Pink Sheet is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

UsernamePublicRestriction
UsernamePublicRestriction

Pfizer Vyndaqel Advisory Committee Pits Orphan Flexibility Vs. Data Shortcomings

This article was originally published in The Pink Sheet Daily

Executive Summary

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.

You may also be interested in...



The Orphan Drug Boom: Gold Rush Or Flash In The Pan?

Rare disease drug development has ballooned in just three years, powered by the success of independent biotechs like Alexion, Big Pharma’s entrée into the field, and pressure from regulators and payors that is dis-incentivizing development of traditional primary care drugs. The positive momentum has intensified dealmaking in the orphan drug space and is giving investors confidence to back rare disease-focused start-ups. Some have coined the resulting movement the “orphan drug bubble,” but interest isn’t likely to deflate soon – at least not as long as Big Pharma continues to invest in the area and industry is able to sustain a favorable reimbursement climate for ultra-premium-priced drugs.

When Is A Single Pivotal Trial “Robust” Enough? FDA’s Katz Offers Examples That Could Spell Trouble For Pfizer’s Vyndaqel

The single pivotal trial of Pfizer’s Vyndaqel couldn’t offer p-values of less than 0.01, nor substantial evidence from multiple sites, which could spell trouble for the new drug application.

Pfizer Rare Disease Plans Back On Track With Resubmission of Tafamidis

Pfizer gained the therapy to treat a rare genetic neurologic disorder in its 2010 acquisition of folded protein company FoldRx.

Related Content

Topics

Related Companies

UsernamePublicRestriction

Register

PS074156

Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

Your question has been successfully sent to the email address below and we will get back as soon as possible. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts

Cancel