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

Mitochondrial Disease Workshop Shows US FDA Commitment To First Approvals

Executive Summary

First FDA-hosted workshop on rare family of mitochondrial diseases offers plenty of opportunities to talk challenges in R&D – and identify some solutions.

You may also be interested in...



Stealth Elamipretide NDA Tests Frontiers Of US FDA’s Flexibility In Rare Diseases

FDA wants another trial in ultra-orphan Barth syndrome, but could not identify a feasible design – so Stealth submitted the NDA anyways, based on a retrospective natural history control trial

Patients’ Preferred Endpoints May Not Satisfy Regulatory Needs, US FDA Says

Just because a measure is relevant to the patient community does not mean it is capable of capturing meaningful change in a clinical trial, US FDA officials say at a meeting on mitochondrial disease drug development.

NDAs, BLAs To Start Getting KASA Quality Risk Assessments After Successful Pilot With ANDAs

Internal review system reduces US FDA’s administrative work for OPQ risk assessments; agency’s standards will not change, and sponsors will not need to modify applications. Advisory committee endorses expansion, which will roll out to different application types over several years.

Topics

Related Companies

Latest News
See All
UsernamePublicRestriction

Register

PS140859

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