Alnylam Gets Orphan Designation for Subcutaneous Hemophilia Drugs

Factor for hemophiliacs may be getting a subcutaneous look after all.  Today, according to a press release from Market Watch of The Wall Street Journal, Alnylam Pharmaceuticals announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted Orphan Drug Designations for ALN-AT3 as an orphan medicinal product for the treatment of hemophilia A and hemophilia B. Alnylam is developing ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT), for the treatment of hemophilia and other Rare Bleeding Disorders (RBD).

“We are very pleased to have received Orphan Drug Designations from the EMA COMP for ALN-AT3, a key program in our ‘Alnylam 5×15’ product development and commercialization strategy. We believe that our subcutaneously delivered RNAi therapeutic represents an innovative approach for the management of hemophilia and has great potential to make a meaningful impact in the treatment of this often debilitating bleeding disorder,” said Saraswathy (Sara) Nochur, Ph.D., Senior Vice President, Regulatory Affairs and Quality Assurance at Alnylam. “Having announced positive top-line results in adult healthy volunteers earlier this year, we look forward to presenting initial Phase 1 results, including data in hemophilia subjects, later in the year.”

Orphan Drug Designation provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union and where no satisfactory treatment is available. Alnylam will be discussing its progress with the ALN-AT3 program with webinars throughout the summer and can be accessed on Alnylam’s website by clicking here.

Photo Credit – Alnylam Pharmaceuticals 

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