Swedish Orphan Biovitrum AB announced that Canada’s Drug Agency (CDA) has issued a positive reimbursement recommendation with conditions for PrEMPAVELI (pegcetacoplan) for the treatment of adult and adolescent patients aged 12 years and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) to reduce proteinuria.
Empaveli is the first treatment authorized in Canada for C3G or primary IC-MPGN. The positive recommendation from CDA marks an important milestone in the reimbursement process for public drug plans across Canada. This recommendation represents an important step towards improving access for Canadians living with rare kidney diseases.
“CDA’s positive reimbursement recommendation for Empaveli is encouraging news for Canadians living with C3G and primary IC-MPGN, two rare and serious kidney diseases with significant unmet need,” said Bob McLay, Vice president and general manager, Sobi Canada. “This recommendation reflects the importance of improving access to innovative therapies for patients and families who have historically faced limited treatment options and a high risk of disease progression and kidney failure.”
The CDA recommendation was informed by data from the phase 3 VALIANT study, the largest single clinical trial conducted in patients with C3G and primary IC-MPGN. The study evaluated pegcetacoplan in 124 patients aged 12 years and older and demonstrated clinically meaningful reductions in proteinuria, stabilization of kidney function, and clearance of C3 deposits.
“Patients living with C3G and primary IC-MPGN face significant uncertainty due to the progressive nature of these diseases and the lack of disease modifying treatment options historically available” said Dr. Christoph Licht, head, division of nephrology, SickKids, The Hospital for Sick Children, Toronto. “A positive reimbursement recommendation for Empaveli is an important step toward helping eligible patients access a therapy specifically designed to target the underlying complement-mediated disease process.”
C3G and primary IC-MPGN are rare kidney diseases driven by uncontrolled activation of the complement system, leading to inflammation and kidney damage. Approximately 50 per cent of people living with these diseases experience kidney failure within five to 10 years of diagnosis, often requiring dialysis or kidney transplantation. Disease recurrence following transplant is also common.
Empaveli (pegcetacoplan) is a targeted C3 and C3b therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases.
Following CDA’s recommendation, reimbursement discussions with participating public drug plans will proceed through the pan-Canadian Pharmaceutical Alliance (pCPA).
C3G and primary IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure. Approximately 50% of people living with C3G or primary IC-MPGN suffer from kidney failure within five to 10 years of diagnosis, requiring a burdensome kidney transplant or dialysis therapy. Additionally, approximately 90% of patients who previously received a kidney transplant will experience disease recurrence. The diseases are estimated to affect 5,000 people in the United States and up to 8,000 in Europe.
The VALIANT phase 3 study (NCT05067127) was a randomized, placebo-controlled, double-blinded, multi-centre study that evaluated pegcetacoplan efficacy and safety in 124 patients who were 12 years of age and older with C3G or primary IC-MPGN. It is the largest single trial conducted in these populations and the only study to include paediatric and adult patients, with native and post-transplant kidneys. Study participants were randomized to receive pegcetacoplan or placebo twice weekly for 26 weeks. Following this 26-week randomized controlled period, patients were able to proceed to a 26-week open-label phase in which all patients received pegcetacoplan. The primary endpoint of the study was the log transformed ratio of urine protein-to-creatinine ratio (UPCR) at Week 26 compared to baseline.
Empaveli (pegcetacoplan) is a targeted C3 and C3b therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. It is the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older approved in the United States, European Union, Canada and other countries globally.
Empaveli is also approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) in Canada, the United States, European Union, and other countries globally. |