Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Navigating FDA Shakeups and Obesity Drug Wars

Behind the Headlines Episode 28

Ask the Expert

Closing the Gap in Modern Process Control and Automation for QC/QA

Drug Digest December 2025

How It Helps Avoid Facility and Equipment Problems

A “Plan, Prevent, Prove” Approach

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

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Nelson Labs, headquartered in Salt Lake City, Utah under the umbrella of Ohio-based Sotera Health, announced on March 4, 2025 that its rapid sterility testing approach, a way of performing product-sterility testing through rapid microbiological methods (RMMs), is now being offered at three of its laboratory sites: in Salt Lake City and Itasca, Ill. in the United States and in Europe at a site in Wiesbaden, Germany (1,2).

In a press release, Nelson Labs said its testing solution is unique because it supports a wide range of both pharmaceutical products and medical devices, broadening the scope of sterility testing using RMMs, which until now, according to the company, has mostly focused on those products that have biological components or a short shelf life (1). Rapid sterility testing can further expand capabilities to testing products that require prompt release to market or are manufactured in small batches (2).

Sterility testing ensures product safety by detecting microbial contamination, according to Nelson Labs (2). 

) General Chapter <71>, which the company said has guided previous product-sterility testing, specifies a customary 14-day incubation period in growth media to allow contaminants to grow, followed by a visual inspection (1,3). This length of time can expand to 18 to 20 days with a sub-culture (2). Nelson Labs’ rapid sterility testing—depending on product-specific validation—is designed to reduce the incubation time to as little as six days, although the company said it cannot guarantee specific outcomes (1). The testing complies with 

 <71> as well as <1223>, for validating alternative microbiological methods, according to Nelson Labs (1,4).

“We’re thrilled to offer rapid sterility testing as part of our sterility-testing solutions for our customers,” Nina Moreno, global segment director of sterility assurance at Nelson Labs, said in the press release (1). “This cutting-edge technology significantly reduces testing times, allowing for faster product release while maintaining the highest standards of quality and safety.”

Information provided on Nelson Labs’ website details the process of rapid sterility testing, using advanced bioluminescence technology to detect microbial adenosine triphosphate—in this case, light being produced at levels elevated enough to indicate contamination (2). The instrumentation used, according to the company, replaces subjective visual inspections for turbidity, or the presence of contamination, with quantitative results, in addition to having a high capacity for throughput and being easy to perform. In practice, this has reduced testing time by at least half, Nelson Labs said (2).

Nelson Labs said it is offering its team of experts at an affiliated company, Regulatory Compliance Associates, to assist customers in the medical device and pharmaceutical drug manufacturing fields with validation and regulatory submission support, in the interest of bringing products to market in an expedited manner (1).

® Drug Digest video, which features Ask the Expert columnist Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates.

1. Nelson Labs. Nelson Labs Launches Innovative Rapid Sterility Testing to Significantly Expedite Product Sterility Results. Press Release. March 4, 2025.
2. Nelson Labs. Product Sterility–Rapid. 

, 933–940 (Rockville, MD, 2003).
4. USP, 

 <1123>, “Validation of Alternative Microbiological Methods,” 

Articles

The company now performs product-sterility testing through rapid microbiological methods at two laboratory sites in the United States and one in Germany.

Nelson Labs Launches Rapid Sterility Testing, Cutting Incubation Times

Strong leadership in biotechnology requires a balance of inspiration, discipline, and the ability to foster innovation within structured processes. Xiao-Yan Cai, PhD, co-founder and CEO of Accurant Biotech, guides her team with a straightforward yet motivational approach, as evidenced in the following interview with 

®. She discusses her leadership style, strategies for maintaining employee engagement, and how personal passions—such as competitive ballroom dancing and Taekwondo—inform her professional mindset. Cai also highlights the necessity of repetition in mastering any skill, whether in the lab or on the dance floor, reinforcing the idea that persistence fuels innovation.

 Can you tell us more about your leadership style and how you motivate your team?

 You can see my style when I'm leading the team. I often tell people I [have] a more principled thinking style … it's a bit binary; it's ‘yes’ or ‘no,’ it's ‘this’ or ‘that.’ If you think in such a manner, it would be straightforward. You can get to the root cause. So, this is why I'm a very good troubleshooter, because you don't want to complicate your situation. You can further dive it down into the final, only binary situation.

 What strategies do you use to help your employees develop and excel in their roles?

 I believe leading has two styles ... One, I call “inspiration style.” [The other] is called “desperation style.” Desperation style is whipping, forcing, threatening you—'you do this, otherwise you won't get paid’—this sort of connotation. I clearly prefer to do inspirational; I would lead by example, by showing you that 

 want to have a career. You want to be better tomorrow than today ... I don't focus on, ‘So, what is he doing? So how come he's not doing anything? How come he's getting promoted?’ None of that. I'm focusing on my own improvement, my own progress ... I coach my employees in such a manner, inspirational. That's how I believe everyone should. If you can even temporarily focus on this, it will become very exciting when you go to work, in terms of mundaneness.

 Let’s talk about mundaneness—how do you encourage your team to find excitement and innovation in repetitive work?

 [Earlier in my career I] moved from the Schering-Plough discovery zone to the development zone. There is also a common understanding, [a] perception, that discovery is slightly superior than development. It was your truth in the company. So, I went backwards one more time. Employees even asked me, with your credentials and your resume, why are you coming here? My question back to them: So, you think I am so excitingly doing these discovery things because I was a cloner? I cloned genes? Most people think, ‘Oh my God, that's so awesome.’ I said, what do you think the rocket scientist is doing every day? [A] rocket scientist is so exciting, so amazing. Oh my God, that's like the elite, top scientist perceived, right? So, what do you think they do every day? You also have a field of study, and then each day, you can innovate all you want, but there has to be a mundaneness to the component. I can say this because cloning was also perceived back in the day as a ‘holy cow’-ish skill. What do you think I'm [doing] cloning a gene? What am I doing every day? ... What do you think we're doing every day? In order for anyone to be superb at what they do, there has to be repetitiveness. You wouldn't be good without that piece. [But] that doesn't stop you from innovating.

 What role do your personal interests and activities play in your professional life?

 I always [tell people], oh, I started when I was a toddler—not true. I was not a genius. I'm pretty smart, but ... I never jumped a grade. I'm a normal person. Graduated the same age as everyone else. Never stayed behind either, so I was just on par. But I know I look young, and, because I'm very fit, I treat everything in my life this way. I'm a competitive ballroom dancer. I rank in national competition in my age group. I'm going to the final second-degree black belt Taekwondo. I treat everything I touch with passion, with energy, with positive thinking. With this understanding [of] the repetitiveness. Because what does not need repetitiveness. Ballroom dancing, are you kidding me? It's repetitive. You need to train your muscles to remember this, how to move, everything has repetitiveness, but everything has innovation as well, so that's how I feel. If everybody would do this way, your life would be a happier life. You come to work happy every day.

How do you personally stay informed and engaged with the latest industry developments, even in the face of challenges?

 [During the COVID-19 pandemic] I traveled to China, quarantined, high-risk, 14 plus seven plus seven [days of isolation] in order for me to go to Beijing to see my mom. I've done that. Most people think it's impossible. I think you set your mind, understand where you're going into. Prepare. I'm very prepared. I brought my yoga mats, jump ropes, juggling balls. I'm just crazy. I like to play. I brought sunflower seeds because it's one of my snacks. And so, you prepare. In the hotel room, you have a wi-fi. I can work, no complaint. They provide food, the breakfast and lunch and dinner. Great, I got my food, I'm not starving today. I walked in my room every day, achieved my goal of 10,000 steps every day.

® following AAPS PharmSci 360 in October 2024.

AAPS PharmSci 360 2024: The Future of Bioanalysis

Xiao-Yan Cai, PhD, shares insights into her leadership style, the importance of motivation in the workplace, and how she balances repetition with innovation, discussing how hobbies and resilience uniquely shape her professional approach, emphasizing the value of perseverance and preparation in both science and life.

Inspiration Over Desperation: Accurant Biotech CEO Talks Leadership, Innovation, and Motivation

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has given a positive opinion and recommendation for marketing authorization to Fabhalta (iptacopan), a Novartis treatment for adults with C3 glomerulopathy (C3G) (1). 

Highlights from CHMP’s February 24–27 meeting

 described C3G as “ultra-rare” and clarified that Fabhalta has already been granted orphan drug designation (1,2).

Novartis announced CHMP’s decision in a press release on February 28, which was the 2025 edition of 

 Since 2008, this occasion has been recognized on 

 including February 29 in leap years—the rarest date on the calendar.

C3G is a progressive kidney disease often affecting young adults, and the prognosis is poor, according to Novartis; approximately half of patients progress to kidney failure within 10 years of diagnosis and thereafter require lifelong dialysis and/or a kidney transplant (1).

CHMP’s opinion was based on data from a randomized, placebo-controlled Phase III study (APPEAR-C3G) that Novartis said achieved a statistically significant and clinically meaningful 35.1% reduction in proteinuria using iptacopan versus placebo after six months (1).

“C3G has no approved treatments, and patients face challenges with current options,” David Kavanagh, professor of complement therapeutics and honorary consultant nephrologist at the National Renal Complement Therapeutics Centre at Newcastle University, UK, and APPEAR-C3G Steering Committee Member, said in the Novartis press release (1). “With its strong body of evidence, oral Fabhalta targets the underlying cause of C3G in both native and recurrent patients and can bring hope to patients who currently have a poor prognosis.”

As Kavanagh mentioned, Fabhalta is an oral, Factor B inhibitor of the alternative complement pathway that previously received FDA and European Commission approval in December 2023 and May 2024, respectively, for treatment of adults with paroxysmal nocturnal hemoglobinuria (1). FDA also granted iptacopan accelerated approval in August 2024 for reduction of proteinuria in adults with primary IgA nephropathy at risk of rapid disease progression. It is currently being studied, according to Novartis, for use in treating other rare kidney diseases such as atypical hemolytic uremic syndrome, immune complex membranoproliferative glomerulonephritis (IC-MPGN), and lupus nephritis.

In the Fabhalta C3G program, comprised of more than 100 patients, the drug was well-tolerated, according to Novartis (1).

“C3G is a debilitating condition, often affecting young people and severely impacting their physical and mental health,” Marianne Silkjær Nielsen, founder of CompCure, a Danish nonprofit committed to improving outcomes for individuals with C3G and IC-MPGN, said in the Novartis press release. “Screening to secure timely diagnosis and access to targeted treatments are critical for patients, their families and society. This milestone is highly welcomed by the patient community, marking progress toward better patient care for people living with C3G.”

“If approved, Fabhalta will be the first C3G treatment available for patients living with this severe progressive disease,” said David Soergel, MD, global head, Cardiovascular, Renal and Metabolism Development Unit, Novartis. “Building on our longstanding expertise in nephrology and recent advancements in kidney diseases, this positive CHMP opinion marks an important step forward for our exciting multi-asset kidney pipeline, underscoring our commitment to making meaningful progress for patients with unmet needs.”

1. Novartis. Novartis Oral Fabhalta (iptacopan) Receives Positive CHMP Opinion for the Treatment of Adults Living with C3 Glomerulopathy (C3G). Press Release. Feb. 28, 2025.
2. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 24–27 February 2025. Press Release. Feb. 28, 2025.

CHMP gave its opinion and a recommendation for marketing authorization to Fabhalta (iptacopan), an oral, Factor B inhibitor of the alternative complement pathway.

Novartis Treatment for C3G Gets Positive Opinion from EMA

Siegfried Schmitt, PhD, is Vice President Technical at Parexel International, Siegfried.Schmitt@parexel.com.

Question mark background | Image Credit: © Leigh-Prather - © Leigh Prather - stock.adobe.com

Q: There appear to be a lot of recent changes in the requirements for aseptic processing. Are there any other key developments in this field we should expect in 2025?

A: The two issues that will most likely be top of the list are compliance and competitiveness. Regarding compliance, although Annex 1, the European Union’s guide to the manufacture of sterile products (1), has been in force for over a year, many companies are still somewhat unsure as to how to comply with certain aspects of this document. In particular, establishing the contamination control strategy (CCS) proves to be more complex and resource intensive than many expected (2,3). Creating this document requires access to many disparate sources of information from several departments. Unless this information is accessible electronically, the task will be made excessively difficult.

When it comes to competitiveness, increasing demands from regulatory bodies, including those governing environment and health, place a burden on industry to find solutions (e.g., minimize waste even when using single-use equipment) that do not compromise compliance.

A good source to keep abreast of developing trends is to look at events and activities organized by leading industry associations that traditionally cover aseptic processing; for example, the International Society for Pharmaceutical Engineering (4,5) or the Parenteral Drug Association (6).

These and other organizations (7) are also providing fora for interested parties that prepare guidance documents (e.g., technical reports, good practice guides, points to consider, or similar) from industry experts for industry peers. Active participation in such groups provides early access to these highly valuable documents.

An area of special interest is to bring manufacturing as close as possible to the points of need or care. Rather than shipping product from a central manufacturing hub to a large number of locations, the idea is to establish more manufacturing sites, or even mobile manufacturing units (8) that will bring a lot more flexibility to manufacturing schedules, and deliver resilience to interrupting factors like environmental disasters or new trade barriers. Aseptic processing validation under these circumstances may need careful consideration.

Annex 1 Manufacture of Sterile Medicinal Products, 

Schmitt, S. Contamination Control Strategy–Are We There Yet? 

Schmitt, S. Creating a Contamination Control Strategy. 

ISPE. Building Resilience in ATMP Manufacturing: Insights and Innovations at the 2025 ISPE Aseptic Conference, 

PDA Good Aseptic Manufacturing Conference 2025

Regulatory Affairs Professionals Association. Fundamentals of Pharmaceutical and Biologics Regulations, A Global Perspective. 2nd edition, 

PDA. PDA Points to Consider Mobile Manufacturing, 2025. www.pda.org, [publication planned for 2025].

Siegfried Schmitt, PhD, is vice president, Technical at Parexel.

When referring to this article, please cite it as Schmitt, S. Developments in Aseptic Processing. 

Compliance and competitiveness are key factors in aseptic processing, says Siegfried Schmitt, PhD, vice president, Technical at Parexel.

Developments in Aseptic Processing

Mike Hennessy Jr. is the Chairman and CEO of 

High Angle Shot of a Working Desk of an Successful Person in Office with Cityscape Window View. | Image credit: © Gorodenkoff - stock.adobe.com

Technological advances are continuing to push boundaries for global industries. Artificial intelligence (AI) is one such advancement and is being increasingly employed in research and scientific work across multiple disciplines as a result of its ability to improve efficiencies.

A recent example of how AI is improving efficiency at the research stage has been demonstrated by co-scientist, Google’s new AI research tool, which solved a complex problem about superbugs—that had taken microbiologists a decade to work out—in a mere 48 hours (1). Google’s AI tool, built on Gemini 2.0, has been created to help scientists generate novel hypotheses and research proposals, which it does through a combination of specialized agents based on scientific methodology (2).

“The AI co-scientist represents a promising advance toward AI-assisted technologies for scientists to help accelerate discovery,” said Juraj Gottweis, Google Fellow, and Vivek Natarajan, Research Lead, in a blog post (3). “This project illustrates how collaborative and human-centered AI systems might be able to augment human ingenuity and accelerate scientific discovery.”

On the theme of collaboration, earlier this year, in January 2025, accelerated computing company, NVIDIA, announced new partnerships with IQVIA, Illumina, Mayo Clinic, and Arc Institute, to transform the healthcare and life sciences industry with AI (4). Using AI agents, NVIDIA’s partners are seeking to accelerate clinical trials, advance drug discovery, and support industry growth.

Looking a little further along the development lifecycle, towards the larger-scale commercial manufacturing end, the benefits of smarter technology and AI implementation are also being seen. However, as highlighted in this issue’s Focus piece, which can be found on pages 10–13, while technological advances are accelerating progress, there are still hurdles to overcome. “Plenty of challenges stand in the way, including cost of implementation, regulatory compliance, and more. We have heard from experts who anticipate that fully automated, ‘lights-out’ biomanufacturing facilities could become a reality within the next five to 10 years,” explains Anthony Christopher, commercial manager of bioprocessing at Kent Elastomer Products (5).

Gerken, T. AI Cracks Superbug Problem in Two Days that Took Scientists Years. 

Haigney, S. Google Launches AI Co-Scientist System. 

Gottweis, J.; Natarajan, V. Accelerating Scientific Breakthroughs with AI Co-Scientist. Blog Post. Google Research. Research.google. Feb. 19, 2025.

NVIDIA. NVIDIA Partners with Industry Leaders to Advance Genomics, Drug Discovery, and Healthcare. Press Release, Jan. 13, 2024.

Mirasol, F. Accelerating Adoption of Smart Tools to Advance Manufacturing. 

Mike Hennessy Jr is President and CEO of MJH Life Sciences®

When referring to this article, please cite it as Hennessy, M. Pushing Tech Boundaries. 

Artificial intelligence, among other technological advances, is pushing innovation boundaries.

Pushing Tech Boundaries

Eli Lilly and Company (Lilly) announced on Feb. 28, 2025 that its Jaypirca (pirtobrutinib), a non-covalent or reversible Bruton's tyrosine kinase (BTK) inhibitor, was given a positive opinion by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP), for treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) previously treated with a BTK inhibitor (1).

one of 16 drugs for which CHMP extended therapeutic indications

 at its February meeting, EMA said on February 28 (2).

"We are pleased to receive a positive opinion from the CHMP, signaling that the European Union may lead the way in broadening patient access to Jaypirca for those with relapsed or refractory CLL in the post-BTK inhibitor setting," said Jacob Van Naarden, executive vice president and president of Lilly Oncology, in a Lilly press release (1). "There are currently no treatment options that have been specifically studied in a randomized Phase III trial in this patient population, and we are hopeful Jaypirca will be a meaningful new option for patients. We look forward to the European Commission [EC]'s decision in the coming months."

It could be one to two months for that decision from the EC, according to Lilly, following the release of data from—as Van Naarden alluded to—a Phase III trial (BRUIN CLL-321) that is the first randomized study in CLL ever to be conducted specifically among patients previously treated with a BTK inhibitor (1). Included in those results was a 46% reduction in the risk of disease progression or death after treatment with pirtobrutinib versus two global standards of care, either idelalisib or bendamustine plus rituximab.

 at its July 2024 meeting, for treatment of not only CLL but also rheumatoid arthritis, in the form of Ituxredi, a biosimilar referencing Roche’s MabThera (3).

In its press release, Lilly detailed CLL as a slow-growing form of non-Hodgkin lymphoma developed from lymphocytes, and for which cancer cells are present in the blood (1). There are approximately 100,000 new global cases of CLL each year, and it is one of the most common types of leukemia in adults.

"Results from the BRUIN CLL-321 trial show that Jaypirca delivers clinically meaningful outcomes in a post-BTK inhibitor setting with markedly prolonged time to next treatment, including in those with high-risk characteristics often associated with poor prognosis," Paolo Ghia, MD, professor of medical oncology at Università Vita-Salute San Raffaele and IRCCS Ospedale San Raffaele, Milano, Italy, said in the Lilly press release (1). "Jaypirca allows for continued targeting of the BTK pathway following treatment with a covalent BTK inhibitor and has the potential to be an important new option in a setting with significant unmet need. The CHMP opinion is an important step toward bringing Jaypirca to patients in the European Union."

In the United States, Jaypirca was approved by FDA in 2023 under an accelerated approval pathway for treatment of adult patients with CLL or small lymphocytic lymphoma who have received at least two prior lines of therapy including a BTK inhibitor and BCL-2 inhibitor, or for treatment of adults with relapsed or refractory mantle cell lymphoma after at least two lines of systemic therapy (1).

 of four new US manufacturing sites, more than doubling its manufacturing investment in the country since 2020 to in excess of $50 billion total (4).

1. Eli Lilly and Company. Lilly's Jaypirca (pirtobrutinib) Recommended by CHMP for Approval in the European Union for Adults with Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) Previously Treated with a BTK Inhibitor. Press Release. Feb. 28, 2025.
2. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 24–27 February 2025. Press Release. Feb. 28, 2025.
3. EMA. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 22–25 July 2024. Press Release. July 26, 2024.
4. Eli Lilly and Company. Lilly Plans to More Than Double US Manufacturing Investment Since 2020 Exceeding $50 Billion. Press Release. Feb. 26, 2025.

Jaypirca (pirtobrutinib) has received a conditional marketing authorization from EMA for mantle cell lymphoma. The drug was approved in the United States in 2023 for several indications.

EMA Gives Positive Opinion to Lilly Treatment for Chronic Lymphocytic Leukemia

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February 28, 2025 marked the annual edition of Rare Disease Day, established in 2008 by the European Organization for Rare Diseases (EURORDIS) (1,2). On leap years, Rare Disease Day has been recognized on February 29, the rarest date on the calendar.

The day’s official website said that more than 70 patient organizations partner with EURORDIS on a yearly basis to help build an international community with the objectives of achieving “equity in social opportunities, healthcare, and access to diagnosis and therapies for people living with a rare disease”—some 300 million people worldwide, according to the site (1).

According to the Rare Disease Day website, a “rare disease” is defined as that which affects fewer than one in 2000 people (1). More than 6000 such diseases are known to exist, 72% of them being genetic in nature.

With February 2025 drawing to a close, positive trial results and regulatory approvals have been reported for numerous rare diseases. The following is a brief and non-exhaustive rundown of the latest developments:

While congenital deafness present at birth affects approximately 1.7 in 1000 children born in the United States, a specific condition caused by variations in the otoferlin (OTOF) gene is considered ultra-rare, according to Regeneron (3). That information was part of an announcement that the company’s investigational gene therapy, DB-OTO, showed 72-week progress in speech and development in a first child diagnosed at 10 months of age, and a “notable response” in 10 out of 11 other children enrolled in a Phase I/II study (CHORD) so far. A surgical procedure that administers DB-OTO is similar to that of a cochlear implementation, Regeneron said in a press release, allowing it to be deployed in young infants. DB-OTO has received orphan drug designation from both FDA and the European Medicines Agency, and additional fast track, rare pediatric disease, and regenerative medicine advanced therapy designations from FDA.

DM1 affects an estimated one in 8000 people, and there are approximately 40,000 patients in the US, 75,000 in Europe, and 15,000 in Japan (4). That’s according to Boston-based PepGen, which on February 24 announced positive initial results from a Phase I trial (FREEDOM-DM1) of its investigational candidate PGN-EDODM1.

DM1 is a monogenic, autosomal dominant, and progressive disorder primarily affecting skeletal, cardiac, and smooth muscles, as well as the central nervous system. In severe cases, life expectancy may be reduced due to the increased mortality rates that accompany pulmonary and cardiac complications—alongside the customary symptoms of muscle weakness, myotonia, cardiac and respiratory abnormalities, gastrointestinal problems, early cataracts, cognitive or behavioral issues, or fatigue (4).

CTX is a lipid storage disease caused by a mutation in the CYP27A1 gene. This results in a deficiency of an enzyme that is important 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. 

 that it had approved Ctexli (chenodiol), from California-based Mirum Pharmaceuticals, as the first drug authorized by the agency to treat CTX (5). The recommended dosage for chenodiol is 250 milligrams, taken orally three times a day. 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.

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

 that its seladelpar, an oral, peroxisome proliferator-activated receptor (PPAR)-delta agonist, was granted conditional marketing authorization by the European Commission for treatment of 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 (6). (The drug is marketed as Livdelzi in the US.)

The pruritus, or chronic itch, and fatigue that are often symptoms of PBC—related to cholestasis, or impaired bile flow—can be debilitating, and a Phase III trial showed that improvement of pruritus was more pronounced after six months of treatment with seladelpar than with placebo (6).

TGCT is a non-malignant type of tumor that forms within or near joints. It is caused by the dysregulation and subsequent overproduction of the CSF1 gene and, if recurring or left untreated, can damage or degenerate the affected joint and surrounding tissues, leading to the potential of significant disability. 

 Ono Pharmaceutical of Osaka, Japan announced that FDA had approved its subsidiary Deciphera Pharmaceuticals’ Romvimza (vimseltinib), a treatment in capsule form for adults with symptomatic TGCT for whom surgical resection would cause potential worsening functional limitation or severe morbidity (7).

Even though Rare Disease Day comes around just once annually, news on treatments for rare diseases is always developing, and is a genre worth following year-round.

1. Rare Disease Day. What is Rare Disease Day? 

 (accessed Feb. 27, 2025).
2. EURORDIS. Who We Are. 

 (accessed Feb. 27, 2025).
3. Regeneron. Latest DB-OTO Results Demonstrate Clinically Meaningful Hearing Improvements in Nearly All Children with Profound Genetic Hearing Loss in CHORD Trial. Press Release. Feb. 24, 2025.
4. PepGen. PepGen Announces Positive Initial Results, Including Robust Splicing Correction, from Ongoing FREEDOM-DM1 Trial in Patients with DM1. Press Release. Feb. 24, 2025.
5. FDA. FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease. Press Release. Feb. 21, 2025.
6. Gilead Sciences. Gilead’s Seladelpar Granted Conditional European Marketing Authorization for the Treatment of Primary Biliary Cholangitis. Press Release. Feb. 20, 2025.
7. Ono Pharmaceutical. U.S. FDA Grants Full Approval of Deciphera’s Romvimza (vimseltinib) for the Treatment of Symptomatic Tenosynovial Giant Cell Tumor (TGCT). Press Release. Feb. 14, 2025.

More than 6000 rare diseases—those which affect fewer than one in 2000 people each—are acknowledged in the medical community, with 72% of them being genetic.

Rare Disease Day Celebrated on February 28

Rare Disease Day, which is recognized on the final day of February each year—hence, February 29 in leap years, the rarest date on the calendar—shines a light not only on the 

6000 or more diseases that affect some 300 million people

 around the world, but also the medicines and therapies in development to help those impacted (1).

On Feb. 28, 2025, the European Medicines Agency 

 to Vyjuvek (beremagene geperpavec), a topical gene therapy for the ultra-rare genetic skin-blistering condition, dystrophic epidermolysis, which is caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene (2).

Earlier in February, FDA granted breakthrough therapy designation to radiprodil, GRIN Therapeutics’ investigational, potent negative allosteric modulator selectively targeting the N-methyl-D-aspartate receptor subtype 2B (NR2B or GluN2B), for treatment of seizures associated with GRIN gene-related neurodevelopmental disorder with gain-of-function mutations (3).

Meanwhile, FDA’s Center for Drug Evaluation and Research’s Accelerating Rare Disease Cures program, established in 2022, released its Year Two Annual Report in October 2024, and the Learning and Education to Advance and Empower Rare Disease Drug Developers initiative continues to offer insights to those inquisitive about the design and conduct of rare disease clinical trials, according to an FDA update (4).

According to data from Elsevier’s Scopus, the amount of literature on rare diseases experienced a steady climb starting in 2018 and hit a peak in 2021 after the onset of the COVID-19 pandemic, having leveled off since then but still well above pre-2018 levels (5). The United States has been dominating other countries in terms of rare disease research output, although China is a clear second and outpaces the US in most other disease areas. And since 2020, there has been an explosive increase in inquiries as to how artificial intelligence can be used to shape this research.

Yet despite this increased focus, challenges remain when it comes to ensuring effective clinical trials for rare diseases, as outlined by Phesi in a February 2025 report (6). The five main obstacles listed in that report are: limited patient populations or difficulties with recruitment; geographic dispersion and uncertainty of investigator site selectivity; heterogeneity in disease presentation and progression; a lack of historical clinical trial data in many cases; and regulatory and/or ethical concerns.

Growing uncertainty at the federal level may also become a mounting challenge for the rare disease community. Just this week, the FDA and NIH-sponsored Rare Disease Day 2-day event was postponed (7) with little explanation in the wake of sweeping layoffs within the FDA and NIH. The event "seeks to bring together a broad audience including patients, patient advocates, caregivers, health care providers, researchers, trainees, students, industry representatives, and government staff," according to the event website, but the postponement has left many in the community concerned about the future support for rare diseases. 

This adds to growing concern about future federal support for rare disease research and drug development, as the Rare Pediatric Disease Designation and Priority Review Voucher Programs also ended in December 2024 and were not renewed (8), leaving little financial motive for pharmaceutical companies to fund rare disease drug development. 

Rare Disease Day Celebrated on February 28.

, Feb. 27, 2025.
2. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 24–27 February 2025. Press Release. Feb. 28, 2025.
3. GRIN Therapeutics. GRIN Therapeutics Receives FDA Breakthrough Therapy Designation for Radiprodil. Press Release. Feb. 25, 2025.
4. FDA – CDER. FDA Continues to Make Inroads in Rare Disease Drug Development. Press Release. Feb. 28, 2025.
5. Elsevier – Scopus – Rare Disease Research. Received via email correspondence on Feb. 27, 2025.
6. Phesi Rare Disease Showcase, 

5 Challenges of Delivering Effective Clinical Trials for Rare Diseases—And Strategies for Overcoming Them

; Phesi, February 2025.
7. FDA-NIH Rare Disease Day 2025. FDA.gov. Updated February 25, 2025. https://ncats.nih.gov/news-events/events/rdd
8. Rare Pediatric Disease Designation and Priority Review Voucher Programs. FDA.gov. Update September 27, 2024. https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs

Regulatory clearances and approvals continue as agencies work to disseminate literature, raise awareness, redouble research efforts, and improve the efficacy of clinical trials.

Rare Disease Day 2025: Pharmaceutical Treatments Advancing in the US, EU

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The European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) recommended marketing authorization approval for four medicines in February 2025. The recommended drugs treat a number of types of cancer, immunodeficiencies, and a rare genetic skin blistering disease.

Conditional marketing authorization was given to Lynozyfic (linvoseltamab) for the treatment of the bone marrow cancer, relapsed and refractory multiple myeloma. The generic drug, Trabectedin Accord (trabectedin), was granted approval to treat advanced soft tissue sarcoma and relapsed platinum-sensitive ovarian cancer.

Deqsiga (human normal immunoglobulin) was granted approval as areplacement therapy for primary or secondary immunodeficiencies and immunomodulation in patients with certain autoimmune diseases.

A treatment for the ultra-rare genetic skin blistering condition, dystrophic epidermolysis bullosa, was given a positive opinion. The orphan drug, Vyjuvek (beremagene geperpavec), went through the agency’s PRIority MEdicines scheme, which offers early and enhanced regulatory support. Dystrophic epidermolysis bullosa is caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene.

In addition to the new drug approval recommendations, CHMP extended therapeutic indications for 16 drugs, including Abrysvo, Calquence, Columvi (an orphan drug), Darzalex (an orphan drug), Enhertu, Imfinizi, Jaypirca, Prevymis (an orphan drug), Rinvoq, Stelara, Supemtek Tetra, and Tremfya.

The indications for Kaftrio (ivacaftor/tezacaftor/elexacaftor) and Kalydeco (ivacaftor) were extended to include patients aged two years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “This positive opinion brings disease-modifying treatment to all cystic fibrosis patients with modulator responsive mutations,” the agency said in a press release (1).

Ixchiq’s (chikungunya vaccine (live)) indication was extended to include immunization for adolescents 12 years and older to protect against the Chikungunya virus, a viral disease transmitted to humans by infected mosquitoes. 

The orphan drug, Fabhalta (iptacopan), had its indication extended to include treatment of adult patients with the ultra-rare kidney disease, complement 3 glomerulopathy.

At the meeting, CHMP also provided the results from its re-examination of the initial opinion for the indication extension for Keytruda (pembrolizumab) to treat unresectable non-epithelioid malignant pleural mesothelioma. The committee’s original decision was upheld. CHMP also held up its rejection of marketing authorization for Kizfizo (temozolomide) to treat neuroblastoma.

EMA also noted that the application for Pelgraz Paediatric (pegfilgrastim), for the treatment of neutropenia and prevention of febrile in children with cancer was withdrawn. The application of Rilonacept FGK Representative Service GmbH (rilonacept) to treat idiopathic pericarditis was also withdrawn.

The application to extend the indication of Dupixent to treat moderate-to-severe chronic spontaneous urticaria in adults and adolescents was withdrawn as well. In addition, the authorization holder for the COVID-19 vaccine, Bimervax, withdrew the application to include an adapted version to target the JN.1 strain of SARS CoV-2.

At the February meeting, CHMP also stated that the marketing authorization for Leqembi (lecanemab), which received a positive opinion in November 2024 for the treatment of mild cognitive impairment or dementia due to Alzheimer’s disease, does not need to be updated. “In January 2025, the European Commission (EC) asked the committee to consider information on the safety of Leqembi that became available after the adoption of the positive opinion and whether this would require an update of their recommendation. The CHMP has now concluded that its opinion recommending the marketing authorization does not need to be updated and has provided a response to the EC, which will now resume the decision-making process for this medicine,” EMA stated in the press release.

EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 February 2025. Press Release. Feb. 28, 2025.

Treatments for autoimmune diseases, multiple cancers, and skin blistering have been recommended for marketing approval.

EMA Recommends Four Medicines for Approval in February

Felicity Thomas is the former Associate Editorial Director of Pharmaceutical Technology, Pharmaceutical Technology Europe, BioPharm International.

Industry Outlook 2025: Interesting Relations and Detailed Discussions on Data

The political landscape in the United States has shifted, and while industry is learning about the various changes and new appointments, the full impact is hard to predict, not least because of the continuation effect from previous governments, points out Preeya Beczek, managing director and co-founder of Beczek.COM Ltd. However, there are three or four areas that she believes will be of potential interest with the new administration in place.

“I'm quite interested to think about China relations with the US and this BIOSECURE Act. I think that's going to perhaps impact the import tariffs, and then this could have a ripple effect on the global pharma industry, pharma economy,” Beczek explains. “The second thing, and this is not a new thing, is around anti-vaccine, anti-pharma sentiments, perhaps with the RFK [Robert F. Kennedy, Jr.] influence.”

Additionally, there is also discussion around the cost of drugs and provision of care and privatization that will continue throughout 2025, Beczek adds. “And then this final point I'm going to make is around digital health, health technology, AI [artificial intelligence], and mix that in the balance with data privacy,” she says.

Looking across the Atlantic, towards Europe, which has also undergone political shifts in 2024, industry is still working to get to grips with recent legislative changes, such as the new pharmaceutical legislation and the European Union clinical trials regulation, Beczek remarks. “So, coming back to that thing around information, data security, and what data [are] out in the public domain, right? There're still decisions that need to be made there at the very detailed level, and companies can expect to have detailed discussions around this,” she says.

Preeya Beczek, Managing Director and Co-Founder of Beczek.COM Ltd

Preeya Beczek is Managing Director and Co-Founder of Beczek.COM Ltd, a specialist in regulatory affairs and compliance. Preeya has worked in the regulatory affairs and compliance space for over 25 years, focusing on regulatory affairs activities at a strategic and operational level. Prior to her current role, Preeya worked in industry performing a lot of infrastructure and process development to support companies with new systems, initiatives with AI automation, regulatory, information management, system software modernization, and so on.

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Preeya Beczek from Beczek.COM gives her thoughts on the areas to watch with the new US administration and how Europe might be finalizing preparations for previous legislative changes in an interview with Pharmaceutical Technology®.

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