EU's first-ever gene therapy bumps up against renewed regulatory scrutiny
- Glybera, Europe's first-ever approved gene therapy, is authorized to treat lipoprotein lipase deficiency and costs $1.18 million. But it's facing some new hurdles, RAPS reports.
- When Amsterdam Molecular Therapeutics first submitted its application for Glybera to the EMA, it was rejected. It was finally approved after four re-assessments for use under "exceptional circumstances" in 2012.
- UniQure is now the owner of Glybera.
A new re-assessment report from the EMA's Committe on Advanced Therapies (CAT) has now looked at clinical and follow-up data, which puts Glybera's efficacy in question. In fact, the assessor has determined that Glybera lacks efficacy.
The new recommendation to the EMA is that more information is needed from UniQure. Later this month, the CAT is meeting to review the evidence, and there is the distinct possibility that Glybera will lose its authorization.
In fact, few gene therapy products have been approved or even reached advanced stages of development, so although this seems like a long, confusing and drawn-out process, it could help better define the regulatory pathway for gene therapies.
- Regulatory Affairs Professional Society First EU-Authorized Gene Therapy Hits Snag, Benefit Assessment Delayed in Germany as EMA Reviews New Report