With the U.S. FDA approval of Attruby (acoramidis) for transthyretin amyloidosis with cardiomyopathy (ATTR-CM), Bridgebio Pharma Inc. is taking on an industry giant. The next-generation, oral, small-molecule stabilizer of transthyretin will take on Pfizer Inc.’s Vyndamax (tafamidis, Vyndaqel), a TTR stabilizer approved in 2019 for ATTR-CM but expected to lose exclusivity in late 2028. The approval was based on a phase III study that showed Attruby significantly reduced death and cardiovascular-related hospitalizations. The NDA, which had a Nov. 29 PDUFA date, was approved Nov. 22.
Armed with strong phase III safety data in Japanese patients, Palo Alto, Calif.-based Bridgebio Pharma Inc. is planning to file for Japan approval of its investigational drug acoramidis for a rare heart disorder.
Armed with strong phase III safety data in Japanese patients, Palo Alto, Calif.-based Bridgebio Pharma Inc. is planning to file for Japan approval of its investigational drug acoramidis for a rare heart disorder.
Following an unexpected FDA complete response letter (CRL), Alnylam Pharmaceuticals Inc. said it will no longer pursue an expanded indication for Onpattro (patisiran) in the U.S. The RNAi therapeutic was approved in 2018 to treat polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis and seemed well on its way to snagging a second U.S. indication after the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 9-3 in September that the drug’s benefits outweighed its risks as a treatment for cardiomyopathy of ATTR amyloidosis. The FDA disagreed with the committee, saying in the CRL that patisiran’s clinical meaningfulness had not been established in the proposed indication.
Although Pfizer Inc. has the only drugs approved in the U.S. to treat a rare, progressive heart disease, the U.S. Court of Appeals for the Second Circuit agreed this week with the Department of Health and Human Services, and a lower court, that Pfizer’s proposed copay assistance program for middle-income Americans covered by Medicare would violate the federal Anti-Kickback Statute – even if the company has no “corrupt” intent.
Citing the First Amendment, the Fifth Amendment and the logic of fair play, Pfizer Inc. is taking on a Health and Human Services’ (HHS) antitrust policy that prohibits drug companies from helping Medicare beneficiaries with hefty copays.
CLEVELAND – The list is out, and a dual-acting osteoporosis drug and a device for expanding the use of minimally invasive mitral valve surgery have come out on top.
