Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store. Adverse events should also be reported to Roche Products Ltd. Please contact Roche Drug Safety Centre by emailing welwyn.uk_dsc@roche.com or calling +44 (0)1707 367554.

Evrysdi

Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies5

Evrysdi is now recommended by NICE as an option for treating 5q spinal muscular atrophy (SMA) in people 2 months and older with a clinical diagnosis of SMA types 1, 2 or 3 or with pre-symptomatic SMA and 1 to 4 SMN2 copies. It is recommended only if the conditions of the managed access agreement (MAA) are followed.8

▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. Adverse events should be reported. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store. Adverse events should also be reported to Roche Products Ltd. Please contact Roche Drug Safety Centre by emailing welwyn.uk_dsc@roche.com or calling +44 (0)1707 367554.

For full details, please refer to the Evrysdi® Summary of Product Characteristics (GB, NI)

Abbreviations

CNS, central nervous system; GB, Great Britain; mRNA, messenger ribonucleic acid; NI, Northern Ireland; SMA, spinal muscular atrophy; SMN2, survival motor neuron 2.

References

  1. Chiriboga CA, Bruno C, Duong T, et al. JEWELFISH: safety and pharmacodynamic data in non-naïve patients with spinal muscular atrophy receiving treatment with risdiplam. Presentation at: 2020 Virtual Cure SMA Conference; June 8-12, 2020; Virtual.
  2. Darras BT, Masson R, Mazurkiewicz-Bełdzińska M, et al. Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls. N Engl J Med. 2021
  3. Day JW, Annoussamy M, Baranello G, et al. SUNFISH Part 1: 24-month safety and exploratory outcomes of risdiplam (RG7916) treatment in patients with Type 2 or 3 spinal muscular atrophy (SMA). Presentation at: 2020 Virtual Cure SMA Conference; June 8-12, 2020; Virtual.
  4. Mercuri E, Barisic N, Boespflug-Tanguy O, et al. SUNFISH Part 2: efficacy and safety of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). Presentation at: American Academy of Neurology Annual Meeting; April 25-May 1, 2020; Virtual.
  5. Evrysdi® Summary of Product Characteristics (GB, NI)
  6. Baranello G, Darras BT, Day JW, et al. Risdiplam in Type 1 Spinal Muscular Atrophy. N Engl J Med. 2021.
  7. Mercuri E, Baranello G, Masson R, et al. SUNFISH Part 1: Safety, tolerability, PK/PD and exploratory efficacy data in patients with Type 2 or 3 spinal muscular atrophy (SMA) treated with risdiplam (RG7916). Presentation at: The 24th International Annual Congress of the World Muscle Society; October 1-5, 2019; Copenhagen, Denmark.
  8. NICE Technology appraisal guidance [TA755]. Available at https://www.nice.org.uk/guidance/ta755/resources/risdiplam-for-treating-spinal-muscular-atrophy-pdf-82611377117125 (accessed Dec 2021)

M-GB-00005712

Date of preparation: December 2021