-- GIVLAARI reduces Chronic Pain, Improves Quality of Life and is the First and Only Therapy Proven to Prevent AHP Attacks --
"The
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.
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® 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
About GIVLAARI® (givosiran)
GIVLAARI® 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
About Acute Hepatic Porphyria
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. 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.
About
______________________________ |
1 GIVLAARI Product Monograph. |
2 Puy, |
3 Anderson, Ann. Intern. Med. 2005. |
4 GIVLAARI Product Monograph. |
5 GIVLAARI Product Monograph. |
6 https://cadth.ca/givosiran |
SOURCE
© Canada Newswire, source