EMA-COMP Backs Alnylam's Orphan Drug Status Application For RNAi Drug

Alnylam Pharmaceuticals announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) is recommending the approval of ALN-TTRsc for orphan drug designation.

Genzyme and Alnylan have entered into a collaboration to develop and commercialize ALN-TTRsc in North America and Western Europe. The companies applied for ALN-TTRsc’s Orphan Drug designation for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR).

Saraswathy (Sara) Nochur, SVP of Regulatory Affairs and Quality Assurance at Alnylam, said, “We are very pleased to have received a positive opinion from the EMA COMP on our application for Orphan Drug Designation for ALN-TTRsc. We believe RNAi therapeutics represent a promising new approach for the treatment of ATTR, with the potential to make a meaningful impact for patients with this progressive and debilitating disease. We look forward to sharing Phase 2 clinical data from our ALN-TTRsc program later in the year, and, assuming continued positive results, we plan to advance to a Phase 3 pivotal trial in ATTR patients with TTR cardiac amyloidosis by the end of the year.”

Transthyretin (TTR)-mediated amyloidosis (ATTR) is an inherited and progressively debilitating disease resulting from mutations in the TTR gene.  Mutations in TTR lead to the build-up of abnormal amyloid proteins causing damage to body organs and tissue. This then leads to intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy. The disease is often fatal and presents a significant need for new therapeutics to treat patients afflicted with TTR amyloid polyneuropathy and/or cardiomyopathy.

RNA interference (RNAi) is one of the most promising frontiers in biology and drug discovery. Alnylam’s RNAi therapeutic platform target disease causes by ‘silencing’ specific mRNAs and preventing disease-causing proteins from being generated in the first place. ALN-TTRsc is currently undergoing a pilot Phase II clinical trial for the treatment of ATTR patients with TTR cardiac amyloidosis.

If approved, the company will gain a range of benefits from the Orphan Drug Designation by the European Commission. These include regulatory and financial incentives as well as a 10-year period marketing exclusivity in the European Union after product approval.