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UniQure's Glybera OK with European Ad Comm; requests additional info on Type II variation request • 11:57 AM
Douglas W. House, SA News Ed...
The European Medicines Agency's (EMA) Committee for Advanced Therapies (CAT), which advises the EMA's Committee for Human Medicinal Products (CHMP) on gene therapies does not agree with a rapporteur's earlier report that uniQure's (QURE +0.9%) Glybera (alipogene tiparvovec) lacks efficacy with a negative benefit-risk balance. Its recommendation, determined by majority vote, is that "the efficacy of Glybera needs to be considered in its totality as defined in the initial approval, taking into account the criteria considered at time of initial approval."
Since Glybera's November 2012 approval by the European Commission, uniQure has submitted several Type II variation requests to its Marketing Authorization Application (MAA) which includes additional six-year follow-up data. The variations have yet to be approved. CAT has not completed its review of the follow-up data and has requested supplemental information, which the company is currently preparing.
Part of the variation request process is a review of the benefit-risk analysis of Glybera. UniQure continues to strongly believe in Glybera's efficacy and favorable benefit-risk profile, but there remains some risk of an adverse decision from the EMA.