Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the European Commission (EC) has granted marketing authorization 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.
"Today's 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," said Theresa Heggie, Head of Europe, Alnylam Pharmaceuticals. "This is the start of a new chapter in the treatment of this rare, rapidly progressive, fatal disease. We want to express our profound gratitude to the patients who participated in the patisiran clinical trials and their families and caregivers who supported them, as well as the investigators and study staff, without whom this important milestone would not have been achieved. We look forward to applying our innovative therapeutic approach to help patients with other rare diseases in the future."
"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," said Professor Philip N. Hawkins, Ph.D, FRCP, FRCPath, FMedSci, National Amyloidosis Centre, Division of Medicine, and University College London Medical School, Royal Free Hospital, UK. "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."
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 EC decision was based on the evaluation of the effects of patisiran in hATTR amyloidosis patients with polyneuropathy and its safety profile as demonstrated in the APOLLO Phase 3 study. The Summary of Product Characteristics (SmPC) includes data from APOLLO on primary and secondary endpoints, as well as exploratory cardiac endpoints. The European Medicines Agency reviewed patisiran under the accelerated assessment procedure that is granted to medicines judged to be of major interest for public health and therapeutic innovation.
Source: http://investors.alnylam.com/news-releases/news-release-details/alnylam-receives-approval-onpattrotm-patisiran-europe
CRISPR-based therapy shown to be safe, effective for people with transthyretin amyloidosis