New Treatments Available for Patients with Spinal Muscular Atrophy

Released on March 17, 2022

Two new medications for treating Spinal Muscular Atrophy (SMA) are now covered in Saskatchewan. Zolgensma and Evrysdi are covered through the Drug Plan as eligible benefits for patients who meet certain medical criteria.

"Early diagnosis and treatment are crucial for better outcomes for patients with SMA," Health Minister Paul Merriman said. "The availability in Saskatchewan of newborn screening along with these cutting-edge medications will greatly improve the prognosis and quality of life for patients born with SMA and their families."

Zolgensma (onasemnogene abeparvovec) is a high-cost gene therapy for the treatment of pediatric patients that targets the underlying genetic defect in SMA by delivering a fully functional copy of the gene into the patient's appropriate cells. It is administered as an intravenous infusion, as a single lifetime dose. Evrysdi (risdiplam) is the first oral drug available to treat SMA. It works by increasing the protein that is deficient in SMA patients.

Spinraza, another drug for the treatment of SMA, is also covered under the Saskatchewan Formulary for patients who meet eligibility criteria.

Spinal Muscular Atrophy is a rare neuromuscular disorder caused by a genetic defect that can lead to impaired mobility, swallowing and breathing, and may result in early death. SMA is the number one genetic cause of infant mortality and it occurs in one in 10,000 babies born. Prior to availability of drug therapies, SMA had been managed by supportive care interventions that did not improve survival, motor development or progression of symptoms.

Treatments for SMA are more likely to be effective if introduced early in the course of the disease. Saskatchewan recently expanded its Universal Newborn Screening Program to include SMA. The program enables families to receive much earlier diagnosis and treatment.

Patients or caregivers who think they may benefit from these new medications are encouraged to speak with their physician regarding treatment options.

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