Alnylam makes progress on gene therapy in Europe
Alnylam Pharmaceuticals Inc., a Cambridge company that has allied with Genzyme to develop RNAi therapeutics across the world, has received a positive opinion from European officials on an application for orphan drug status to treat a rare genetic disease that causes fatal organ and nerve damage.
The company said in a statement that the European Medicines Agency Committee for Orphan Medicinal Products adopted a positive opinion recommending its drug, ALN-TTRsc, for designation as an orphan medicinal product.
This designation provides drug companies with financial and regulatory incentives to develop and market therapies that treat rare, life-threatening and debilitating diseases affecting no more than five in 10,000 people in the European Union. It includes marketing exclusivity for 10 years after product approval.
Alnylam is still conducting clinical trials for ALN-TTRsc, which employs RNAi (RNA interference) gene therapy to treat the degenerative disease known as transthyretin-mediated amyloidosis (ATTR).The company expects to present data from its Phase 2 clinical trial in late 2014.
In January, Alnylam and Genzyme Corp., which has operations in Framingham, formed an alliance to accelerate and expand development of RNAi therapeutics, with Alnylam retaining product rights in North America and Western Europe and Genzyme obtaining rights to commercialize three Alnylam products.
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