FDA takes steps to support novel therapy innovation

FDA takes steps to support novel therapy innovation

The FDA is leading the way in facilitating the development of novel therapeutics, including for metabolic disorders and rare diseases. The agency has been investing in resources to get products approved faster and in supporting innovation. 

Notably, in December 2023, the FDA announced the establishment of a new advisory committee to evaluate genetic metabolic disease treatments. The committee will, when asked, provide the FDA with advice and recommendations on relevant scientific and policy issues related to products for metabolic diseases i 

The creation of a committee is just the latest step by FDA to ensure expert support and guidance in the development and review of products for genetic metabolic diseases. In 2020, the agency established the Division of Rare Diseases and Medical Genetics to oversee the development of such products. The division is part of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine (ORPURM) within the Center for Drug Evaluation and Research (CDER). 

“This new advisory committee will provide a forum for discussion of complicated issues with specialized and diverse technical and scientific experts in the field of metabolic genetics,” said Janet Maynard, M.D., M.H.S., director of the ORPURM, in the FDA announcement. 

The agency is seeking nominations for the committee until 11 February 2024, and is eager to get the perspective of technical and scientific subject matter experts from diverse backgrounds to serve on the committee ii 

Guidance to support innovation 

Also in December, the FDA issued final guidance entitled “Rare Diseases: Considerations for the Development of Drugs and Biological Productsiii. The guidance seeks to help drug and biologics developers to conduct efficient and successful drug development programs for rare diseases. It offers greater flexibility with nonclinical development programs for drugs to treat severely debilitating or life-threatening rare diseases, such as with some toxicology studies and modified proof-of-concept studies.  

The focus on innovation can be seen in the huge number of novel agents granted approval in 2023, during which the agency approved 72 novel agents, including first CRISPR–Cas9-based gene editing product, vaccines for respiratory syncytial virus (RSV) and several breakthrough gene therapies iv. 

In December, FDA approved Vertex and CRISPR Therapeutic’s Casgevy (exa-cel), a CRISPR therapy for the treatment of sickle cell disease v. The European Medicines Agency (EMA) also recommended approval of Casgevy, with the agency’s Committee for Advanced Therapies expert committee finding the product’s benefits outweigh possible risks vi 

Over the past two years, there have been increased efforts by the FDA to accelerate rare disease innovation. In 2022, CDER  launched the Accelerating Rare disease Cures (ARC) Program to help speed up development of effective and safe treatments for rare diseases with unmet medical needs vii. The program brings together multiple stakeholders to try to address the challenges in bringing new drugs to market.  

The FDA also established a Rare Disease Endpoint Advancement (RDEA) Pilot Program in October 2022, which enables sponsors to work together with the agency during the efficacy endpoint development process, encourages learnings on novel endpoints to be shared, and increases FDA capacity to use novel endpoints to evaluate rare disease therapies viii. 

Working with industry 

In addition, the Center for Biologics Evaluation and Research (CBER) is committed to working with innovative cell and gene therapy companies to facilitate development of their therapies. CBER’s director, Dr. Peter Marks, has noted that the agency recognizes the difficulty with patient recruitment and will work with developers to find alternative ways to expedite the process, as long as they present good clinical data ix. CBER also launched a pilot program, Trials Advancing Rare disease Therapeutics (START), in September 2023 that lets sponsors get advice and ask to communicate with reviewers on development issuesx. 

The EMA also has initiatives that seek to support innovation, including incentives and support for micro, small and medium-sized enterprises (SMEs) xi. The PRIME scheme gives developers of priority medicines enhanced interaction and dialogue xii. There are also opportunities to speak with national committees in Europe, who, experience has shown, often provide helpful advice on starting clinical trials in specific countries. 

Through the EU Pharmaceutical Legislation, the European Commission is taking other steps to support innovators, including streamlining the rules for clinical trials of medicines consisting of, or containing genetically modified organisms xiii 

Across both the US and the EU, there are increased efforts to support innovators with potentially life-saving and life-changing therapies that industry can and should take advantage of. How will these initiatives impact your interactions with the health authorities and potentially even how you innovate? We would be interested in hearing your perspective.  


About the author: 

Jürgen Hönig, Ph.D., is Senior Director, Regulatory Business Intelligence, at PharmaLex. He has played a significant role in the design of the regulatory intelligence strategy at PharmaLex and provides analysis on trends in the pharmaceutical and medical device industries. Jürgen is a pharmacist by training and received his Ph.D. from Johann-Wolfgang-Goethe University, Germany. 


The contents of this blog are solely the opinion of the author and do not represent the opinions of PharmaLex GmbH or its parent Cencora. PharmaLex and Cencora strongly encourage readers to review available information related to the topics mentioned herein and to rely on their own experience and expertise in making decisions related thereto. 



i FDA Creates New Advisory Committee for Evaluation of Genetic Metabolic Disease Treatments, FDA, Dec 2023. https://www.fda.gov/news-events/press-announcements/fda-creates-new-advisory-committee-evaluation-genetic-metabolic-disease-treatments 

ii Genetic Metabolic Diseases Advisory Committee (GeMDAC), FDA. https://www.fda.gov/advisory-committees/human-drug-advisory-committees/genetic-metabolic-diseases-advisory-committee-gemdac 

iii Rare Diseases: Considerations for the Development of Drugs and Biological Products, FDA, Dec 2023. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/rare-diseases-considerations-development-drugs-and-biological-products

iv 2023 FDA approvals, Nature Reviews Drug Discovery, Jan 2024. https://www.nature.com/articles/d41573-024-00001-x 

v Vertex and CRISPR Therapeutics Announce US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease, Vertex Pharmaceuticals, Dec 2023. https://investors.vrtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-announce-us-fda-approval 

vi First gene editing therapy to treat beta thalassemia and severe sickle cell disease, EMA, Dec 2023. https://www.ema.europa.eu/en/news/first-gene-editing-therapy-treat-beta-thalassemia-and-severe-sickle-cell-disease 

vii CDER Continues to Advance Rare Disease Drug Development with New Efforts, Including the Accelerating Rare Disease Cures (ARC) Program, FDA. https://www.fda.gov/drugs/our-perspective/cder-continues-advance-rare-disease-drug-development-new-efforts-including-accelerating-rare-disease

viii Rare Disease Endpoint Advancement Pilot Program, FDA. https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program 

ix Planning for CBER and EMA success with cell and gene therapy, Dr. Cori Gorman, PharmaLex. https://www.biopharma-excellence.com/planning-for-cber-and-ema-success-with-cell-and-gene-therapy/ 

x CBER chief says pilot program will justify increasing reviewer headcount, RAPS Regulatory News, Dec 2023. https://www.raps.org/news-and-articles/news-articles/2023/12/cber-chief-says-pilot-program-will-justify-increas 

xi Support to SMEs, EMA. https://www.ema.europa.eu/en/about-us/support-smes 

xii PRIME: priority medicines, EMA. https://www.ema.europa.eu/en/human-regulatory-overview/research-and-development/prime-priority-medicines 

xiii Revision of the EU pharmaceutical legislation, European Parliament, Sept 2023. https://www.europarl.europa.eu/RegData/etudes/BRIE/2023/747464/EPRS_BRI(2023)747464_EN.pdf 


The contents of this article/presentation are solely the opinion of the author and do not represent the opinions of PharmaLex GmbH or its parent AmerisourceBergen Corporation. PharmaLex and AmerisourceBergen strongly encourage readers/listeners to review available information related to the topics mentioned herein and to rely on their own experience and expertise in making decisions related thereto.

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