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

David McErlane, Group President of Biologics, Catalent, reacts to trends in cell and gene therapy development and manufacturing and comments on Catalent's ESG strategy to support people, the environment, and communities.

The Evolution of Cell & Gene Therapy: Development and Manufacturing Insights and the Role of CDMOs

Contract Development and Manufacturing Organizations (CDMOs) play a critical role in advancing the field of cell and gene therapies by providing specialized expertise, state-of-the-art facilities, and scalable manufacturing solutions. Their technical knowledge and ability to adapt to evolving regulatory standards enable faster development timelines and ensure compliance, supporting biotech companies in bringing life-changing therapies to patients more efficiently. By leveraging their established infrastructure, CDMOs also offer cost-effective solutions that allow biotech firms to focus resources on groundbreaking R&D while reducing the need for significant investment in in-house production facilities. This combination of expertise, flexibility, and cost efficiency ultimately accelerates the path to market for innovative treatments.

As cell and gene therapies continue to revolutionize the treatment of previously untreatable diseases, key trends are shaping the future of manufacturing in this field. Streamlined regulatory approval processes, such as the anticipated FDA Platform Technology Designation Program, aim to reduce the time and complexity of bringing therapies to market. Advancements in automation, artificial intelligence, and digitalization enhance the efficiency, scalability, and reliability of manufacturing processes, reducing costs and barriers to patient access. Additionally, innovations in supply chain management improve reliability and traceability, ensuring timely delivery of these highly individualized therapies.

Looking ahead, the balance between insourcing and outsourcing in cell and gene therapy manufacturing is likely to shift, with many biotech companies prioritizing funding for core innovation rather than maintaining in-house production capacity. CDMOs with phase-appropriate expertise and facilities are positioned to become essential partners, particularly as companies seek to offset high development costs and manage the complexities of production. Allogeneic cell therapies, with their streamlined requirements, are especially well-suited to outsourcing, further underscoring the growing importance of CDMOs in the industry.

The commitment to Environmental, Social, and Governance (ESG) principles is another area where CDMOs like Catalent can demonstrate leadership. By fostering a diverse and inclusive workplace, minimizing environmental impact through science-based targets, and giving back to local communities, CDMOs align their operations with the broader goals of sustainability and social responsibility. Sustainable manufacturing practices, ethical sourcing, and robust governance frameworks further reinforce their dedication to ethical and environmentally conscious operations. This holistic approach not only builds trust with stakeholders but also supports healthier communities and a more sustainable planet.

Want to learn more? Watch episode 3 of this series 

Videos

David McErlane, Group President of Biologics, Catalent, reacts to trends in cell and gene therapy development and manufacturing and comments on Catalent's ESG strategy to support people, the environment, and communities.


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

Pharmaceutical Technology chats with Roger Viney from ICE Pharma about bile acids in detail, including how they are useful in helping to improve drug solubility and absorption.

Improving Solubility and Bioavailability with Bile Acids

Poor solubility and bioavailability are becoming increasingly problematic for drug developers as more complex molecules are entering the development pipeline. “Bile acids are nature’s surfactants, their primary role is to promote the absorption of fat-soluble nutrients,” says Roger Viney, PhD, chief commercial officer for ICE Pharma—a bile acid expert. “So, depending on the bile acid, you can vary the hydrophilic lipophilic balance [and] improve bioavailability of poorly soluble drugs.”

“Furthermore, the bile acids can play a role as enhancer of permeability,” Viney continues. “[Bile acids are] able to modulate the cell membranes to allow certain drugs to cross the biological barriers more effectively. So, we have examples today, existing examples of oral and injectable applications formulations that use bile acids as excipients, and we are expecting to see this expand into things like transdermal and even nasal applications.”

Bile acids have been used in traditional forms of medicine for thousands of years; however, they did not enter modern and Western medicines until the 1960s, Viney notes. “The past 20 years have seen a very significant amount of research done into bile acids and their recognition that they're more than nature's surfactants, that they're, in fact, signaling molecules that have metabolic function,” he adds. “And so, this kind of research has opened up a whole new field where bile acids can potentially treat oncology, liver diseases, even neurodegenerative diseases.”

Roger Viney, PhD, is the chief commercial officer for ICE Pharma and is responsible for the global commercial functions. Roger has a 30-year career, starting in R&D and then moving across to sales, marketing, key account management, and business and product management for the pharma market.

In this episode of the Drug Solutions podcast, Felicity Thomas, Associate Editorial Director, chats with a host of experts about artificial intelligence (AI) and the potential impact AI and machine learning may have on the global bio/pharma industry in the near future.

 is chief scientific officer for Biorelate—an AI platform company that provides data solutions to pharma companies

 is CEO of Qinecsa Solutions, which is a UK headquartered but international pharmacovigilance technology and services solution provider.

 is vice president of Regulatory Quality and Compliance, Arriello—a life sciences consultancy.

 is CEO of Autolomous, which uses smart technology to help ATMP manufacturers.

is COO of Unbiased Science and vice-president of the Medical Information Leaders in Europe (MILE).

 presents the Drug Solutions podcast, where the editors will chat with industry experts from across the pharmaceutical and biopharmaceutical supply chain. Join us as experts share insights into your biggest questions—from the technologies, to strategies, to regulations related to the development and manufacture of drug products.

Podcasts

This episode is a compilation of comments taken from a series of interviews where experts are discussing how AI might impact the global bio/pharma industry in the future.

Drug Solutions Podcast: Realizing AI’s Potential 

Feliza Mirasol is the science editor for 

Orexo, a Sweden-based commercial-stage pharmaceutical company, announced on Dec. 17, 2024, that it has entered into a collaboration with Abera Bioscience (Abera), a Sweden-based platform and vaccine developer. Under the agreement, the companies will use AmorphOX, Orexo’s powder-based drug delivery technology, to develop mucosal vaccine candidates based on Abera's vaccine platform. This collaboration will be funded by Abera through grants it has previously received, mainly from the Coalition for Epidemic Preparedness Innovation (CEPI).

The AmorphOX technology is designed to improve properties of new or existing drugs. The technology aims to develop formulations that aid in significantly stabilizing active ingredients to enable alternative and more effective routes of administration. AmorphOX-based formulations are applicable for a range of active ingredients, from small molecules and peptides to larger biomolecules, such as vaccines, according to a company press release (1).

Orexo and Abera’s partnership will develop powder-based intranasal vaccine candidates with a focus on Abera's influenza vaccine candidate. Because an intranasal influenza vaccine has potential to easily and effectively reduce the spread of infections and prevent disease, they can play a significant role in controlling potential future pandemics.

“Influenza is a family of viruses that could mutate and cause the next pandemic, but seasonal influenza is already a major problem where vaccines are often not effective enough. Orexo has an impressive knowledge and technology for powder formulations of medicines that perfectly complements our expertise in vaccine development. Their innovative technology, AmorphOX, has shown to improve the properties of a wide range of different active ingredients. There is therefore great potential in this area, and we will be able to take many important development steps in the coming year,” said Maria Alriksson, CEO, Abera Bioscience, in the press release (1).

In a historical context, an influenza pandemic in 1918 caused at least 20 million deaths (2). Controlling another influenza pandemic will depend on innovations in vaccine production, according to researchers (3). A long-term goal for influenza vaccination should rely on a more universal vaccine, but there remains debate as to what exact traits a universal vaccine should have. A universal vaccine against all epidemic and pandemic viruses is not realistic, however, but having a vaccine that covers more drift variants may mean there could be less need for vaccine updates and a reduced need for yearly vaccinations, researchers note (4).

“Abera has an innovative and promising vaccine platform for the development of mucosal vaccines, as confirmed by their recent funding from CEPI. The collaboration is fully in line with our strategic development of the AmorphOX platform, and, through collaborations, we can showcase the potential and value of our technology. We look forward to working with Abera, where our experience and technologies are highly complementary,” said Robert Rönn, senior vice-president and head of R&D at Orexo, in the press release (1).

1. Orexo. Orexo Enters into Collaboration with Abera to Develop Nasal Powder Vaccines Based on the Amorphox Technology. Press Release. Dec. 17, 2024.
2. Crosby, A.W. 

America’s Forgotten Pandemic: The Influenza of 1918

; Cambridge University Press, 1989.
3. Kilbourne, E. D. Influenza Pandemics: Can We Prepare for the Unpredictable? 

 (3). DOI: 10.1089/vim.2004.17.350
4. Kim, H.; Webster, R. G.; Webby, R. J. Influenza Virus: Dealing with a Drifting and Shifting Pathogen. 

Articles

The companies will use Orexo’s powder-based drug delivery technology to develop mucosal vaccines in an inhaled formulation.

Orexo and Abera Partner to Develop Nasal Powder Vaccines

In this episode of Drug Digest, industry experts discuss their experiences in dealing with the challenges of developing and manufacturing next-generation biotherapeutics as well as triumphs that move the industry as a whole forward.

Challenges and Triumphs in Next-Generation Biotherapeutic Development

The development of next-generation biotherapeutics is in full swing, which means that developers and biomanufacturers are tackling new challenges in the drug development and manufacturing lifecycle. Two areas of focus are in the maintenance of an aseptic environment throughout the manufacturing process and the search for solutions to establishing alternative drug delivery methods that don’t require a parenteral route of administration. In this episode of 

, experts from N4 Pharma and Cellular Origins discuss their experiences in dealing with these challenges, as well as triumphs in creating innovations to move the industry as a whole forward.

Nigel Theobald is CEO of N4 Pharma, a specialist pharmaceutical company developing novel delivery systems for oncology, gene therapy and vaccines. Theobald has over 25 years’ experience in healthcare, in building businesses, strategy development and implementation, and in a strong network covering all aspects of pharmaceutical product development and commercialization. He was the head of healthcare brands at Boots Group plc in 2002 before leaving to set up a series of successful businesses, including Oxford Pharmascience Group plc, which he grew over five years into an AIM-quoted company with a market capitalization of £40 million (US$53 million) upon departure. Theobald formed N4 Pharma in 2014.



Dan Strange, PhD, Chief Technology Officer, Cellular Origins

Dan Strange has been working at the intersection of engineering and biology for almost 15 years, and he is an inventor on 20 patents. After completing a PhD in regenerative medicine at the University of Cambridge, he moved to TTP where he developed complex drug delivery devices. He was instrumental in the creation of multiple technology platforms successfully licensed to large pharma in deals worth over $100 million. For the past four years, Strange has focused on cell therapy manufacturing equipment. Strange is an active member of various automation working groups and was a reviewer on the Alliance for Regenerative Medicines A-Cell report on cell therapy manufacturing.

Drug Digest is a tech talk video series with the 

® editors, who interview industry experts to discuss the emerging opportunities, obstacles, and advances in the pharmaceutical and biopharmaceutical industry for the research, development, formulation, analysis, upstream and downstream processing, manufacturing, supply chain, and packaging of drug products.

Innovations in Biopharmaceutical Development

Drug Digest: Challenges and Triumphs in Next-Generation Biotherapeutic Development

Scinai Immunotherapeutics (Scinai), an Israel-based biopharmaceutical company focused on developing inflammation and immunology biological products, announced on Dec. 16, 2024 the establishment of a US-based subsidiary for its contract development and manufacturing organization (CDMO) services. The company provides CDMO services through its Scinai Bioservices business unit. This US subsidiary, incorporated in Delaware, will operate under the name Scinai Bioservices Inc.

The Scinai Bioservices CDMO business unit was launched in 2024 and operates from the company's site in Jerusalem, Israel. The Jerusalem facility comprises 1850 square meters (approximately 20,000 square feet) of clean rooms and laboratories, which operate under current good manufacturing practice (CGMP) conditions in compliance with European Medicines Agency (EMA) and FDA requirements, according to a company press release (1).

"We are thrilled and energized by the opening of our new subsidiary in the [United States]. Last week, we signed our first contract with US customer, Serpin Pharma, to support their clinical manufacturing,” said Amir Reichman, CEO of Scinai, in the press release (1). “I believe that our expanded presence in the US will significantly enhance the prospects of our CDMO unit."

From its launch, the CDMO unit has executed drug development projects for nine different biotech companies, which included upstream and downstream process development, scale-up, analytical methods development, and drug manufacturing for pre-clinical and clinical supplies. The company has also partnered with Ayana Pharma, an Israel-based pharmaceutical company specializing in liposomal therapeutics for treating cancer, under which Scinai will provide liposomal encapsulated drug development services.

The Scinai Bioservices unit operates on projects at the early stage, where the company noted in its press release capacity is strained because of the complex manufacturing processes associated with biologics. “Additionally, the BIOSECURE Act, introduced in January 2024, aims to prevent ‘foreign adversary biotech companies’ from receiving US federal funding,” the company stated in its press release (1).

Scinai's new US subsidiary aims to serve US-based early stage biotech companies with the goal of providing required CDMO services that are currently in short supply. The US subsidiary will also allow early stage biotech startups, which are supported by US government grant funding, to use their grant money in contract with Scinai, which would allow them to avoid concerns related to the BIOSECURE Act, Scinai stated in its press release.

To put the situation into context, Parviz Shamlou, senior vice-president of Science and Technology, Abzena, spoke to the PharmTech Group during BIO 2024 this past June about the impact the act will have on the bio/pharmaceutical industry and how the industry is preparing for it. “We appreciate that [the BIOSECURE Act] is an important act designed to protect the health of citizens of this country. And we know we must do everything we can to avoid any unintended, potentially harmful use of our services as highlighted in the act. [We] have put in place a robust contingency plan as well as a CMC [chemistry, manufacturing, and controls] strategy that are designed to mitigate any such risk to supply chain and the development of the assets that our clients bring to us,” Shamlou stated in the interview (2).

1. Scinai Immunotherapeutics. Scinai Immunotherapeutics Establishes US Subsidiary for CDMO Business Unit, Scinai Bioservices Inc. Press Release. Dec. 16, 2024.
2. Haigney S. 

Impact of Biosecure Act on the Pharma Industry (BIO 2024)

Scinai Bioservices Inc. has been established in Delaware as the company's new US-based subsidiary, which will serve biotech companies in early stage drug development.

Scinai Immunotherapeutics Establishes US CDMO Business Unit

AbbVie has signed a definitive agreement to acquire Nimble Therapeutics, a biotechnology company specializing in oral peptide therapeutics, which would add Nimble’s lead asset, an investigational oral peptide interleukin 23 receptor (IL23R) inhibitor that is in preclinical development for treating psoriasis. to AbbVie's immunology pipeline. In addition to the lead asset, AbbVie would also acquire a pipeline of other novel oral peptide candidates that show potential across several autoimmune diseases, AbbVie announced in a Dec. 13, 2024 company press release.

With the acquisition, AbbVie also gains Nimble's peptide synthesis, screening, and optimization platform. This platform uses proprietary technology to facilitate rapid discovery and optimization of peptide candidates for a range of targets.

Under the agreement, AbbVie will pay $200 million in cash at the close of transaction, which is subject to certain customary adjustments. AbbVie will also pay certain interim funding payments, and Nimble's shareholders will be eligible to receive potential payment upon the achievement of a development milestone, according to the press release (1).

"The addition of Nimble's pipeline to AbbVie's existing pipeline, combined with our deep clinical and translational expertise in immunology, represents an important growth opportunity," said Jonathon Sedgwick, PhD, senior vice-president and global head of discovery research, AbbVie, in the press release (1). "Together, AbbVie and Nimble have the potential to help address the significant unmet medical need for people living with autoimmune diseases."

Nimble's lead candidate, which targets IL23R, has potential in the treatment of psoriasis and inflammatory bowel disease (IBD). According to AbbVie, IL23R is a clinically validated therapeutic target in certain autoimmune diseases and is known to be a major contributing factor in psoriasis and IBD pathogenesis as well as progression by way of increased inflammation and amplified immune responses (1).

"Nimble Therapeutics is committed to transforming the discovery of oral peptide-based medicines. With AbbVie's … expertise in developing and commercializing medicines on a global scale, Nimble's novel oral therapies will be well-positioned to reach more people living with autoimmune diseases," said Jigar Patel, PhD, founder and chief executive officer, Nimble Therapeutics, in the release. "The talented, passionate, and dedicated team at Nimble has made great progress over the past few years, and we are pleased that AbbVie has recognized the tremendous potential of our proprietary platform and emerging immunology pipeline."

Nimble is the latest in a series of acquisitions that AbbVie has conducted this year. Also in December 2024, AbbVie completed its 

$1.4 billion acquisition of Aliada Therapeutics

$8.7 billion acquisition of Cerevel Therapeutics

, both of which strengthen AbbVie’s neuroscience pipeline (2,3). In June 2024, AbbVie 

acquired Celsius Therapeutics for $250 million in cash

, which included an antibody drug candidate in clinical development for IBD (4). In May 2024, AbbVie completed its acquisition of Landos Biopharma for approximately $137.5 million in cash, which included that company’s lead asset, NX-13, an oral nucleotide oligomerization domain-like receptor family member X1 agonist in clinical development for treating ulcerative colitis and Crohn's disease (5).

1. AbbVie. AbbVie to Acquire Nimble Therapeutics, Further Strengthening Immunology Pipeline. Press Release. Dec. 13, 2024.
2. AbbVie. AbbVie Completes Acquisition of Aliada Therapeutics. Press Release. Dec. 11, 2024.
3. AbbVie. AbbVie Completes Acquisition of Cerevel Therapeutics. Press Release. Aug. 1, 2024.
4. AbbVie. AbbVie Acquires Celsius Therapeutics. Press Release. June 27, 2024.
5. AbbVie. AbbVie Completes Acquisition of Landos Biopharma. Press Release. May 23, 2024.

AbbVie's latest acquisition target is Nimble Therapeutics, which will give AbbVie a lead asset, an oral peptide for treating psoriasis.

AbbVie to Boost Immunology Pipeline with Acquisition of Nimble Therapeutics

Text sign showing Industry News. Business photo text delivering news to the general public or a target public | Image Credit: © Artur - © Artur - stock.adobe.com

The European Medicines Agency (EMA) announced on Dec. 16, 2024 that a report (1) has been published that details the results of a pilot program for the creation and testing of electronic product information (ePIs) in regulatory procedures. According to the report, the European Union’s regulatory system is prepared for the introduction of ePIs, and implementation of ePIs can be phased into regulatory procedures. More development in functionality, however, still needs to be done, according to EMA. This development will begin in 2025. EMA and the Heads of Medicines Agency developed the ePI project, which was also supported by the EU4Health funding program.

The pilot’s report recommends that a phased implementation approach happen after all functionalities are in place. This approach will begin with voluntary adoption for centrally authorized products. Implementation will expand to nationally authorized products depending on the readiness and resources of each European Union (EU) Member State.

Drug product information is provided with each authorized drug in the EU and includes a summary of product characteristics, labeling, and packaging leaflet. These documents, which can be found in the drug’s packaging and as a PDF on regulators’ websites, explain how the drug should be prescribed and used.

“The introduction of product information in an electronic format (ePI) compatible with digital platforms opens new possibilities for patients and healthcare professionals to access and interact with vital, up-to-date information about their medicines,” EMA states in the press release (2).

Key indicators that were evaluated during the pilot included time needed to create an ePI, the percentage of ePIs that are successfully created and published, and usefulness of IT tools and guidance materials. These indicators were factored into the recommendations regarding future work that might be needed to implement ePI into current regulatory processes. The report states that enhanced guidance to support users and an update to business processes should be performed to ensure minimal disruption during implementation.

“In order to promote ePI adoption by companies, it is recommended to consider putting a process in place for a limited time period to allow first-time ePI submission and publication outside of a regulatory procedure. Importantly, regulators participating in the pilot found that it will be necessary to link ePI submission to the electronic application form and, for centrally authorized products, to the IRIS procedure management system, which will require a significant development effort,” the report states (1).

The pilot involved EMA, the national authorities of Denmark, the Netherlands, Spain, and Sweden, and industry. Twenty-three ePIs from real regulatory procedures were published as part of the pilot, of both centrally and nationally authorized medicines. The ePIs were created following the EU ePI Common Standard, “which provides a harmonized structure throughout all Member States ensuring the information works across different e-health platforms for citizens in the EU.” These ePIs can be found on the 

1. EMA; HMA. ePI Pilot Report. Experience Gained from Creation of ePI during Regulatory Procedures for EU Human Medicines. Dec. 16, 2024. 

https://www.ema.europa.eu/en/documents/report/electronic-product-information-epi-report-experience-gained-creation-epi-during-regulatory-procedures-eu-human-medicines_en.pdf


2. EMA. Successful Pilot Paves the Way for Implementation of ePI. Press Release. Dec. 16, 2024.

A pilot program to explore creation and testing of ePIs in regulatory procedures shows positive results.

Electronic Product Information Moves Forward in Europe

Novo Nordisk announced on Dec. 16, 2024 that it will invest DKK 8.5 billion (US$1.2 billion) to build a new production facility in Odense, Denmark, for the manufacture of multiple product types for treating rare diseases, such as hemophilia, both at present and in the future. This is the first investment Novo Nordisk has made for breaking new ground in Denmark in this century, the company stated in a press release.

The new site will include a state-of-the-art finished production facility and warehouse spanning more than 40,000 m

. The production facility will be designed to be modular and flexible to enable the manufacture of multiple rare disease-treating products.

"The facility will utilize advanced technology and innovative equipment to ensure the highest quality to patients and meet the growing global demand for our life-changing medicines,” said Henrik Wulff, executive vice-president, Product Supply, Quality & IT of Novo Nordisk, in the press release (1). “We are proud to build on our heritage in Denmark and look forward to embarking on this journey in Odense, a well-connected city with a dynamic community and talented workforce."

The new facility will be built with an eye toward preserving the nature in the area. More than 4000 new trees will be planted at the site, and buildings will be equipped with solar panels for enhancing on-site electricity generation. In addition, the landscape will be designed by reusing excess soil, wooden materials, and other sustainable resources to create lakes, forests, and community spaces that will be open to the public, the company stated in its release.

Construction work has begun, and completion is expected in 2027. More than 400 permanent jobs are expected to be created upon completion of the facilities, and up to 1000 external employees will be working on-site during construction.

Meanwhile, as of Dec. 14, 2024, all regulatory closing conditions related to the review of Novo Holdings’ pending acquisition of Catalent have been fulfilled, including expiry of the timing agreement in the United States, which means the parties are now free to close the transaction (2). With the close of the transaction, Novo Nordisk will fulfill its subsequent 

acquisition of three Catalent manufacturing sites

 from Novo Holdings, which Novo Nordisk originally announced in February 2024 (3). Novo Nordisk will also be acquiring Novavax’s manufacturing site in the Czech Republic, which was announced earlier in December 2024 (4).

1. Novo Nordisk. Nordisk Invests DKK 8.5 Billion in New Production Facility in Odense, Denmark. Press Release. Dec. 16, 2024.
2. Novo Nordisk. Novo Nordisk A/S: The Acquisition of Catalent by Novo Holdings, and the Related Acquisition by Novo Nordisk of Three Manufacturing Sites from Novo Holdings, is Cleared To Close. Press Release. Dec. 14, 2024.
3. Novo Nordisk. Novo Nordisk to Acquire Three Fill-Finish Sites from Novo Holdings A/S in Connection with the Catalent, Inc. Transaction. Press Release. Feb. 5, 2024.
4. Novavax. Novavax Announces Sale of Czech Republic Manufacturing Site to Novo Nordisk for $200 Million. Press Release. Dec. 4, 2024.

Novo Nordisk will use the DKK 8.5 billion (US$1.2 billion) to build a new modular and flexible production facility in Odense, Denmark, that will produce multiple products for rare diseases. 

Novo Nordisk to Establish New Production Facility for Rare Disease Therapies in Denmark with DKK 8.5 Billion Investment

The European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) recommended 17 drugs for market authorization at its December meeting, EMA announced on Dec. 13, 2024. The approvals include five orphan drug recommendations and six biosimilar recommendations.

Among the approval recommendations is a monoclonal antibody, Kavigale (sipavibart), for COVID-19 prevention in immunocompromised people aged 12 and older. “Following the recommendation for approval of Kavigale, EMA’s Emergency Task Force (ETF) has issued an updated statement on the loss of activity of anti-spike protein monoclonal antibodies due to emerging SARS-CoV-2 variants and recommends healthcare professionals to check the current epidemiological situation in their region,” the agency stated in a press release (1). Kostaive (zapomeran), a self-amplifying messenger RNA vaccine, was also recommended for the prevention of COVID-19.

Orphan drugs recommended by CHMP include the following:

Andembry(garadacimab), which prevents recurrent attacks of hereditary angioedema

Beyonttra (acoramidis), which treats a wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy

Rytelo(imetelstat), which treats adults with transfusion-dependent anemia due to very low, low, or intermediate risk myelodysplastic syndromes

Seladelpar Gilead(seladelpar lysine dihydrate), which treats primary biliary cholangitis

Emcitate(tiratricol), which treats peripheral thyrotoxicosis in patients with Allan-Herndon-Dudley syndrome.

The following six biosimilars were recommended for authorization:

Avtozma (tocilizumab) was given positive recommendation for treating rheumatoid arthritis, systemic juvenile idiopathic arthritis, polyarticular juvenile idiopathic arthritis, giant cell arteritis, chimeric antigen T cell (CAR-T cell)-induced severe or life-threatening cytokine release syndrome and COVID-19.

Eydenzelt (aflibercept) treats eye diseases such as neovascular age-related macular degeneration, macular oedema secondary to retinal vein occlusion, diabetic macular oedema, and myopic choroidal neovascularization.

Osenvelt (denosumab) prevents bone complications in adults with advanced bone cancer and treats adults and skeletally mature adolescents with giant cell bone tumors.

Stoboclo (denosumab) treats osteoporosis in menopausal women, bone loss linked to hormone ablation in men at increased risk of fractures, and bone loss associated with long-term treatment with systemic glucocorticoid.

Yesintek (ustekinumab) treats adults and children with plaque psoriasis and adults with psoriatic arthritis or Crohn’s disease.

Zefylti (filgrastim) treats neutropenia and the mobilization of peripheral blood progenitor cells.

Other positive opinions given by CHMP include:

Nemluvio (nemolizumab), which treats atopic dermatitis and prurigo nodularis

Welireg(belzutifan), to treat tumors associated with von Hippel-Lindau disease and advanced clear cell renal cell carcinoma

Paxneury (guanfacine), which treats children with attention-deficit hyperactivity disorder

Tuzulby (methylphenidate hydrochloride) to treat children with attention-deficit hyperactivity disorder.

The December recommendations bring the total of drugs recommended for approval by CHMP for 2024 to 114.

CHMP also extended recommendations for several drugs, including Ofev for the treatment of progressive fibrosing interstitial lung diseases (ILDs) in children and adolescents, Blincyto, Bridion, Flucelvax Tetra, Jemperli, Omvoh, Rekambys, and Vocabria.

Also during the December meeting, CHMP chose to withdraw Alofisel (darvadstrocel) from the market after data showed its effectiveness was not what had been anticipated. Alofisel had been approved to treat complex anal fistulas in adults with Crohn’s disease.

Meeting highlights from the Committee for Medicinal Products for Human Use(CHMP) 9-12 December 2024

EMA recommended 17 drugs for marketing authorization in December, including treatments for rare heart conditions, anemia, and liver disease.

Featured Content

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

Behind the Headlines Episode 28

Ask the Expert

Drug Digest December 2025

A “Plan, Prevent, Prove” Approach