After a long regulatory road that included a complete response letter in May, Stealth Biotherapeutics Inc. finally got its Barth syndrome drug across the finish line, with the U.S. FDA granting accelerated approval to Forzinity (elamipretide HCl) to improve muscle strength in those with the ultra-rare pediatric mitochondrial cardioskeletal disease.
Starpharma Holdings Ltd.’s stock shot up 73% on the news that it is outlicensing its dendrimer enhanced product drug delivery technology to Roche Holding AG subsidiary Genentech Inc. in a deal worth more than $569 million.
As the first – and so far only – drug to enter clinical testing for the rare neurogenetic disorder Alexander disease, there were some unknowns heading into the readout of the pivotal study testing Ionis Pharmaceuticals Inc.’s zilganersen in children and adults. But the top-line data yielded a clear win for the antisense oligonucleotide candidate, which demonstrated a disease-modifying impact, including statistical significance on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test vs. control at week 61.
The U.S. FDA approved Merck & Co. Inc.’s Keytruda Qlex (pembrolizumab and berahyaluronidase alfa-pmph) injection on Sept. 19, making it the first and only subcutaneously (SC)-administered immune checkpoint inhibitor that can be administered in about a minute.
MBX Biosciences Inc. posted positive top-line phase II study data in treating chronic hypoparathyroidism, positioning itself to take on a U.S. FDA-approved therapy for the rare endocrine disease. MBX’s Avail study of canvuparatide hit its primary endpoint in treating adults, demonstrating statistical significance at week 12 and positive six-month results from the open-label extension study. MBX said it is poised to begin a phase III study of once-weekly canvuparatide next year.
Regulatory snapshots, including global drug submissions and approvals, and other regulatory decisions and designations: Astrazeneca, Biogen, Curatis, Deciphera, J&D, Minovia, Replimune.
Biopharma happenings, including deals and partnerships, and other news in brief: Arvinas, Alcyone, Austrianova, Avant, Biocardia, Biogen, Bluebird, Genetix, Klothonova, Infinimmune, Immunome, Novo Nordisk Pfizer, X4.
RSS
