EMA concedes Alnylam’s MAA for treatment of ATTR amyloidosis

European Medicines Agency asserted that Alnylam Pharmaceutical’s application for Marketing Authorisation has been accepted for review of for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR (hATTR) amyloidosis.  In the U.S., patisiran has Fast Track Designation, Breakthrough Therapy Designation, and an expanded Orphan Drug Designation for ATTR amyloidosis from the FDA.

Eric Green, Vice President and General Manager of the TTR program at Alnylam said: “We plan to work closely with the EMA and Committee for Medicinal Products for Human Use (CHMP) toward the goal of bringing patisiran to patients with hATTR amyloidosis in the EU as quickly as possible.”

The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted designation of patisiran as a Promising Innovative Medicine (PIM), supportive of its consideration for entry into the UK’s Early Access to Medicines Scheme (EAMS). EAMS vows to give access to medicines to patients with life-threatening or seriously debilitating conditions that do not yet have a marketing authorization when it is essentially mandated for treatment.