The FDA is set to deliver close to 70 approvals and label expansions this year, with oncology as the top disease area. But uncertainty has lingered in recent months as the agency struggles to deal with internal and political turmoil, and new Commissioner Makary continues to push for faster approvals. A recent report from Leerink Partners suggests that submissions at greatest risk of delay include drugs that target novel diseases or use novel endpoints, and those with ambiguous trial results.
Drug Approvals, Advancing Clinical Trials, and More
The year 2024 is on track to be one of the most prolific for new drug approvals in decades. The FDA approved a total of 50 new drugs, with small molecules leading the way (62% of approvals). Highlights included Voranigo for gliomas with mIDH1/2 mutations, Alyftrek and Rezdiffra for cystic fibrosis, and a combination therapy for relapsed/refractory multiple myeloma.
One of the FDA’s biggest approvals of the year is expected to be Ionis Pharmaceuticals’ Donidalorsen for hereditary angioedema (HAE). The first-in-class drug reduces attacks by blocking clot-forming enzymes in the body. Analysts expect it to generate up to $8 billion in sales.
In other drug approval news, the FDA approved Pfizer’s Hympavzi and Novo Nordisk’s Alhemo to treat hemophilia A and B. JOURNAVX (suzetrigine) received approval for pain in adults with moderate to severe acute pain. The oral drug is a highly selective NaV1.8 pain signal inhibitor, and the first of its kind approved in more than 20 years.