Nusinersen and risdiplam for treating spinal muscular atrophy

Published on 04 Jun 2024
Last Updated on 04 Jun 2024

Guidance Recommendations

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

Risdiplam 0.75 mg/mL powder for oral solution is recommended for inclusion on the Medication Assistance Fund (MAF) for the abovementioned indications from 1 August 2024.

Risdiplam should be used in line with the additional clinical criteria listed in the Annex.

MAF assistance does not apply to nusinersen for treating SMA.

Nusinersen and risdiplam for treating spinal muscular atrophy (Published 4 Jun 24) [PDF, 193 KB]

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