GIVLAARI® (givosiran injection) Now Widely Reimbursed in Canada For the Treatment of Acute Hepatic Porphyria (AHP) in Adults

GIVLAARI is the first and only therapy in Canada proven to prevent AHP attacks, reduce chronic pain and improve quality of life¹

MISSISSAUGA, ON, Dec. 23, 2024 /CNW/ - Alnylam Canada ULC is pleased to announce that GIVLAARI^® (givosiran injection) is now funded across Canada through both public and private insurance plans for patients living with acute hepatic porphyria (AHP), including those diagnosed with acute intermittent porphyria (AIP) - the most common type of AHP.

GIVLAARI is approved by Health Canada for the treatment of acute hepatic porphyria (AHP) in adults.¹ It is the first and only therapy in Canada that is proven to prevent AHP attacks, reduce chronic pain and improve quality of life for this ultra-rare, genetic condition.¹

"We have collaborated with governments and stakeholders to ensure those living with AHP have funded access to GIVLAARI," said Colleen Coxson, Country General Manager, Alnylam Pharmaceuticals. "I want to congratulate all the Canadian jurisdictions on this important milestone, as providing coverage for a treatment option specifically targeted for this ultra-rare and serious disease can be transformative for patients."

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

"Historically, individuals suffering from AHP attacks only had access to treatment for symptom management, so they lived life under the constant threat of a debilitating attack that could send them to the hospital," said Michelle Capon, President, Canadian Association for Porphyria. "The seriousness of these attacks can be life threatening, but at minimum, their unpredictability make it challenging to participate in daily activities, like social engagements or work, leading to significant anxiety and impacts on quality of life. GIVLAARI offers a therapeutic option that can prevent future attacks, increasing their ability to live a normal life."

The positive recommendations for reimbursement were 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 in patients with a documented diagnosis of acute hepatic porphyria (AHP).¹ 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 AIP over six months.¹ 

About GIVLAARI^® (givosiran injection)¹
GIVLAARI (givosiran) is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of adults with acute hepatic porphyria (AHP). In the pivotal study, givosiran was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam's first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare professional. GIVLAARI works by specifically reducing elevated levels of aminolevulinic acid synthase 1 (ALAS1) messenger RNA (mRNA), leading to reduction of toxins associated with attacks and other disease manifestations of AHP.

About Acute Hepatic Porphyria^2,3
Acute hepatic porphyria (AHP) refers to a family of ultra-rare, genetic diseases characterized by debilitating, potentially life-threatening attacks and, for some patients, chronic manifestations that negatively impact daily functioning and quality of life. AHP is comprised of four subtypes: acute intermittent porphyria (AIP), hereditary coproporphyria (HCP), variegate porphyria (VP), and ALA dehydratase-deficiency porphyria (ADP). Each type of AHP results from a genetic defect leading to a lack of certain enzymes needed to produce heme in the liver, which leads to an accumulation of porphyrins in the body to toxic amounts. AHP disproportionately impacts women of working and childbearing age, and symptoms of the disease vary widely. Severe, unexplained abdominal pain is the most common symptom, which can be accompanied by limb, back, or chest pain, nausea, vomiting, confusion, anxiety, seizures, weak limbs, constipation, diarrhea, or dark or reddish urine. AHP is life-threatening due to the possibility of paralysis and respiratory arrest during attacks. The nonspecific nature of AHP signs and symptoms can often lead to misdiagnoses of other more common conditions such as gynecological disorders, viral gastroenteritis, irritable bowel syndrome (IBS), and appendicitis. Consequently, on a global perspective, patients with AHP can wait up to 15 years for a confirmed diagnosis, with the risk of addiction problems. In addition, long-term complications and comorbidities of AHP can include hypertension, chronic kidney disease or liver disease, including hepatocellular carcinoma.

About RNAi 
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today.⁵ Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine.⁶ By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors that encode for disease-causing or disease pathway proteins – thus preventing them from being made.⁵ This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its "Alnylam P⁵x25" strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. Alnylam Canada is headquartered in Mississauga, Ontario with established operations since June 2018.

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¹ GIVLAARI Product Monograph. October 8, 2020

² Puy, The Lancet 2010

³ Anderson, Ann. Intern. Med. 2005.

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06, 2024

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