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

Mechanism-Based Drug Rejection? Ataluren’s Unlikely Personalized Medicine Milestone

Executive Summary

US FDA has shown it can be very flexible in expanding indications for a targeted therapy with a clearly established mechanism of action. But, as PTC Therapeutics learned, the agency’s willingness to apply mechanistic knowledge for targeted therapies cuts both ways.

You may also be interested in...



Cystic Fibrosis Failure Weakens PTC's Duchenne Argument, FDA Says

US FDA sees parallels in ataluren’s development in Duchenne and firm's unsuccessful efforts to find efficacious subsets in cystic fibrosis.

Patients Can't Clear Translarna's Data Hurdles As PTC Falls Short At FDA Panel

Patient testimony to the contrary, advisory committee members were not persuaded that PTC Therapeutics' ataluren, which failed its primary endpoint in two trials, is effective in treating nonsense mutation Duchenne muscular dystrophy.

Kalydeco Expands Indication Without Clinical Data; Keytruda Is Latest Bladder Cancer Approval

Keeping track of US FDA approvals last week included watching the creative and the familiar.

Related Content

Topics

Related Companies

UsernamePublicRestriction

Register

PS121699

Ask The Analyst

Please Note: You can also Click below Link for Ask the Analyst
Ask The Analyst

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