Isn’t it exciting when a new medication comes out that can treat an unmet medical need, address an existing need in a new way, or enhance a patient’s quality of life?  Not including added indications, new dosage forms, and formulation changes, the FDA approved 50 new molecular entities in 2024.  The total number of novel approvals was slightly down from 2023’s 55 approvals. The following chart depicts the number of new drug approvals in recent years.

Source: FDA Center for Drug Evaluation and Research Advancing Health Through Innovation: New Drug Therapy Approvals 2024; Page 6.

Trends in Approvals

• 48% (24 of the 50) of the approvals were first-in-class therapies.
• 52% (26 of the 50) of the approvals were for rare or “orphan” diseases.
• The FDA achieved 94% of their PDUFA goals (47 of the 50 approvals).
• First cycle approvals accounted for 74% (37 of the 50) of the novel products.
• Oncology led the charge with 16 novel approvals.
• 68% (34 of the 50) of approvals are NDAs, and 32% are BLAs.

As we have seen in past years, the majority of new drug approvals will be in the hospital or outpatient specialty pharmacy space.  By PHSL’s estimate, only five of these agents (Orlynvah, Cobenfy, Ohtuvayre, Sofdra, and Tryvio) are expected to be dispensed in retail pharmacies.

Expedited development and review pathways played a large role in 2024.  While 44% (22 of 50) of the products had Fast Track approvals, 36% (18 of 50) of the products were considered breakthrough therapies.  Priority reviews accounted for 56% of the approvals (28 of 50), and 14% (7 of 50) had accelerated approvals.  Of the 50 novel drug approvals in 2024, 33 products used at least one form of expedited development; PHSL expects that manufacturers will continue to seek these expedited approval pathways, so long as they remain available.

Oncology

Oncology is the therapeutic category with the most drug approvals in 2024.  The FDA approved 16 new drugs for different forms of cancer:  Anktiva, Bizengri, Duvyzat, Ensacove, Imdelltra, Itovebi, Lazcluze, Lumisight, Ojemda, Revuforj, Rytelo, Tevimbra, Unloxcyt, Voranigo, Vyloy, and Ziihera.

FDA Program Benefits

The FDA Generating Antibiotic Incentives Now (GAIN) program is generating results. Three novel approvals for infectious disease were granted extended exclusivity via this incentive program. Orlynvah treats uncomplicated urinary tract infections, Exblifep treats complicated urinary tract infections, and Zevtera treats certain bloodstream infections, bacterial skin and associated tissue infections, and community-acquired bacterial pneumonia.

PHSL often helps manufacturers prepare for the health IT considerations behind the scenes when planning for a first-in-class approval to ensure that prescribers, pharmacists, payers, and other stakeholders receive important information about these products.  This article only contains new molecular entities approved in 2024.  To see all of the approvals that occurred in 2024, please visit the FDA’s Drugs@FDA website. The FDA report on novel approvals is a great resource for additional details about these 50 products.

Winter 2025 Newsletter:

What pharmacies need to know about dispensing e-prescriptions during a cyberattack