The Food and Drug Administration closed out 2024 with a series of significant drug approvals, including new medicines developed by Vertex Pharmaceuticals, Novo Nordisk, and Bristol Myers Squibb for cystic fibrosis, hemophilia, and cancer, respectively.
This year, the FDA’s main review office cleared 50 novel medicines, slightly fewer than the 55 approvals in 2023 but still on the higher end of annual totals. In addition, the FDA’s biologics office issued 10 new medicine approvals in 2024.
Vertex Pharmaceuticals received approval for Alyftrek on December 20, extending the company’s leadership in cystic fibrosis treatment. Alyftrek, a three-drug combination similar to Vertex’s Trikafta, offers a once-daily dosing regimen and demonstrated non-inferiority to Trikafta in lung function improvement. The drug is priced at around $370,000 per year, slightly higher than Trikafta.
Novo Nordisk’s Alhemo was approved on the same day for the prophylactic treatment of bleeds in individuals with hemophilia A or B with inhibitors. This approval provides an alternative for patients who develop an immune response to other therapies, unlike Pfizer’s Hympavzi.
Bristol Myers also secured approval for Opdivo Qvantiq on December 27, a subcutaneous form of the cancer immunotherapy Opdivo. This new formulation allows for quicker administration and is protected by newer sets of patents, with the main patent set to expire in 2028.
Lastly, Lilly’s Zepbound received approval for the treatment of obstructive sleep apnea in adults with moderate-to-severe cases and obesity. This expands the indications for incretin medicines, which have shown benefits for heart failure, kidney disease, and liver disorders.
Overall, the FDA’s approvals in 2024 reflect the ongoing innovation in drug development and the commitment of pharmaceutical companies to addressing unmet medical needs. These new treatments offer hope for patients with cystic fibrosis, hemophilia, cancer, and sleep apnea, showcasing the advancements in medical science.
