Alnylam receives approval of RNAi therapeutic ONPATTRO

Alnylam Pharmaceuticals has announced that the European Commission (EC) has granted marketing authorisation for ONPATTRO ™ (patisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy.

ONPATTRO is based on Nobel Prize-winning science and is the first-ever RNA interference (RNAi) therapeutic to be approved in the European Union.

“A lack of treatments to halt or reverse the progression of disease resulted in a gradual and inescapable decline in their day-to-day functioning”

Many serious, chronic and life-threatening diseases, such as hATTR amyloidosis, are caused by a fault or mutation that interferes with the way the body manufactures proteins. RNAi is a completely new approach to the treatment of these diseases; targeting the faulty protein that is causing the disease rather than treating the symptoms. RNAi therapeutics are an entirely new class of medicines.

The marketing authorisation for patisiran was based on positive results from the global Phase 3 APOLLO study, the largest-ever study in hATTR amyloidosis patients with polyneuropathy. In APOLLO, the safety and efficacy of patisiran were evaluated in a diverse, global population of hATTR amyloidosis patients in 19 countries. The study showed that patisiran improved measures of polyneuropathy, quality of life, activities of daily living, ambulation, nutritional status and autonomic symptoms relative to placebo in adult patients with hATTR amyloidosis with polyneuropathy.

Theresa Heggie, Head of Europe, Alnylam Pharmaceuticals said, “This approval is the result of many years of dedicated effort and marks the next step towards bringing a potentially life-changing treatment to patients with hATTR amyloidosis and their families. Patisiran has been shown to improve polyneuropathy, quality of life and activities of daily living.”

Professor Philip N. Hawkins, Ph.D, FRCP, FRCPath, FMedSci, National Amyloidosis Centre, Division of Medicine, and University College London Medical School, Royal Free Hospital, said, “Until recently, patients diagnosed with hereditary ATTR amyloidosis faced an uncertain future. A lack of treatments to halt or reverse the progression of disease resulted in a gradual and inescapable decline in their day-to-day functioning, placing a heavy burden not just on the patient themselves but on their partners and families, many of whom end up being full time carers. Patisiran has the potential not only to transform these patients’ lives but to change the way in which we think about and treat hereditary ATTR amyloidosis.”