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Creating a Yearly Snapshot for Approvals
CDER began publishing the annual New Drug Therapy Approvals report in 2011 to highlight that year’s approvals with active ingredient(s) that had not been previously FDA-approved. The report served as a single source document featuring not only these approvals, but also the various programs and regulatory efficiencies, such as the FDA’s expedited approval programs, that helped bring them to market. The report was expanded in 2017 to also include previously approved drugs approved to treat expanded patient populations, different conditions, new dosage forms and drug formulations.
Much of the information in the New Drug Therapy Approvals report is also accessible through publicly available FDA websites. The report takes drug approval information from these sites and adds robust historical analysis that isn’t available elsewhere. For example, the percentage of rare disease approvals is measured each year and can be compared to other years. This information helps reveal trends in approvals and examines different FDA expediated approval programs.
By: Patrizia Cavazzoni, M.D., Director, Center for Drug Research and Evaluation
Each year, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) evaluates and, as appropriate, approves many therapies for patients and consumers. CDER publishes an annual New Drug Therapy Approvals report to showcase these novel approvals and the regulatory tools used to streamline the review and approval process.
As in previous years’ reports, the 2024 New Drug Therapy Approvals report features drug approvals that CDER considers likely to have a significant impact on public health. The 2024 report includes approvals for therapies that collectively treat a wide range of conditions, including a first-in-class drug for schizophrenia, three novel antibiotics and new drugs for many types of cancer.
New Rare Disease Therapies in 2024
In 2024, CDER approved 50 new drugs that had active ingredients not previously approved by the FDA. These approvals included 26 new therapies that had previously received an orphan drug designation to prevent, diagnose or treat a rare disease or condition. This included several therapies for rare conditions with no previously approved treatment, such as Neimann-Pick disease type C and WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis).
Other new rare disease therapies were approved by CDER in 2024 to expand treatment options for patients, such as:
- Duchenne muscular dystrophy, a severe, inherited disease that causes progressive muscle weakness and muscle wasting.
- Familial chylomicronemia syndrome, a disease that prevents the body from digesting fats.
- Primary biliary cholangitis, a chronic autoimmune disease that damages the bile ducts in the liver.
- New treatments for rare cancers such as HER2-positive (IHC3+) tumors, which have higher levels of the human epidermal growth factor receptor 2 (HER2) protein that enables faster growth and spread, and gastroesophageal junction adenocarcinoma, a rare type of cancer that starts in the gastroesophageal junction.
A Record Year for Biosimilar Approvals
CDER surpassed a major approval milestone for biosimilar biological products (biosimilars) in 2024 when it approved the 60th biosimilar since the first biosimilar approval in 2015. This milestone is due to CDER approving in 2024 a record total of 18 biosimilars for 8 reference products, more than any previous year. Also in 2024, CDER approved 9 interchangeable biosimilars (for new and previously approved biosimilars). An interchangeable biosimilar may be substituted for the reference product at the pharmacy without the intervention of the prescriber, subject to state pharmacy laws (similar to how generic drugs are substituted for brand name drugs).
CDER’s Effectiveness Bringing New Therapies to Market
CDER uses innovative regulatory approaches to help expedite drug review. While preserving the FDA’s high standards for safety and effectiveness, CDER relies on innovation and efficiency to approve new therapies quickly, as evidenced by the following:
- User Fee Goal Performance: In 2024, CDER met or exceeded its Prescription Drug User Fee Act goal date for 47 of the 50 novel drug approvals (94%).
- First Cycle Approvals: 37 of the 50 novel drugs approved in 2024 (74%) were approved on the “first cycle” of review.
- Approval in the U.S. Before Other Countries: In 2024, 34 of the 50 novel drugs approved by CDER (68%) were approved in the U.S. before being approved in other countries.
- Expedited Programs: In 2024, CDER approved 33 of the 50 new therapies (66%) using one or more expedited approval programs: fast track designation, breakthrough therapy designation, priority review designation, and Accelerated Approval. The FDA uses these four programs to facilitate and expedite development and review of drugs for serious or life-threatening conditions.
The 2024 New Drug Therapy Approvals report emphasizes our review teams’ hard work and dedication to advancing patient care through timely and thorough review and approval of safe and effective therapies. We know our decisions impact the health and wellness of patients and consumers across the U.S., and we are committed to approving drug and biologic applications that meet our high regulatory standards.
I am honored to have led the CDER teams behind the 2024 new drug approvals. I have confidence they will have a positive impact on public health and improve the lives of the patients and consumers that will use them. We look forward to 2025 and the advances it will bring for drug development and approvals.
