The provincial government is covering costs for risdiplam, or Evrysdi, the medication used to treat a severe and rare neuromuscular disease.
Spinal muscular atrophy (SMA) affects spinal cord nerve cells and impacts the muscles used for breathing, eating, crawling and walking. People living with SMA are not able to make a specific kind of protein and without it, the cells that control muscles die.
Symptoms can first appear before birth, while others start in the first year of life. The most common symptoms include progressive muscle weakness and poor muscle tone and control, which impair motor development and mobility. Patients may never walk or may gradually lose the ability to walk, sit or move.
SMA is inherited and a leading genetic cause of death in infants and toddlers. It is estimated that there are 23 pediatric patients and 10 adults eligible for risdiplam in B.C.
The annual cost of risdiplam at list price ranges from $93,456 to $354,000, depending on the age and weight of the patient. British Columbia participated in pan-Canadian Pharmaceutical Alliance negotiations with the manufacturer to finalize its funding decision.
The Ministry of Health will provide funding for risdiplam through the BC Expensive Drugs for Rare Diseases (EDRD) process for eligible SMA patients. EDRDs are considered non-benefits, but some drugs and patients may be eligible for coverage on a case-by-case, last-resort basis. Patients will need to discuss their eligibility with their health-care provider.
Risdiplam is an oral drug with clinical evidence that supports improvement in motor function and survival in SMA patients.
The Ministry of Health also funds other treatments for SMA, including nusinersen, or Spinraza, and onasemnogene abeparvovec, or Zolgensma.