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

Orphan Exclusivity For Gene Therapies Hinges On Two Big Factors

Executive Summary

US FDA draft guidance says that ‘minor differences’ in transgenes and/or vectors won’t be enough for orphan exclusivity, but agency will consider ‘additional features of the final product’ in making the determination.

You may also be interested in...



Bluebird’s Lost Voucher: Lyfgenia Contains Same ‘Active Ingredient’ As Zynteglo, US FDA Says

Surprise denial of rare pediatric disease priority review voucher could prompt calls for the agency to re-examine how it interprets the term ‘active ingredient’ for gene therapies. Bluebird asserts there are significant differences between its gene therapies for treating sickle cell and beta-thalassemia.

US FDA Tweaks Guidance On Sameness Of Gene Therapies, But Questions Remain

In response to industry input, the agency made a few changes to its draft guidance but several issues remain uncertain, such as what constitutes ‘minor differences’ between gene therapy products.

Gene Therapies: US FDA Clarity Needed On ‘Sameness’ Determinations For Products From Same Vector Class

Agency also should better define, and provide examples of, the types of minor differences and additional features that would affect orphan drug designation and exclusivity determinations, industry sponsors say in comments on January draft guidance.

Related Content

Topics

Latest Headlines
See All
UsernamePublicRestriction

Register

PS141584

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