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

SMA Patient Meeting Showcases FDA Flexibility – And Appreciation From Advocates

Executive Summary

US agency's top neurology review officials received warm reception during Patient Focused Drug Development meeting for Spinal Muscular Atrophy. They also delivered a strong message of continued engagement beyond the first drug approval.

You may also be interested in...



XLH Patient Meeting Showcases Value Of Post-Approval Feedback To US FDA

X-Linked Hypophosphatemia patient feedback meeting follows recent approval of Ultragenyx’ Crysvita. Like an SMA meeting in 2017, it showed how post-approval meetings can help sustain momentum in a new class.

Orphan Drug Development May Be Well-Suited To Digital/Wearables

Pfizer feels that orphan products may present a particularly good area for testing wearable monitors and other digital devices as research tools. The small patient populations and fine distinctions in functional improvement or delay of disease progression may be ideally suited for the digital collection of data.

FDA’s Remarkable ALS Drug Approval: Call It The 'Hendeles Standard'

US FDA’s approval of Mitsubishi's Radicava (edaravone) is yet another demonstration of the agency’s flexibility for drugs to treat rare diseases – and pre-emptive action to fend off “Right to Try” laws. It is also a belated endorsement of one of the stranger votes by an FDA advisory committee member almost a decade ago.

Related Content

Topics

Related Companies

Latest News
See All
UsernamePublicRestriction

Register

PS120496

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