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.
Gene and cell therapies will drive innovation for the next 10 years, Claus Zieler, the chief commercial officer at Astellas Pharma Inc., said in the newest episode of the BioWorld Insider podcast. Developers are on the cusp of breakthroughs because a gene can now be replaced “and that means we can potentially cure a disease rather than intervening in a disease.”
A growing foray of pharmacy benefit managers’ (PBMs) private labels into the U.S. biosimilar space is intensifying concerns about the antitrust aspects of PBMs’ vertical integration that has them serving as price negotiator, formulary setter, payer, group purchasing organization, pharmacy, provider and now drug "manufacturer."
Round 2 of the U.S. Medicare price negotiations officially began late Oct. 2 with CMS’ release of its final playbook and timeline, which build on lessons learned from the first round and comments on a draft negotiations guidance that was published in May. In response to the feedback CMS received, the second round will allow for more patient-focused sessions and offer increased engagement with the makers of the drugs selected for negotiations.
It’s well past time for the U.S. FDA to end its silence on what device patents can be listed in the Orange Book as part of a drug-device combination product, Sen. Bill Cassidy (R-La.) said in an Oct. 1 letter that took FDA Commissioner Robert Califf to task for letting the FTC do the FDA’s job.
Akeso Pharmaceuticals Inc. scored two approvals from China’s National Medical Products Administration on Sept. 30 before the long Labor Day holiday – one for its PCSK9 inhibitor, ebronucimab, and the second to expand use of PD-1/CTLA4 bispecific antibody cadonilimab in unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma, marking the second indication for cadonilimab in China.
Meeting the deadline the U.S. Health Resources & Services Administration imposed in a Sept. 27 final warning letter, Johnson & Johnson (J&J) notified the agency Sept. 30 that it would not implement its proposed 340B rebate model pending resolution of the issues.
GE Healthcare Technologies Inc. received U.S. FDA approval for its novel radiotracer, Flyrcado (flurpiridaz F-18), for use in the diagnosis of myocardial ischemia or infarction in patients with known or suspected coronary artery disease.
Capricor Therapeutics Inc. just wrapped up a visit with the U.S. FDA and is prepping to file a BLA in October for its Duchenne muscular dystrophy treatment. Linda Marbán, the company’s CEO, is the guest on the newest BioWorld Insider podcast and she talks about deramiocel (CAP-1002), the company’s allogeneic cardiac-derived cell therapy, for treating the rare disease and how the FDA has made strong efforts in helping lay the groundwork for deramiocel.
The U.S. FDA has lifted the full clinical hold it imposed in June on Biomea Fusion Inc.’ s phase I/II studies of BMF-219 in types 1 and 2 diabetes. A safety review of the phase IIb expansion study was encouraging and none of the elevated lab values confirmed serious liver injury or impairment, said Biomea’s CEO, Thomas Butler.
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