Scrip is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

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

Sarepta Pursues DMD Gene Therapy Accelerated Approval To Be First To Market

New Data Analyses Swayed US FDA To Allow Early Filing

Executive Summary

Sarepta has experience pursuing accelerated approvals in the US for Duchenne muscular dystrophy therapies, but with a costly gene therapy will payers require data from a controlled Phase III study?

You may also be interested in...



Spotlight On High Doses Again After Pfizer Duchenne Gene Therapy Death

The death in Pfizer’s Phase I study could reflect wider safety issues for AAV-based gene therapy and could spur intervention from regulators in 2022.

With Sarepta’s Vyondys, US FDA Staff Saw Opportunity To Right Perceived Wrongs With Exondys

ODE I director Ellis Unger chafed at the notion of approving the Duchenne muscular dystrophy drug golodirsen on the same unvalidated surrogate marker, this time in the face of infection and renal toxicity concerns; OND’s Peter Stein granted Sarepta’s appeal while also calling attention to the company’s failure to timely conduct the eteplirsen confirmatory trial.

Bluebird’s Zynteglo Approval Kicks Off Commercial Operations

Bluebird bio’s Tom Klima told Scrip launch preparations are well under way, including negotiations with payers on outcomes-based reimbursement agreements.

Topics

Related Companies

Latest News
See All
UsernamePublicRestriction

Register

SC146799

Ask The Analyst

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

Thank you for submitting your question. We will respond to you within 2 business days. 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