CHMP positive on first gene therapy, Glybera; and more opinions

Reversing an earlier negative opinion (The Pharma Letter April 22), the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) on Friday issued a positive opinion that recommends marketing authorization of Netherlands-based uniQure’s Glybera (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD).

The therapy was originated by Amsterdam Molecular Therapeutics, which was taken over by venture capital backed uniCure earlier this year, in what was seen as the only solution to gaining funding to continue the Dutch firm’s stem cell therapies research and development activities (TPL February 22).

“The evaluation of this application has been a very complex process, but the use of Glybera in a more restricted indication than initially applied for, which targets the patient population with greatest need for treatment, and additional analyses by the Committee for Advanced Therapies (CAT) have added to the robustness of the data provided and allowed the CHMP to conclude that the benefits of Glybera are greater than its known risks,” said Tomas Salmonson, acting chairman of the CHMP. “Our established ways of assessing the benefits and risks of Glybera were challenged by the extreme rarity of the condition and also by uncertainties associated with data provided. In close cooperation with the CAT we have worked out a way to ensure robust and close follow-up of the quality, safety and efficacy of Glybera while giving patients who have to live with this rare disease access to a medical treatment,” he explained.