Risdiplam, originally developed under contract with the SMA Foundation by PTC Therapeutics and licensed to Roche for sale under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy and the first oral medication approved to treat this disease.
The following are links to KEI’s work on risdiplam.
- July 27, 2021. Access to SMA therapy: Iman’s Story.
- July 8, 2022. KEI letter to Roche requesting voluntary license to patents on risdiplam, a treatment for Spinal Muscular Atrophy.
- November 15, 2022. KEI asks WHO to include risdiplam, treatment for Spinal Muscular Atrophy, on the Essential Medicines List (EML).
- March 1, 2024. Request for Amendment to add Risdiplam for Spinal Muscular Atrophy (SMA) to Schedule 1 of the Canadian Patent Act.
- November 1, 2024: Proposal for Addition Of Risdiplam to WHO Model List Of Essential Medicines, for the treatment of Spinal Muscular Atrophy (SMA).
- April 7, 2025. Research Note on the Development Timeline of Risdiplam.
- April 23, 2025. The cost of trials used to support the initial FDA approval of risdiplam.
- October 10, 2025. Delhi High Court Rejects Roche’s Appeal, Paving Way for Affordable Generic Risdiplam.