Stealth Biotherapeutics Inc. had hoped the U.S. FDA would have approved its lead candidate, elamipretide, as the first treatment for Barth syndrome by now. Instead, it’s headed to a meet-up with the agency’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) Oct. 10. The discussion and vote at that meeting could be make-or-break for patients with the ultra-rare debilitating mitochondrial disease that has no approved therapies. “Barring support from CRDAC, the future of elamipretide for Barth syndrome in the U.S. is tenuous," Stealth CEO Reenie McCarthy told BioWorld.
Recordati SpA is shelling out $825 million up front for global rights to Enjaymo (sutimlimab), the only therapy approved for treating the rare disease cold agglutinin disease. In the deal with Sanofi SA, which won U.S. FDA approval of the antibody drug in 2022, the Italian pharma agreed to pay up to $250 million more should net sales reach certain thresholds.
Both chambers of the U.S. Congress put aside their election year politicking Sept. 25 long enough to pass a continuing resolution that will keep the government running at its current funding level through Dec. 20.
Both chambers of the U.S. Congress put aside their election year politicking Sept. 25 long enough to pass a continuing resolution that will keep the government running at its current funding level through Dec. 20. The spending bill is now awaiting President Joe Biden’s signature.
A little more than a year after the U.S. FDA refused to review the NDA for Biohaven Ltd.’s ultra rare disease treatment, new and positive phase III data have changed the treatment’s momentum. The upbeat results came as a surprise to analysts and investors, with the stock having a strong day and the company prepping an NDA for a fourth quarter submission.
After missing the primary and secondary endpoints in its phase III study of losmapimod in treating the rare disease facioscapulohumeral muscular dystrophy (FHSD), Fulcrum Therapeutics Inc. is yanking the program’s plug. The selective p38α/β mitogen activated protein kinase inhibitor had a lot of money behind it. It was originally in-licensed from GSK plc and then, in May, Sanofi SA signed on to help Fulcrum develop and commercialize losmapimod for FHSD worldwide, excluding the U.S., in a deal worth $1.06 billion.
With a successful phase III study of plozasiran in hand, Arrowhead Pharmaceuticals Inc. plans to file an NDA with the U.S. FDA for treating the rare genetic disease familial chylomicronemia syndrome. While the data are strong, the company is playing catch-up to Ionis Pharmaceuticals Inc., which has a December 2024 PDUFA for its candidate, olezarsen, in the same indication.
While the Biden administration continues applauding the savings it claims will be delivered by the first round of Medicare negotiations, many U.S. patients and their families are worried about the cost of the biopharma price-setting program – a cost they measure not in dollars and cents, but in worsening illness and lives that may be lost to a downturn in innovation and an upturn in barriers to access.
After many months of jockeying, the U.S. FDA has approved Ascendis Pharma A/S’ hormone replacement therapy Yorvipath (palopegteriparatide) for treating hypoparathyroidism. Ascendis said this is the first and only treatment for adults with the rare endocrine disease.
A T-cell therapy from Adaptimmune Therapeutics plc has received accelerated approval from the U.S. FDA to treat advanced synovial sarcoma (SS). Tecelra (afamitresgene autoleucel), a CAR T targeting MAGE-A4, is the first engineered T-cell therapy for solid tumors and the first treatment option for the indication in more than a decade.
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