Pink Sheet 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

Accelerated Approval For Bluebird’s Skysona Gives Teeth To US FDA Data Questions

Executive Summary

Two required confirmatory studies will bolster application that relied on historical controls for orphan neurodegenerative disease.

You may also be interested in...



RWE On The Runway: Trio Of Upcoming Approval Decisions Will Test US FDA’s Thinking On RWE For Efficacy

Real-world evidence provides external controls for bluebird bio’s eli-cel and Ipsen’s palovarotene, while Reata used RWE to address FDA concerns about the pivotal single-arm study of omaveloxolone. In the post-market, Intercept is gearing up to seek full approval of Ocaliva relying in part on claims database.

Bluebird’s Eli-Cel Gene Therapy Gets US Panel Nod For Some Cerebral Adrenoleukodystrophy Patients

Committee endorses use for those who lack a matched donor for allogeneic hematopoietic stem cell transplant and says the treatment should be an option for patients with a matched, but unrelated, donor.

Lecanemab Conversion From Accelerated To Full Approval Could Be Among Fastest Ever

Biogen/Eisai’s Phase III data is on track for a Q1 2023 submission, potentially just weeks after lecanemab is expected to gain accelerated approval for early Alzheimer’s.

Topics

Related Companies

Latest News
See All
UsernamePublicRestriction

Register

PS147027

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