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

BioMarin’s Orphan Drug Clinical Strategy Is Rooted In Natural History

This article was originally published in Pharmaceutical Approvals Monthly

Executive Summary

BioMarin has emerged at the forefront of orphan drug development by grounding its clinical trials in natural history studies. The specialty firm’s recent investor day highlighted how clinical trials in ultra-orphan disorders are informed by efforts to quantify the clinical course of often poorly-understood conditions to determine appropriate endpoints and dosing strategies.

You may also be interested in...



Baby Steps To Real-World Evidence Of Efficacy: External Controls Gain Popularity In Rare Disease Trials

The Pink Sheet takes a closer look at recent clinical announcements showing how rare disease therapy sponsors are already increasingly reliant on natural history studies to guide drug development.

FDA Orphan Approval Flexibility Remains Steady As Pressure Increases

Frank Sasinowski updates his 2012 landmark analysis and finds that FDA maintained its rate of flexibility in the evidentiary standards for approval of orphan products.

Acer’s ‘Misunderstood Drugs’ Strategy Faces Regulatory Hurdles

Acer repurposes drugs with available data (like a 40-year-old European antihypertensive) into rare disease treatments (like Edsivo for vascular Ehlers-Danlos syndrome), but approval remains elusive, with a CRL for ACER-001 just as Edsivo is poised to enter a new clinical trial, armed with a new breakthrough designation. The Pink Sheet delves into regulatory history and strategy with CEO Chris Schelling.

Topics

Related Companies

Latest News
UsernamePublicRestriction

Register

PS005851

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