Benzinga | Oct 18, 2021 08:13AM EDT

Alnylam Pharma Reports Received Positive Reimbursement Recommendation From Canadian Agencies For Drugs And Technology In Health For Use Of GIVLAARI As Treatment Of Acute Hepatic Prophyria

Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) is pleased to announce that GIVLAARI(r) (givosiran) has now received a positive recommendation for reimbursement from the Canadian Agency for Drugs and Technologies in Health (CADTH). GIVLAARI(r) is approved by Health Canada for subcutaneous use for the treatment of acute hepatic porphyria (AHP) in adults.1

"The Health Canada market authorization of GIVLAARI provides new hope for those living with AHP. For the first time, all patients have a treatment option specifically targeted for this rare and serious disease," says Colleen Coxson, Country General Manager, Alnylam Pharmaceuticals. "We are proud of what's been accomplished and are working towards full national reimbursement so that patients across Canada can have access to a treatment that will improve their overall health and quality of life."

AHP is an ultra-rare condition in which patients can experience debilitating attacks of severe abdominal pain, vomiting and seizures. It can be life-threatening due to the possibility of paralysis and respiratory arrest during attacks. Many patients also experience chronic symptoms, including severe pain, which continues to be present between attacks, linked to increased rates of liver cancer, kidney failure and hypertension.2, 3

"Patients suffering from this very rare, genetic disease often require urgent healthcare assistance and hospitalization, due to serious symptoms such as porphyria attacks which can involve severe abdominal pain, vomiting, breathing problems, seizures, and can lead to long term complications like chronic pain, chronic kidney failure and liver damage," says Dr. Colin Van Zoost MD, FRCPC, Assistant Professor of Medicine Dalhousie University. "Not knowing when the next attack will can cause severe anxiety for many patients, making engagement in day-to-day life, work and social situations difficult. GIVLAARI(r) gives us a treatment option that can help patients avoid ever having another attack, allowing them to re-engage with society and live as close to a normal life as possible."

The positive recommendation for reimbursement was supported by the results of the ENVISION Phase 3 Study, a randomized, double-blind, placebo-controlled, global, multicenter study to evaluate the efficacy and safety of GIVLAARI(r) in patients with a documented diagnosis of acute hepatic porphyria (AHP).4 The primary endpoint was reduction relative to placebo in the annualized rate of composite porphyria attacks, defined as those requiring hospitalization, urgent healthcare visit, or intravenous hemin administration at home, in patients with acute intermittent porphyria (AIP, the most common subtype of AHP) over six months.5 Full details on the recommendation is available on the CADTH website.6