OXLUMO® (lumasiran injection) Now Reimbursed in Canada For the Treatment of Primary Hyperoxaluria Type 1 (PH1) in Pediatric and Adult Patients

Cision Canada

3 days ago

OXLUMO has been shown to significantly reduce urinary oxalate, which drives the progression of PH1 Disease 1
MISSISSAUGA, ON, July 2, 2025 /CNW/ - Alnylam Canada ULC is pleased to announce that OXLUMO ® (lumasiran) is now funded across Canada through both public and private plans. OXLUMO is an RNA interference (RNAi) therapeutic administered via subcutaneous injection, indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in paediatric and adult patients. 1
OXLUMO is the first therapy for PH1 approved in Canada, and among the first medications to be included on the common list of new drugs for rare diseases, as part of the Government of Canada's National Strategy for Drugs for Rare Diseases. The common list was developed to help patients with rare diseases have access to treatments as early as possible.
PH1 is an ultra-rare and debilitating genetic disease of the liver characterized by oxalate overproduction. 2 Oxalate is an end-product of metabolism and high levels of it are toxic because it cannot be broken down by the human body. Oxalate overproduction results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones, nephrocalcinosis (renal deposition of calcium oxalate crystals), progression to kidney failure, and systemic organ dysfunction. 2
"Funding OXLUMO through the National Strategy for Drugs for Rare Diseases provides a new treatment option for people diagnosed with this debilitating, genetic disease – many of whom are infants and children," said Colleen Coxson, Country General Manager, Alnylam Canada ULC. "I want to congratulate the Canadian government for prioritizing access to therapies for those living with rare conditions, as there are often very limited options for patients."
There are several types of primary hyperoxaluria (PH), however, PH1 is the most common and the most severe form, accounting for 70 to 80 per cent of all PH cases. 3 PH1 affects approximately four individuals per million, with some regions – such as the Middle East and North Africa – having a higher genetic prevalence. 4 Symptom onset ranges from early infancy to sixty years of age, with the median age being four to six years. 4 The remainder of affected cases present in adulthood with 20 to 50 per cent presenting late stages of chronic kidney disease when diagnosed. 4
"This funding decision marks a major step forward in the management of hyperoxaluria type 1, offering hope to patients of all ages living with this ultra-rare genetic condition," said Dr. Vladimir Belostotsky, Division Head for Pediatric Nephrology at McMaster Children's Hospital. "The medication has been shown to effectively lower urine oxalate levels, reducing the burden that leads to severe clinical symptoms."
The positive recommendation for reimbursement was supported by results of the ILLUMINATE clinical studies, including ILLUMINATE-A: a randomized, double-blind, placebo-controlled clinical study in patients six years and older with PH1, ILLUMINATE-B: a single-arm clinical study in patients less than six years of age with PH1 1 and ILLUMINATE-C: a single-arm trial in patients of all ages with advanced PH1, including patients on dialysis. 5 The ILLUMINATE-A study showed that OXLUMO met its primary endpoint, evidenced by a 53 per cent mean reduction in urinary oxalate, and a 65 per cent mean reduction in urinary oxalate relative to baseline. 1 In ILLUMINATE-B, OXLUMO demonstrated a 72 per cent mean reduction in spot urinary oxalate:creatinine ratio from baseline to month six (averaged from months three to six), meeting its primary endpoint. 1 In ILLUMINATE-C, OXLUMO met its primary endpoint, demonstrating a 33% least squares (LS) mean reduction in plasma oxalate (POx) levels in patients not on dialysis (Cohort A) and a 42% LS mean reduction in POx levels in patients on hemodialysis (Cohort B) from baseline to month six. 6
About OXLUMO ® (lumasiran) 1
OXLUMO ® (lumasiran) is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in the liver to deplete the production of the glycolate oxidase (GO) enzyme. By silencing HAO1 and depleting the GO enzyme through RNA interference, OXLUMO in turn reduces the amount of oxalate that is produced. This process helps address the root cause of the rare, genetic disease since an overproduction of urinary and plasma oxalate levels is the underlying cause of PH1.
About Primary Hyperoxaluria Type 1 (PH1)
PH1 is an ultra-rare genetic disease that is characterized by oxalate overproduction in the liver, causing renal damage. Renal damage is caused by a combination of tubular toxicity from oxalate, calcium oxalate deposition in the kidneys, and urinary obstruction by calcium oxalate stones. PH1 is associated with a progressive decline in kidney function, which exacerbates the disease as the excess oxalate can no longer be effectively excreted, resulting in subsequent accumulation and deposition of oxalate in bones, eyes, skin, and heart, leading to severe illness and death. Management options to date have been limited to hyperhydration, crystallization inhibitors and, in a minority of patients with a specific genotype, pyridoxine (vitamin B6). These measures only delay the inevitable progression to kidney failure and the need for intensive dialysis as a bridge to a dual or sequential liver/kidney transplant. Other impacts of the disease include: infants often fail to thrive, meaning they are weak and not growing or developing at a normal rate. 7 Affected children frequently face developmental challenges, with social barriers and the requirement of accommodations to be made at school to meet their special medical needs. 6
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. 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 P 5 x25" 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.