ALNY - Potential Label Restrictions Loom For Alnylam's Patisiran Despite Approval Prospects | Benzinga
On Wednesday, an FDA advisory committee voted 9-3 in favor of Alnylam Pharmaceuticals Inc's (NASDAQ: ALNY) patisiran. It has a favorable benefit-risk profile in the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.
The FDA has set an action date of October 8, 2023, under the Prescription Drug User Fee Act.
In a briefing document released before the Cardiovascular and Renal Drugs Advisory Committee, the FDA questioned the drug's efficacy.
While the committee's concerns about the degree of benefit, lack of outcomes data, and limited information on combination use with tafamidis are understandable, William Blair (Reiterates Outperform rating) maintains that the study showed statistical significance on measures ...