Approvals
New analysis from Jefferies shows that rare disease and cancer drugs granted the status are especially likely to be approved.
In the Phase III FIBRONEER-IPF study, Jascayd demonstrated significant lung capacity improvements over placebo.
Due to policies regarding industry user fees, the FDA will not be able to accept any new drug applications for the duration of the government shutdown, according to Leerink Partners.
The first oral BTK blocker for chronic spontaneous urticaria, Rhapsido offers a more convenient treatment option for patients who still show symptoms after antihistamine treatment.
The FDA in September issued two rejections for spinal muscular atrophy therapies—both linked to manufacturing problems—and granted approvals in Barth syndrome and for a subcutaneous version of Merck’s Keytruda that could be key to the blockbuster’s future earnings.
The regulatory greenlight was backed by two Phase III trials that showed normalized growth hormone levels in patients with the rare pituitary condition. It’s the first approval for Crinetics Pharmaceuticals and something CEO Scott Struthers predicted “will transform people’s lives.”
For the last two years, Keytruda has reigned as the world’s top-selling drug—a distinction under threat with key patent protections expiring in 2028.
Perhaps the most interesting of the pile of FDA rejection letters was for Lykos Therapeutics’ MDMA therapy. Letters sent to Stealth BioTherapeutics, Regeneron and more were also released as the agency also promised future CRLs “promptly after they are issued to sponsors.”
While the approval of Leqembi Iqlik bodes well for Biogen and Eisai’s planned application for a subcutaneous induction regimen next year, its financial impact remains “uncertain,” as potentially higher revenues from the injection could be offset by steeper costs of production, according to Jefferies.
Aside from the rare disease market, Novo Nordisk also scored a key regulatory win last month for its blockbuster GLP-1 drug Wegovy, which can now be used to treat patients with metabolic dysfunction-associated steatohepatitis.
PRESS RELEASES