EU's Gene Therapy Approval Represents Major Advancement

First Gene Therapy Approval by a Major Health Regulatory Authority

November 7, 2012
Manufacturing Group
Materials People/Facilities

Officials for Cardium Therapeutics report that uniQure's Glybera (alipogene tiparvovec) approval by the European Commission, the first gene therapy approval by a major health regulatory authority, represents a significant milestone and validation for the gene therapy industry. 

Glybera is a treatment for patients diagnosed with an inherited metabolic disease called familial lipoprotein lipase deficiency (LPLD or familial hyperchylomicronemia), who suffer from severe or multiple pancreatitis attacks despite dietary fat restrictions.  The European Commission's marketing authorization of Glybera covers all 27 European member states and uniQure plans to apply for regulatory approval in the United States, Canada, and other countries.

"The EU approval of Glybera represents a major milestone for the global gene therapy industry," states Christopher J. Reinhard, chairman and CEO of Cardium. "This is an important step forward for our field and the millions of patients expected to benefit from new and innovative gene-based therapeutics.  Gene therapy offers the opportunity to simplify treatments for serious medical problems and to develop new products for which there are no current medical treatments."

Cardium's late-stage gene therapy Generx product candidate (Ad5FGF-4) is a disease-modifying interventional cardiology biologic being developed as a one-time non-surgical treatment for patients with coronary artery disease. Generx can be delivered using a standard cardiac catheter and is capable of promoting and enhancing cardiac perfusion in the heart through the enlargement of pre-existing collateral arterioles (arteriogenesis) and the formation of new capillary vessels (angiogenesis).