Monograph

The Ministry of Health’s Drug Advisory Committee has recommended:

• Risdiplam 0.75 mg/mL powder for oral solution for treating:
  • Symptomatic Type 1, 2 or 3 spinal muscular atrophy (SMA) in patients who are/were 18 years of age or under at the time of initial treatment with risdiplam or nusinersen;
  • Symptomatic Type 1, 2 or 3 SMA in patients aged 19 years or above who had not initiated treatment with risdiplam or nusinersen prior to 19 years of age despite onset of signs/symptoms of SMA;
  • Pre-symptomatic SMA in patients who are/were under 3 years of age at the time of initial treatment with risdiplam or nusinersen; and
  • SMA in patients who have experienced a regression in a developmental state despite treatment with gene therapy.

Funding status