Pfizer’s orphan drug tafamidis will be reviewed by FDA’s Peripheral and Central Nervous System Drugs Advisory Committee in May. The scheduling of a panel during a priority review is some indication FDA has concern, but the panels of outside experts are generally receptive to rare disease treatments.
Pfizer Inc.will face another test of its rare disease group when its transthyretin familial amyloid polyneuropathy drug Vyndaqel (tafamadis meglumine) goes before FDA’s Peripheral and Central Nervous System Drugs Advisory Committee May 24.
FDA officials have repeatedly noted the difficulty of holding an advisory committee review in the tight timeframe of a six-month...
Nonvoting industry representative’s presence at Pediatric Advisory Committee meeting was required by statute, the agency said, a de facto acknowledgement that an April directive precluding industry reps from sitting on advisory committees has had little practical effect.
Akin to the 505(b)(2) NDA, a new biologic pathway could reduce the need for animal testing, FDA toxicologist says. Expanded use of generally accepted scientific knowledge (GASK), which few sponsors have attempted despite a 2023 draft guidance, would also help.
Seven weeks before its action date and a month after an advisory committee was hastily cancelled, Capricor gets a CRL for its cell therapy deramiocel in DMD-related cardiomyopathy citing efficacy and manufacturing concerns. It has a Phase III study nearing readout, though.
Teva seeks a dedicated development program and first-cycle approval metrics for complex generics. Role of artificial intelligence in the generic drug review process is a talking point for both agency and industry at reauthorization kickoff meeting.
