Approvals
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.
The drug, for hereditary angioedema, is Ionis’ second wholly owned asset.
In Phase III studies, Tonmya showed significantly superior analgesic effects in patients with fibromyalgia versus placebo. The sublingual pill also led to better clinical outcomes.
Papzimeos is the first immunotherapy approved for recurrent respiratory papillomatosis, a rare lung disorder involving the development of benign tumors in the airways.
Insmed’s Brinsupri is the first DPP1 inhibitor approved by the FDA and the first treatment for bronchiectasis to reach the market.
PRESS RELEASES