Caution Needed When Updating Europe's Orphan Drugs Procedures, Industry Says
This article was originally published in SRA
Executive Summary
The European Federation of Pharmaceutical Industries and Associations is urging caution in any updating or revising of Europe’s orphan drug procedures. The current process “works well, corrects a market failure and has allowed industry to develop more than 100 orphan drugs for more than 81 rare disorders,” the association told Scrip Regulatory Affairs.
You may also be interested in...
Pipeline Watch: Phase III Readouts in Wilson Disease, Parkinson's Disease And COVID-19
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
AZ's Rare Disease Unit Reports Positive Phase III Results For Copper-Binding ALXN1840
Alexion/AstraZeneca has announced positive top-line results from the Phase III study of ALXN1840 in patients with the rare genetic disorder, Wilson disease, including in patients already on standard-of-care therapies. The compound is ahead of two candidate gene therapies from other companies at early-stage clinical development.
Theravance's Gut-Selective JAK Inhibitor Disappoints In Ulcerative Colitis Study
Top-line results from a Phase IIb study of Theravance’s potential gut-selective JAK inhibitor izencitinib have not matched expectations, and results from a similar early-stage study in Crohn’s disease with the Janssen-partnered candidate are now awaited at the end of the year or in early 2022.