OBR OncologyFDA Ends Busy Year with Three Drug Rejections

Advances and Setbacks in Drug Approvals: A Glimpse into FDA’s Rigorous Evaluation Process

The Food and Drug Administration’s (FDA) role in regulating drug approvals is a pivotal aspect of healthcare, influencing treatment options and outcomes for various diseases. In 2023, while the FDA approved a significant number of new drugs, it also issued rejections to key applications by major pharmaceutical companies. These decisions highlight the intricate balance between innovation and safety, reflecting the FDA’s commitment to rigorous evaluation standards.

Key Points:

• FDA Rejections: The FDA concluded 2023 by rejecting applications from Amgen, Zealand Pharma, and Merck & Co., for cancer, low blood sugar, and chronic cough treatments.
• Record Drug Approvals: The FDA’s main review office approved 55 new drugs in 2023, the highest since 2018, signaling a fruitful outcome of biotech investments and clinical successes.
• Conditional Approval and Challenges: Amgen’s Lumakras, for lung cancer, received conditional approval but now faces a four-year deadline to prove its superiority over existing treatments.
• Amgen’s Clinical Trial Hurdles: In Amgen’s CodeBreak 200 trial, patient dropouts and minimal survival benefits in the chemotherapy arm led to skepticism about full approval.
• Zealand’s Manufacturing Setback: Zealand’s dasiglucagon for congenital hyperinsulinism was rejected due to issues at a third-party manufacturer, not efficacy or safety concerns.
• Merck’s Drug Efficacy Concerns: Merck’s gefipixant for chronic cough was rejected due to insufficient evidence of effectiveness, despite no safety concerns.
• Biologics Office Approvals: The FDA’s biologics office cleared 15 medicines, including groundbreaking vaccines and gene therapies.
• Notable Approvals: The approvals included treatments for Alzheimer’s disease, menopause symptoms, ALS, multiple myeloma, and RSV in babies.
• First CRISPR Medicine: The biologics office also approved the world’s first CRISPR medicine, marking a significant milestone in gene therapy.

HCN Medical Memo
According to the Pharmaceutical Research and Manufacturers of America, the average time from drug discovery to FDA approval is 10-15 years, involving a cost of about $2.6 billion. This underlines the complexity and resource-intensity of bringing new drugs to market.

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