Scrip is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

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
UsernamePublicRestriction

ASH Data Bring UniQure Closer To Filing Hemophilia B Gene Therapy

Late-Breaking Data Suggest Activity At Level Needed For Approval

Executive Summary

The company plans to file with 52-week data for AMT-061 and is confident it won't face the same stringent requirements from the FDA that led to a complete response letter for BioMarin's Roctavian.

You may also be interested in...



Not Such A Sure Thing: FDA Knocks Back BioMarin’s Roctavian

BioMarin’s confidence that its first-ever hemophilia gene therapy would be cleared by the US FDA was shaken by a complete response letter that the company claims changed the requirements for approval.

uniQure Hemophilia B Deal With CSL Leaves M&A Fans Miffed

Observers have been expecting uniQure to be sold off as a whole but the Netherlands-based firm has chosen to sell just one asset - EtranaDez - and focus on advancing its Huntington’s disease gene therapy.

UniQure Aiming To Be First With Hemophilia B Gene Therapy

The company has come a long way since gene therapy flop Glybera, and claims best-in-class status for viral vector.

Topics

Related Companies

UsernamePublicRestriction

Register

OM005385

Ask The Analyst

Please Note: Click here for more information on the Ask the Analyst service.

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