outsourcing in this sector. “These therapies require specialized expertise in areas such as gene and cell therapy production, advanced analytics, and regulatory compliance—expertise that many biopharma companies do not have in-house. Of course, everyone would love to do everything in-house, but the amount of initial investment needed, the need for speed in development, cost efficiency, and enhanced scalability are the main factors driving outsourcing,” says Paule.

“A certain level of complexity multiplies directly in line with” the scale of manufacturing capacity,” adds Jason Jones, global business development lead, Cellular Origins. Jones explains that if processes are complex, variable, and expensive when manufacturing 1000 doses of a next-generation therapy, then these processes will be 100x more complex, variable, and expensive if they are continued to be produced the same way as production scales up 100x.

When referring to this article, please cite it as Mirasol, F. Outsourcing Next-Gen Therapies Relies on Expertise and Technology. 

The value of outsourcing partnerships for next-gen biotherapeutics rests in the expertise and technologies that service providers bring to the table.

Delegating concept. Wooden figurines and arrows as symbol of delegation. | Image Credit: © Vitalii Vodolazskyi - stock.adobe.com

The market research and consulting firm DelveInsight predicts that a global contract development and manufacturing organization (CDMO) market already valued at more than $100 billion could potentially grow by almost half that amount, to an eventual total value of nearly $144 billion, over the second half of the 2020s.

That expansion across the market, which represents a theoretical compound annual growth rate of 6.5%, not only reflects the rise in some serious diseases, various cancers among them, but also the increasing popularity of outsourcing in the drug development process—which, DelveInsight said, comes with certain benefits in cost savings.

The firm was even retained by an unnamed “large pharma client,” looking to improve its API and finished dosage development, to produce a one-off report with the objective of benchmarking top CDMO candidates for outsourcing its projects, analyzing the CDMOs’ R&D, manufacturing, and commercializing capabilities.

Such attention to and emphasis on this research underlines the importance the bio/pharma industry is currently investing in its outsourcing decisions, and as the CDMO market continues to grow, the details of those decisions figure to get more and more precise.

To that end, when CPHI shared its top outsourcing trends for CDMOs across the globe for 2024, several key themes emerged. Chief among these were more careful consideration of geographic diversification and its implications, end-to-end or “one-stop-shop” organizations being able to improve efficiency and scalability, market shifts increasingly driven by merger and acquisition (M&A) activity, and equal responsibility between the drugmaker and the CDMO/contract research organization (CRO) engaged in a partnership—also, to a certain extent, similarly shared risk management responsibility between CDMO/CRO and customer.

Increasingly, the responsibilities referenced in seeking these potential partnerships are centered on sustainability. 

® conducted a series of interviews in January 2025 that explored this aspect, 

perhaps best encapsulated by Saharsh Davuluri,

 vice chairman and managing director for Neuland Laboratories.

“Sponsors are increasingly requiring sustainable or green manufacturing practices when negotiating for partnerships with Neuland,” Davuluri said in the interview. “I think this trend is also kind of aligned with the broader industry shift toward sustainability … we are seeing that a lot of these companies are having their own approach ... or their own frameworks in what they would like to measure their partners with. This is not just about Europe versus [the United States], but also within a particular geography, every company has a different approach.”

As the 2025 market finds its footing, we present a non-exhaustive list of partnerships announced, developed, or forged so far in the year to date, representing companies around the world offering a wide variety of specializations. With this list summarizing only a fraction of the collaborations that have progressed in just mere weeks’ time so far in 2025, there will be plenty to watch for in the months ahead.

When referring to this article, please cite it as Lavery, P. Savings and Sustainability Sought by Pharma Partners. 

With 2025 now in full swing, this article presents a review of the latest developments in outsourcing services, new facilities, and M&A activity thus far this year.

Savings and Sustainability Sought by Pharma Partners

FDA has granted approval to California-based Mirum Pharmaceuticals for Ctexli (chenodiol) for treatment of the genetic metabolic disorder cerebrotendinous xanthomatosis (CTX) in adults. FDA announced the decision in a press release on Feb. 21, 2025 (1).

Ctexli becomes the first FDA-approved drug to treat CTX, which is a rare lipid storage disease that is caused by a mutation in the CYP27A1 gene (1). This results in a deficiency of an enzyme that is important, FDA said, insofar as the body’s ability to break down cholesterol in a normal way, exacerbated by the gene mutation causing reduced bile production in the liver. When that happens, atypical cholesterol metabolites are deposited and cause damage in places in the body such as the brain, liver, skin, and tendons.

“FDA is dedicated to supporting new drug development for rare diseases including very rare metabolic diseases like cerebrotendinous xanthomatosis,” said Janet Maynard, MD, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in FDA’s Center for Drug Evaluation and Research, in the press release (1). "CTX is a progressive multisystemic disorder that significantly impacts patients and previously lacked approved treatments. Today’s approval provides a safe and effective treatment option for CTX.”

 around DCAT Week 2023, Roger Viney, chief commercial officer at ICE Pharma, spoke about the history of bile acid therapeutics up to the current day (2).

“The last 20 years have seen a huge growth of medicinal research in bile acids. As recently as 1999, the scientific community recognized that bile acids are metabolic regulators—and this opened a whole new area of research into bile acid-based medicines ... [including] cholic acid, deoxycholic acid, chenodeoxycholic acid, their salts and taurine and glycine conjugates, which find diverse uses from nutraceuticals to excipients and APIs,” Viney said at the time.

Another treatment for CTX, Sigma-Tau Pharmaceuticals’ chenodeoxycholic acid candidate, was given a positive opinion by the European Medicines Agency’s Committee for Medicinal Products for Human Use 

But in 2021, after Sigma-Tau had rebranded as Leadiant Biosciences, the Netherlands Authority for Consumers and Markets 

 (US$23 million as of 2021) against Leadiant for excessive pricing of its CTX drug—a price the company had raised by 1800%—as part of a regulatory examination of infringements of the Competition Act of 1998 (4).

Meanwhile, two other drugs designed to treat another rare disease affecting the liver, primary biliary cholangitis (PBC), are moving forward early in 2025. Cour Pharmaceuticals 

 from FDA in January 2025 for its CNP-104, which is a biodegradable nanoparticle that encapsulates the E2 component of the mitochondrial pyruvate dehydrogenase complex, a key autoantigen in PBC (5). And in February 2025, Gilead Sciences was 

granted conditional marketing authorization

 by the European Commission for seladelpar, an oral, peroxisome proliferator-activated receptor-delta agonist (6).

Concerning the approval of Ctexli, FDA said in its press release that the recommended dosage for chenodiol is 250 milligrams, taken orally three times a day (1). Prescribing information includes a warning for liver toxicity in all patients, and increased risk of liver damage in patients with pre-existing liver disease or bile duct abnormalities.

1. FDA. FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease. Press Release. Feb. 21, 2025.
2. Playter, G. 

Exploring Bile Acid Therapeutics with Roger Viney (DCAT Week).

, May 1, 2023.
3. Pharmaceutical Technology Editors. 

, Sept. 16, 2016 (accessed Feb. 24, 2025).
4. Thomas, F. Drug Pricing Scrutiny Increases in Europe. 

, 33 (8), 6.
5. Cour Pharmaceuticals. Cour Pharmaceuticals Secures FDA Orphan Drug Designation for CNP-104 in Primary Biliary Cholangitis. Press Release. Jan. 8, 2025.
6. Gilead Sciences. Gilead’s Seladelpar Granted Conditional European Marketing Authorization for the Treatment of Primary Biliary Cholangitis. Press Release. Feb. 20, 2025.

Cerebrotendinous xanthomatosis, or CTX, is a metabolic disorder caused by a gene mutation that results in a deficiency of an important enzyme in the ability of the human body to break down fats.

FDA Approves First Treatment for Rare Lipid Storage Disease Cerebrotendinous Xanthomatosis

Gilead Sciences, headquartered in Foster City, Calif., announced on Feb. 20, 2025 that its seladelpar, an oral, peroxisome proliferator-activated receptor (PPAR)-delta agonist, has been granted conditional marketing authorization by the European Commission (EC) for treatment of the rare liver disease primary biliary cholangitis (PBC), either in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA alone, or as a monotherapy in patients unable to tolerate UDCA (1).

In December 2024, seladelpar was among five drugs recommended to be an orphan designated product 

 green-lighted for market authorization by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) (1,2). It received approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) in January 2025 (1). The EC’s series of decisions concerning seladelpar was based on results of a placebo-controlled Phase III trial (RESPONSE), in which 62% of those who took seladelpar achieved the primary endpoint of composite biochemical response after 12 months, versus 20% of those taking placebo (1).

The effectiveness of treatments for PBC is commonly marked by an improvement in liver biochemical tests, particularly in the normalization of alkaline phosphatase (ALP) levels; ALP values normalized in 25% of trial participants taking seladelpar at month 12, while there was no normalization in those given placebo (1). Improvement of pruritus, or chronic itch, one of the most common symptoms of PBC, was also more pronounced after six months of treatment with seladelpar than with placebo, according to a Gilead press release (1).

“People living with PBC in Europe have been waiting for treatment advancements for many years. Up until now, there has been no approved treatment for PBC addressing both the surrogate biomarkers for underlying disease and pruritus, a common and at times debilitating symptom of PBC,” Timothy Watkins, MD, vice-president, Clinical Development of Inflammation Therapeutics for Gilead Sciences, said in the press release. “We look forward to working with health authorities across Europe to bring this promising new treatment to all those who could benefit.”

PBC is defined as a rare, chronic, and autoimmune disease of the bile ducts, for which there is no cure. In Europe, it affects approximately 22 out of every 100,000 people, according to Gilead (1). Women are more commonly affected than men, and the disease if left untreated can cause liver disease and ultimately liver failure.

As mentioned, the pruritus and fatigue which are often symptoms of PBC—related to cholestasis, or impaired bile flow—can be debilitating. In lieu of a cure, current treatment options include slowing disease progression and mitigating symptoms.

“Today’s decision reinforces the clinical benefit and value of seladelpar and offers people living with PBC in Europe an important new treatment option,” María-Carlota Londoño, MD, PhD, a hepatologist at Hospital Clinic Barcelona, said in the press release. “There are people in Europe who do not have an adequate response to first-line therapy, and seladelpar helps address the unmet need for effective and symptom-directed treatment.”

Continued authorization of seladelpar in the European Union, Gilead said, will be contingent on the verification and description of its clinical benefits in confirmatory trials (1).

Marketed as Livdelzi in the United States, seladelpar was 

 by FDA in August 2024 (1,3). It is currently under regulatory review in Canada and Australia (1).

1. Gilead Sciences. Gilead’s Seladelpar Granted Conditional European Marketing Authorization for the Treatment of Primary Biliary Cholangitis. Press Release. Feb. 20, 2025.
2. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 9–12 December 2024. Press Release. Dec. 13, 2024.
3. Gilead Sciences. Gilead’s Livdelzi (Seladelpar) Granted Accelerated Approval for Primary Biliary Cholangitis by US FDA. Press Release. Aug. 14, 2024.

Seladelpar, which is marketed in the US as Livdelzi, was recommended for an orphan drug designation by CHMP in December 2024.

Gilead Treatment for PBC Granted EC Conditional Marketing Authorization

Incyte, of Wilmington, Del., has announced a strategic collaboration with California-based Genesis Therapeutics for the purposes of researching, discovering, and developing novel small-molecule medicines, making use of generative and predictive artificial intelligence (AI) technology to help create therapeutics for various targets (1).

According to a Feb. 20, 2025 press release, the targets selected for the collaboration will be chosen by Incyte, and Genesis’ proprietary AI platform, known as GEMS (Genesis Exploration of Molecular Space), will be used to discover and optimize small-molecule compounds for those targets (1).

“AI has the potential to redefine how we discover small molecule medicines, and our team is at the forefront of this revolution,” said Evan Feinberg, PhD, founder and CEO of Genesis, in the press release. “We are pleased to establish this world-class partnership to combine our GEMS AI platform with Incyte’s deep expertise and track record in drug discovery and development, with the shared goal of advancing critical treatments for patients with severe diseases.”

Incyte will retain exclusive rights for potential clinical development and commercialization of any products to be produced as a result of the collaboration, according to the release (1).

“As a leader in pharmaceutical innovation, Incyte is continually seeking new technologies that can transform how new medicines are discovered and developed,” said Pablo J. Cagnoni, MD, president and head of R&D at Incyte, in the press release. “Partnering with Genesis Therapeutics presents a unique opportunity to leverage their AI technologies to accelerate the discovery of breakthrough small molecules for high-impact targets in our pipeline.”

 that the rise of AI was one of the key market trends of 2024, and it will continue to be through 2025, but that it is still “in its infancy” and must be improved upon using a data-driven approach (2). Women in 

science, technology, engineering, and mathematics,

 or STEM, and their related industries are regarded as crucially important to developing AI regulations (3).

And as the first quarter of 2025 progresses, the industry is continuing to adapt. ArisGlobal, a Boston-based life sciences technology company, 

 that it had signed the European Commission’s AI Pact, a voluntary framework to prepare stakeholders for the implementation of the European Union’s AI Act (4). And in a Google Blog post on February 19, 

 a multi-agent AI system built with Gemini 2.0, intended to be used as a collaborative tool by scientists and designed to mirror the reasoning process of the scientific method (5).

Regarding the agreement between Incyte and Genesis, the February 20 press release said Genesis would receive a $30 million upfront payment, and the two companies will collaborate on two initial targets, with Incyte authorized to nominate a third target for a predetermined fee (1). Genesis may receive up to $295 million in development, regulatory, or commercial milestone payments, per target, if all milestones are achieved, and will receive tiered royalties on sales of any products which result from the collaboration.

1. Incyte. Incyte and Genesis Therapeutics Announce Strategic AI-focused Research Collaboration. Press Release. Feb. 20, 2025.
2. Thomas, F. 

Industry Outlook 2025: Experts Weigh in on the Most Impactful Trends for Industry.

, Feb. 11, 2025.
4. ArisGlobal. ArisGlobal Signs EU AI Pact in Latest Commitment to Ethical AI Practices. Press Release. Feb. 12, 2025.
5. Gottweis, J. and Natarajan, V. Accelerating Scientific Breakthroughs with AI Co-Scientist. Blog Post. Google Research. 

The collaboration will utilize Genesis’ proprietary AI platform, initially focusing on targets selected by Incyte.

Incyte, Genesis Therapeutics to Use AI Platform to Discover Small Molecule Compounds

Data from a clinical study have shown that CBtru, a cannabidiol (CBD) drug product intermediate from DSM-Firmenich, is absorbed into the bloodstream at a similar level to Jazz Pharmaceuticals’ Epidiolex, the first and currently only commercially approved CBD drug product on the market (1). According to DSM-Firmenich (stylized as dsm-firmenich), it is the first time a solid CBD formulation has demonstrated absorption comparable to a liquid/oil-based reference product.

DSM-Firmenich, which has headquarters in The Netherlands and Switzerland, said in a Feb. 20, 2025 press release that it developed CBtru to have enhanced bioavailability in a solid powder form with a considerably higher drug loading, to combat what the company said is the limited solubility and variable bioavailability of CBD—as low as 6% because of factors such as incomplete absorption in the gut and a significant level of pre-systemic elimination in the liver (1). Epidiolex, by being offered in a liquid, sesame oil-based formulation, is afforded the higher solubility and absorption of CBD typically associated with a high-fat meal, as lipophilic molecules can dissolve in fat content from food.

Oral solid dosage (OSD) forms, however, are desirable for patients who wish to overcome accurate dosing and sensory challenges with oil-based CBD therapies, according to DSM-Firmenich (1). Such products have entered the market for the primary purpose of managing treatment-resistant epileptic syndromes.

The clinical study (NCT06578455) that compared CBtru to Epidiolex involved 32 subjects, divided evenly between men and women, between the ages of 19 and 55 (1). Four treatment phases were conducted, with participants receiving 400 mg CBD doses of both therapies—CBtru in capsule form and Epidiolex via syringe—in both fed and fasted conditions, separated by a two-week washout period after administration. Plasma concentration was analyzed at 24 hours post-treatment, and findings suggested that the DSM-Firmenich product may offer a more consistent, reliable uptake while relying less on food intake.

“These findings represent a significant breakthrough in the CBD space—and the wider pharmaceutical market,” Zdravka Misic, PhD, associate innovation director—Pharma at DSM-Firmenich, said in the press release. “With the data indicating that CBtru is at least as bioavailable as the market-leading liquid oil-based reference product, we’ve opened new pathways for the development of CBD-based therapies using solid oral dosage forms. This is truly innovative, because it paves the way for more patient-friendly delivery systems, such as tablets or capsules. These dosage forms help enhance efficacy, while simultaneously increasing patient convenience and compliance. What’s more, beyond improving patient experience, this breakthrough could accelerate research in therapeutic areas where CBD has shown potential, such as pain management, inflammation, cancer, diabetes, as well as sleep, mood, and psychotic disorders.”

 for a CPHI Pharma Award in the Sustainability category, for its “Sustainability Imp’Act Card,” the digital version of which was launched in 2023, according to the company’s website (2,3). The Imp’Act Card, which is available for 80% of DSM-Firmenich’s beauty and care products, is based on the United Nations Sustainable Development Goals and other industry standards, measuring products’ environmental and social impacts, traceability, and identity, in the interest of providing transparency throughout the wider value chain (3).

However, Schneider Electric ultimately won the Sustainability award 

 for its Energize program, which leverages the scale of the global supply chain in a pre-competitive fashion to drive system-level change (4).

1. DSM-Firmenich. New Clinical Study Validates dsm-firmenich’s CBtru as a Promising Pathway for Patient-Friendly CBD-Based Therapies. Press Release. Feb. 20, 2025.
2. Mirasol, F. 

Pharma Award Finalists for CPHI Barcelona Have Been Announced.

, Sept. 19, 2023.
3. DSM-Firmenich. Sustainability. 

 (accessed Feb. 20, 2025).
4. Haigney, S. 

Winners of the 2023 CPHI Pharma Awards Announced.

A capsule formulation may offer more consistent and reliable uptake versus a syringe, while being less dependent on food intake.

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