Following Negative Comments by Reviewer, EU Committee OK's Gene Therapy

Posted 06 May 2015 | By Michael Mezher 

Following Negative Comments by Reviewer, EU Committee OK's Gene Therapy

Last month, German regulators postponed their review of UniQure's gene therapy Glybera after the European Medicines Agency (EMA) rapporteur assigned to the product claimed new data showed Glybera lacked efficacy and called for it to be reassessed.

Now, EMA's Committee for Advanced Therapies (CAT) has completed its assessment, and is maintaining its positive opinion of Glybera, Reuters reports.


Glybera is the first gene therapy approved for use in the EU, and is intended to treat a rare condition called lipoprotein lipase deficiency (LPLD). The therapy made headlines last year for its record-breaking €1.1 million price tag.

As the first EU-authorized gene therapy, Glybera's development was marred by regulatory setbacks. After initially receiving a negative opinion from CAT and undergoing several reassessments, Glybera was eventually authorized under "exceptional circumstances." Due to the small populations in the clinical trials supporting Glybera's authorization, EMA required its makers to submit additional clinical data annually for six years and establish a registry for long-term patient tracking.

CAT Gives Positive Assessment

After receiving additional follow up data, the CAT rapporteur for Glybera said the new data showed Glybera lacked efficacy and recommended EMA request supplemental information from the company before continuing its recommendation for the product.

According to Reuters, UniQure received CAT's assessment of Glybera, which did not support the findings of the rapporteur. Both CAT and EMA's Committee for Medicinal Products for Human Use (CHMP) discussed Glybera during their April 2015 meetings.

Now that CAT has continued its endorsement of Glybera, it is expected that the German Federal Joint Committee (G-BA) will continue its assessment as planned on 7 May 2015.



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