Affinia Therapeutics Inc. has obtained IND clearance from the FDA for AFTX-201, an investigational genetic medicine for the treatment of BAG3-associated dilated cardiomyopathy (DCM). The phase I/II UPBEAT trial will begin in the first half of this year.
Reauthorization of the rare pediatric disease priority review voucher (PRV) was included in the spending package signed into law Feb. 3 by U.S. President Donald Trump, removing at least one aspect of uncertainty facing rare disease companies, many of which have relied on the PRV’s transferrable option as a lucrative source of funding.
Amgen Inc.’s head of R&D James Bradner said his firm was “surprised” by the request from the U.S. FDA Jan. 16 to take Tavneos (avacopan) off the market – and the company said no. “We're in discussions with FDA, and we'll answer questions as we talk with them,” he added.
