2023 marked a significant year in the pharmaceutical industry as the Center for Drug Evaluation and Research (CDER) of the US Food and Drug Administration (FDA) approved an impressive 55 novel drugs. These approvals, including new molecular entities (NMEs) and new therapeutic biologics, underscore the FDA’s commitment to advancing patient care with innovative therapies. Over the past decade, CDER has delivered a steady flow of healthcare innovations, averaging about 46 novel drug approvals annually. This consistent performance highlights the critical role of the FDA in introducing new and effective treatments for a diverse range of medical conditions.
Pioneering First-in-Class Drugs
A significant portion of the 2023 approvals, 36%, were first-in-class drugs with unique mechanisms of action, offering new therapeutic options. Some of the notable first-in-class drugs include:
- Daybue for Rett syndrome.
- Jesduvroq for anemia in chronic kidney disease patients on dialysis.
- Paxlovid for COVID-19.
- Talvey for multiple myeloma, approved via Accelerated Approval.
- Veozah for menopausal hot flashes.
- Xdemvy for Demodex blepharitis.
Demonstrating the FDA’s dedication to underserved patient populations, 51% of the approved novel drugs received orphan drug designation for rare diseases. Some of the noteworthy approvals for rare conditions include:
- Fabhalta for paroxysmal nocturnal hemoglobinuria.
- Filspari for IgA nephropathy, approved through Accelerated Approval.
- Ojjaara for myelofibrosis.
- Sohonos for fibrodysplasia ossificans progressiva.
Other significant drugs approved by CDER in 2023 addressed a variety of conditions:
- Beyfortus to prevent respiratory syncytial virus (RSV)
- Leqembi for Alzheimer’s disease, initially approved through Accelerated Approval.
- Orserdu for breast cancer post-endocrine therapy.
- Zavzpret for acute migraine treatment.
- Zurzuvae as the first oral medication for postpartum depression.
The FDA made considerable use of expedited development and review pathways to ensure timely access to new therapies for patients with serious conditions lacking satisfactory alternatives. These pathways aim to balance efficiency and flexibility with the FDA’s high standards for safety and effectiveness.
Fast Track Designation: In 2023, 45% of novel drugs received Fast Track status, facilitating development and review through increased communication and rolling review processes. Examples include Agamree, Beyfortus, and Columvi.
Breakthrough Therapy: With the Breakthrough Therapy designation awarded to 16% of the novel drugs, the FDA provided intensive guidance and senior manager involvement to speed up development. Drugs with this status included Elrexfio, Fabhalta, and Izervay.
Priority Review: A majority, 56%, of novel drugs were designated for Priority Review, expediting the review process for drugs that could significantly enhance treatment safety or effectiveness. Examples include Augtyro, Columvi, and Daybue.
Accelerated Approval: The Accelerated Approval pathway was used for 16% of approvals, allowing faster access based on alternative endpoints that predict clinical benefit, accompanied by required post-approval studies. Drugs approved via this pathway include Columvi, Elrexfio, and Epkinly.
Remarkably, 65% of novel drug approvals in 2023 used one or more expedited programs, showcasing the FDA’s commitment to delivering timely treatments without compromising safety and efficacy. This includes drugs like Agamree, Augtyro, and Beyfortus. Indeed, the FDA’s robust pipeline of novel drug approvals in 2023 reflects its ongoing mission to advance public health through the support of innovative treatments, with a particular focus on rare and difficult-to-treat conditions, all facilitated by expedited regulatory pathways.
DDReg has supported its customers with end-to-end regulatory services to facilitate and expedite approvals for a variety of therapies in the US market. Indeed, the US FDA is among the most stringent regulatory agency’s showcasing strict regulations. Read more from DDReg’s expertise: Easing Requirements for Minimal Risk Trials.