1. Scrip
  2. >>Therapy Areas
  3. >>Alimentary/Metabolic

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...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Alimentary/Metabolic

In Brief: Kezar Plots Lupus Revival For Zetomipzomib As FDA Lifts Hold On Hepatitis Trial

 
• By Manas Mishra

The go-ahead to resume a trial for its only drug candidate, zetomipzomib, in hepatitis could give Kezar a glimmer of hope of reviving another halted study in lupus nephritis.

Kailera Eyes Global Obesity Market With Hengrui’s Phase III Dual Agonist

 
• By Joseph Haas

With China’s Hengrui reporting successful Phase III data for its dual GLP-1/GIP agonist, licensee Kailera is readying plans to demonstrate better efficacy than current obesity drugs.

ProKidney Pins Hopes On Accelerated Approval For Rilparencel

 
• By Alaric DeArment

After posting encouraging Phase II results, the company plans to meet with the US FDA about using data from an ongoing Phase III study for an accelerated approval.

Eolo Pharma Advances In Obesity With Thermogenesis Approach

 
• By Aakash Babu

Emerging Company Profile: Early-stage biotech Eolo will seek cash and deals for its novel obesity drug SANA after positive Phase I results for weight loss, lean muscle mass preservation and fat mass reduction.

More from Therapeutic Category

Replimune CRL Could Hold Warning For Accelerated Approvals

 
• By Jessica Merrill

FDA issued a complete response letter to Replimune’s BLA for a novel oncolytic immunotherapy for melanoma in a surprise to management and investors.

Sanofi Doubles Down On Respiratory Vaccines With Vicebio Buy

 
• By Kevin Grogan

The French major is paying $1.15bn For the UK biotech and its bivalent candidate, VXB-241.

Alkermes To Advance Alixorexton After Positive Phase II Narcolepsy Results

 
• By Alaric DeArment

The biotech company is planning for Phase III based on positive topline results from Vibrance-1 in narcolepsy type 1 and remains optimistic about type 2 disease.