CDER approved 46 novel drugs in 2025, half for rare diseases

The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved 46 novel drugs in 2025, representing a slight decrease from the novel approvals in recent years. More than half of these approvals were for rare disease indications, with 25 drugs with orphan designation approved last year.

In 2024, CDER approved 50 novel drugs, as well as a record number of biosimilars and a significant number of therapies with orphan drug designation. FDA approved 55 novel drugs in 2023, 37 in 2022, and 50 in 2021. (RELATED: CDER approved 50 novel drugs in 2024, record number of biosimilars, Regulatory Focus 9 January 2025)

Most of the novel drugs received approval in the latter half of the year, with seven approvals taking place in December alone. The agency experienced significant upheaval throughout 2025, as it saw staff attrition prompted by a wave of early retirements, buyout offers, and a reduction-in-force effort, as well as a high degree of turnover in leadership positions under the Trump administration. CDER itself saw five directors between from the final month of the Biden administration to the end of 2025: Patrizia Cavazzoni, Jacqueline Corrigan-Curay, George Tidmarsh, Richard Pazdur, and Tracy Beth Høeg, who currently leads the center. (RELATED: HHS to cut 10,000 more jobs across HHS, including 3,500 from FDA, Regulatory Focus 27 March 2025).

Most of the drugs that received approval were in the oncology field, with 14 specifically focused on cancer treatment. Notable approvals include Merck’s Keytruda Qlex (pembrolizumab and breahyaluronidase alfa-pmph), for treating both adult and pediatric patients with solid tumors. Another significant approval is Regeneron’s Lynozyfic (linovoseltamab-gcpt), which is designed for treating relapsed or refractory multiple myeloma after patients have completed at least four prior lines of therapy.

Other notable oncology approvals include Bayer’s Hyrnuo (sevabertinib), which is intended for patients with locally advanced or metastatic non-squamous non-small cell lung cancer that possesses activating mutations in the HER2 tyrosine kinase domain. This treatment is specifically for those who have already undergone systemic therapy. Additionally, Kura Oncology has received approval for Komzifti (ziftomenib), designed to treat adults with relapsed or refractory acute myeloid leukemia who have a nucleophosmin 1 mutation and lack satisfactory alternative treatment options.

In 2025, a portion of novel FDA drug approvals were for rare diseases. These include FDA’s approval of CSL Behring’s Andembry (garadacimab-gxii) to prevent attacks of hereditary angioedema; and approval of SpringWorks Therapeutics Gomekli (mirdametinib) for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.

In other therapeutic areas, other notable approvals were for GSK’s Blujepa (gepotidacin) for treating uncomplicated urinary tract infections (UTIs) in females 12 years of age and older, which is the first new class of oral antibiotics for this condition in nearly 30 years.

Mary Thanh Hai, the director of the FDA's Office of New Drugs (OND), spoke at the USSF-Stanford CERSI meeting on Sunday. She noted that 50% of the novel drugs approved last year went through one of the FDA's expedited approval pathways, such as fast track, breakthrough designation, or priority review. Additionally, half of the novel drugs were approved in the US before being approved in other countries.

She further noted that CDER met its review and approval goals under the Prescription Drug User Fee Act (PDUFA) 96% of the time. Additionally, 85% of these new drugs were approved during the first review cycle, which she mentioned is higher than in recent years.

Novel drug approvals

CDER approved 46 novel drugs in 2025, half for rare diseases

Related topics

Recommended content

Welcome to the new RAPS Digital Experience