1. Pink Sheet
  2. >>Geography
  3. >>North America
  4. >>United States

Pfizer Vyndaqel Advisory Committee Pits Orphan Flexibility Vs. Data Shortcomings

 
• By Martin Berman-Gorvine

FDA finds much to question in a single pivotal trial of Pfizer’s orphan drug Vyndaqel (tafamidis meglumine) for familial amyloid polyneuropathy, but the ultra-orphan nature of the disease may outweigh those concerns.

Pfizer Inc. must be hoping to appeal to FDA’s flexibility on orphan drug reviews as the advisory panel tasked with reviewing Vyndaqel (tafamidis meglumine) weighs a dispute between agency and sponsor over the statistical reading of the pivotal trial results, and the agency notes ominously that the bulk of the efficacy seems to come from a single, foreign site.

Most of FDA’s approval decisions for orphan products use unconventional efficacy standards, according to a report released last year by the National Organization for Rare Disorders, which helps make the case for anticipating some regulatory flexibility Also see "

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet 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 United States

Medicare Negotiation Does Not Violate Due Process, Appeals Court Rules, Other Suits At Risk

 
• By Cathy Kelly

Decision in AstraZeneca’s lawsuit against the Health and Human Services Department is a precedent-setting victory for the government and a blow to manufacturer efforts to block the price negotiation program.

Keep Talking: US FDA’s Beleaguered Biologics Center Remains Vital To Regenerative Medicine

 
• By Bridget Silverman

Advisory committee for Capricor’s deriamocel planned, regenerative medicine advanced therapy designations proliferate for osteoarthritis, and the ranks of dual RMAT/breakthrough therapy designation holders grow.

Pink Sheet Podcast: A New Director For US FDA’s Biologics Center, Guidance Production Slowdowns

 
• By Derrick Gingery, Sue Sutter, and Nielsen Hobbs

Pink Sheet editors discuss the appointment of Vinay Prasad as the new director of the Center for Biologics Evaluation and Research and revelations that FDA layoffs now are hindering development of guidance documents.

US FDA Restoring Some Library Resources

 
• By Sue Sutter and Derrick Gingery

Agency staff once again have access to the Lexis-Nexis legal and news database, but are still waiting for access to journals, and laid off librarians have not been reinstated.

More from North America

Recent and Upcoming FDA Advisory Committee Meetings

 
• By Sue Sutter

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.

Most Favored Nation Pricing Policy Floated In Medicaid, Coming Back To Medicare?

 
• By Cathy Kelly

President Trump's ongoing interest in the policy, which would ensure the US government pays no more for prescription drugs than the lowest price available in comparable countries, continues to gain attention.

US FDA Advisory Committee Misperceptions Abound … At HHS

 
• By Michael McCaughan

HHS Secretary Robert F. Kennedy Jr. regularly decries the “conflicts of interest” he believes abound in advisory committees, but his concerns, as well as a recent “policy directive” eliminating industry representatives, seem driven by a misunderstanding of the panels’ jobs.