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Panel: Tafamidis data predictive of benefit, but efficacy evidence not robust

 
• By Donna Young

A panel of advisers to the US FDA on 24 May did not find substantial evidence of efficacy on a clinical endpoint in Pfizer's single study of its experimental drug tafamidis meglumine in treating transthyretin familial amyloid polyneuropathy (TTR-FAP), but said the data were strong enough to match what might be found by a surrogate endpoint likely to predict a clinical benefit.

A panel of advisers to the US FDA on 24 May did not find substantial evidence of efficacy on a clinical endpoint in Pfizer's single study of its experimental drug tafamidis meglumine in treating transthyretin familial amyloid polyneuropathy (TTR-FAP), but said the data were strong enough to match what might be found by a surrogate endpoint likely to predict a clinical benefit.

The FDA's Peripheral and Central Nervous System Drugs Advisory Committee voted 13-4 that the findings of Pfizer's study 005 were...

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More from Alimentary/Metabolic

MASH Updates: Sagimet, Altimmune, GSK Discuss Development Plans At AASLD

 
• By Joseph Haas

Sagimet may shift focus to later-stage MASH while it lines up Phase III financing. Altimmune expects its dual agonist to show efficacy and tolerability, while GSK is readying efimosfermin as the third FGF21 analog into Phase III for MASH.

Madrigal May Have Head Start On Demonstrating Cirrhosis Benefit

 
• By Joseph Haas

At AASLD, Madrigal presented Phase III open-label data showing resmetirom can improve liver stiffness and other liver health measures in patients with cirrhosis. It hopes for outcomes data in 2027.

Novo-Emcure Semaglutide Deal Clever Counter To Indian Rivals, Follows Lilly-Cipla Pact

 
• By Vibha Ravi

Novo Nordisk’s deal with Emcure for semaglutide will launch Poviztra as a second obesity brand in India, improving access to weight loss treatments following a Lilly-Cipla deal for tirzepatide and seems a clever counter to Indian majors planning to launch generic semaglutide next year

Pfizer Wins Out In Metsera Bidding War

 
• By Mary Jo Laffler

Novo backs out, leaving Pfizer to complete its acquisition of the obesity specialist for nearly $3bn more than its initial offer.

More from Therapeutic Category

A Week Is A Long Time In Biotech, Just Ask Galecto

 
• By Kevin Grogan

A long time on the ropes, the US firm is now well-financed to advance potential candidates for essential thrombocythemia and myelofibrosis into the clinic.

AnaptysBio Scraps Rosnilimab Ulcerative Colitis Trial After Phase II Miss

 
• By Sushmita Panda

The biopharma plans to advance the PD-1 agonist in rheumatoid arthritis despite its failure in ulcerative colitis.

Neurocrine’s Depression Drug Misses In Phase II

 
• By Andrew McConaghie

Small study fails to turn up efficacy signals in the major depressive disorder candidate, leaving Neurocrine with one remaining depression candidate in development.