Regulatory Focus™ > News Articles > First EU-Authorized Gene Therapy Hits Snag, Benefit Assessment Delayed in Germany as EMA Reviews New

First EU-Authorized Gene Therapy Hits Snag, Benefit Assessment Delayed in Germany as EMA Reviews New Report

Posted 17 April 2015 | By Michael Mezher 

First EU-Authorized Gene Therapy Hits Snag, Benefit Assessment Delayed in Germany as EMA Reviews New Report

Regulators at the German Federal Joint Commission (G-BA) have postponed their review of the gene therapy Glybera, Reuters reports. The decision was made after the European Medicines Agency (EMA) rapporteur assigned to Glybera found the product lacked efficacy and called for it to be reassessed.

Glybera, which treats a rare condition called lipoprotein lipase deficiency (LPLD), made headlines both for being the first gene therapy approved in Europe and for its record-breaking € 1.1 million price tag.


Gene therapy works by modifying a patient's genes to correct for a genetic issue. Though researchers have been working to develop gene therapies for the past 30 years, few products have reached advanced stages of development. Because so few gene therapies have been taken to the regulatory stage, regulators have struggled with how to assess the products.

Initially, Glybera was given a negative opinion by EMA's Committee on Advanced Therapies (CAT), which was formed in 2007 to assess advanced therapies such as cell and gene therapy. Following a request for reexamination, the CAT reversed its opinion. However, CHMP refused to recommend Glybera, saying the data submitted did not have enough clinical trial subjects to be statistically significant.

Eventually, the European Commission (EC) asked CHMP to reexamine Glybera, and after a total of four reexaminations, CHMP reversed its opinion, agreeing to authorize the product under "exceptional circumstances," on the condition its makers would send the agency additional clinical data each year and establish a registry for long-term tracking of patients.

In 2012, Amsterdam Molecular Therapeutics (AMT), Glybera's original developer, was liquidated and sold its assets to UniQure. In a circular sent to its shareholders, AMT cited the "regulatory setbacks" as a contributing factor to its dissolution.

Rapporteur's Report and G-BA Postponement

According to a US Securities and Exchange Commission (SEC) filing by UniQure, the company received an assessment written by the CAT rapporteur responsible for overseeing Glybera. Based on the clinical evidence and follow up data, the rapporteur concluded that Glybera "lacks efficacy," and recommended that EMA require supplemental information from the company before making a final recommendation.

The CAT is currently meeting to review the report, which will move to CHMP for review from 21-23 April 2015.

Before the report was distributed, G-BA was in the midst of reviewing Glybera to determine its benefit. The assessment conducted by G-BA is used in price negotiations in Germany once a product has been on the market for one year. In light of the rapporteur's report, G-BA says it will delay its assessment, which was initially due by the end of April, until EMA reaches a conclusion.

Reuters, UniQure SEC Filing


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