^{Maryam Tabatabai, PharmD, is the associate vice president of clinical information at Prime Therapeutics.}  

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For the 15th year, the U.S. Food and Drug Administration (FDA)’s Center for Drug Evaluation and Research (CDER) has published an annual report of drug approvals — “Advancing Health Through Innovation: New Drug Approvals.” In 2025, the agency shared, the FDA approved 46 novel drugs — or drugs that have never been approved or marketed in the United States. This is down from 50 approvals in 2024 and 55 in 2023, yet close to the FDA’s 10-year novel drug approval average of about 47 a year.  

Biologic agents  

Drugs approved through CDER were part of the story. On the biologics front for agents approved by the FDA’s Center for Biologics and Evaluation and Research (CBER), the agency approved 12 biologics (11 in 2024), which is higher than the 10-year average of around nine per year.   

Combined, through CBER and CDER, the FDA approved 58 innovative agents in 2025, putting the count above the 10-year average of 56.   

What were the trends in 2025? 

Novel drugs for cancer led the pack. A few examples of notable oncology approvals include: 

• Datopotamab deruxtecan (Datroway), a new antibody-drug conjugate (ADC) for HR-positive, HER2-negative breast cancer (later approved for select patients with EGFR-mutated non-small cell lung cancer). 

• Ziftomenib (Komzifti), a menin inhibitor for certain individuals with acute myeloid leukemia (AML). 

• Taletrectinib (Ibtrozi), a next-generation tyrosine kinase inhibitor for a select type of non-small cell lung cancer.  

Oncology approvals in 2025 reinforce the continued innovation in this therapeutic area and availability of targeted therapies and precision medicine for patients. Following is a look at a handful of other notable high-profile non-oncology approvals in 2025. 

Novel approvals in 2025 marked many firsts. Here are a few:   

• Vertex’s oral suzetrigine (Journavx) is a first-in-class nonopioid medication for acute moderate to severe pain. It is the first new drug approved for acute pain in more than 20 years. 

• GlaxoSmith Kline’s depemokimab-ulaa (Exdensur) is the first twice-yearly shot for severe eosinophilic asthma (for those 12 years of age or older).  

• GlaxoSmith Kline’s gepotidacin (Blujepa) for the treatment of uncomplicated urinary tract infections in female patients aged 12 or older is the first new class of oral antibiotics for this condition in nearly 30 years.  

• Insmed’s brensocatib (Brinspuri) for the treatment of non-cystic fibrosis bronchiectasis is the first approved therapy for bronchiectasis.  

• Stealth Biotherapeutics’s elamipretide (Forzinity) is the first treatment for Barth Syndrome. It helps improve muscle strength for this rare, life-threatening mitochondrial disease.  

Several agents also received new formulations or indications, including:  

• Novo Nordisk’s first oral glucagon-like peptide-1 (GLP-1) receptor agonist medication semaglutide (Wegovy) for obesity and cardiovascular health. 

• Novo Nordisk’s first GLP-1, injectable semaglutide (Wegovy), for metabolic-associated steatohepatitis (MASH), a condition closely related to obesity. 

• Gilead’s lenacapavir (Yeztugo), a new twice-yearly injection for human immunodeficiency viruses (HIV) pre-exposure prophylaxis (PrEP).  

Finally, cell and gene therapy (CGT) saw momentum, with five new CGTs.  

• Fondazione Teleton’s etuvetidigene autotemcel (Waskyra) for Wiskott-Aldrich syndrome, an immunodeficiency condition involving low platelets, eczema and increased susceptibility to infections.  

• Novartis’s onasemnogene abeparvovec-brve (Itvisma) for spinal muscular atrophy with SMN1 gene mutation, which causes a deficiency of survival motor neuron protein, leading to muscle weakness, atrophy and respiratory issues.   

• Precigen’s zopapogene imadenovec-drba (Papzimeos) for recurrent respiratory papillomatosis, which is caused by human papillomavirus (HPV) types 6 and 11, leading to growths in the larynx or airway and causing respiratory issues.  

• Abeona’s prademagene zamikeracel (Zevaskyn) for recessive dystrophic epidermolysis bullosa, a rare skin condition caused by mutations in the COL7A1 gene leading to a lack of collagen VII, leading to fragile skin, painful blisters, scarring and other issues.  

• Neurotech’s revakinagene taroretcel-lwey (Encelto) for macular telangiectasia type 2 (MacTel-2), a rare retinal disease that causes central vision loss as a result of damage to the macula.  

Implications for rare disease treatment 

Half of the novel drug approvals for last year were for orphan drugs, or medications that treat rare diseases affecting fewer than 200,000 individuals in the United States. The number of novel orphan drug approvals is comparable to years past. Drug approvals for rare diseases make a significant difference, as individuals affected by these conditions frequently have limited or no existing therapies available to them.  

Accelerated program helps propel increase in drug decisions 

Almost three-quarters of novel approvals underwent at least one or more of the agency’s expedited programs to accelerate approval for serious conditions, with about 25% designated as “Accelerated Approval.” The FDA’s Accelerated Approval pathway allows for earlier approval of drugs to treat serious conditions and to fill an unmet medical need based on a surrogate endpoint thought to predict clinical benefit. This designation can be challenging for payers to manage, since approval is based on a surrogate endpoint. Confirmatory trials are needed to confirm clinical benefit for these drugs to remain on the market.    

While numbers do not tell the entire story, they represent innovation for patient care and have the potential to advance health for the American public. 

For the latest clinical insights related to the evolving drug approvals, read Prime’s latest Quarterly Drug Approvals publication.  

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