SMA Ireland lodges Patient Submission Document with NCPE regarding Risdiplam

SMA Ireland, the patient representative group for people affected by Spinal Muscular Atrophy (SMA) this week (23.11.21) lodged a formal Patient Submission Document with the HSE’s National Centre for Pharmacoeconomics as part of the ongoing Health Technical Assessment concerning Roche Pharmaceutical’s SMA medicine, Risdiplam (Evrysdi).

Risdiplam is the first drug available as an oral medication for the treatment of SMA. It has undergone extensive clinical trials and has proven efficacy for patients up to 60 years of age. Risdiplam was approved by the European Medicines Agency in March 2021.

Currently children in Ireland who do not qualify for the SMA medicine, Zolgensma, have the option of treatment with Spinraza. This treatment is life changing but requires regular spinal injections. Children with SMA typically suffer from weaker breathing and skeletal issues. The Spinraza spinal injection consequently can prove problematic for some patients regarding general anaesthetics and finding a suitable site for infusion.

The HSE is now considering reimbursement for the drug, Risdiplam. Unlike the previously approved SMA medicines, Spinraza and Zolgensma, Risdiplam is a daily oral medicine that can be taken at home greatly reducing the burden for both child and caregiver.

There is an unusual situation in Ireland that arbitrarily excludes a portion of SMA individuals from treatment for their condition. Currently, Zolgensma is only available to infants under 24 months. To receive Spinraza, the patient must have been under 18 years old at the time the drug was approved in 2019. Therefore, some SMA patients, now in their 20s, continue to receive Spinraza, whereas patients who were in their 20s in 2019 remain excluded from treatment. SMA Ireland strongly believes that having such an arbitrary age cap for access to treatment is discriminatory and unethical.

Thirty adult Irish patients have never received any of the recently developed life changing treatments for SMA. Risdiplam offers the HSE with a viable medicine for all SMA age groups. SMA Ireland calls on the NCPE to approve reimbursement for Risdiplam without a specific age limit, thus removing the unethical status quo and offering an effective treatment to a small number of patients that have been excluded from access to a life-changing medicine.

Commenting, Director of SMA Ireland, Jonathan O’Grady said: “As an oral medication with proven efficacy across all age groups, Risdiplam marks a significant step forward in the treatment of Spinal Muscular Atrophy. It offers doctors the possibility of an alternative SMA medicine without the complications of a spinal injection.

Though their muscles may be weak, people with SMA are tenacious, optimistic and full of spirit. They love and are loved. This medicine offers everyone with SMA the chance to arrest the worst effects of the disease and an opportunity to live a fuller, happier, higher quality life. No one should be denied this medicine, least of all because of an arbitrary age threshold.

SMA Ireland calls on the National Centre for Pharmacoeconomics to make this important medicine available in Ireland, including for the small number of adults who have until now been excluded from any treatment.”