Reimbursement Approval of Evrysdi® for the Treatment of Spinal Muscular Atrophy

September 6th, 2023

On 1 September the HSE announced that Risdiplam (Evrysdi®) has been approved for reimbursement in Ireland. This makes Risdiplam the third SMA medicine available in Ireland, alongside Zolgensma and Spinraza.

Developed by Roche Pharmaceutical, Risdiplam is the only non-invasive SMA therapy, administered daily at home in liquid form by mouth or by feeding tube, making it more accessible and less invasive than other treatments.

MDI welcomes this announcement and recognises it is a significant milestone in the fight for access to life changing therapies. It is especially good news for younger people in the community and their families, expanding the treatment options available.

Risdiplam has been approved by the European Medicines Agency (EMA) for all ages groups with SMA Type 1, Type 2, or Type 3 with four or fewer copies of the SMN2 gene. This includes a recent approval by the EMA to extend inclusion to infants from birth to two months.

It is therefore disappointing that the HSE has only approved access for people in Ireland who are aged between two months and under 18 years of age.

As a result, there continues to be no treatment options for people in Ireland with SMA over 18 years of age. ZOLGENSMA is a gene therapy used to treat children less than 2 years old with SMA and Spinraza is also capped to people under 18 years of age in Ireland, even though it is available for adults in other European countries.

Individuals living with SMA over 18 years of age shouldn’t be discriminated because of their age. Access to these lifesaving therapies should be available to everyone, when there is evidence to show how important and effective, they are.

MDI call for the age restrictions to be lifted on access to Risdiplam in Ireland as SMA doesn’t go away when you become an adult, it continues to progress, and we want to stop or slow this down. No one should be left behind, and MDI will keep being a voice for this.

About Risdiplam

Risdiplam sold under the brand name Evrysdi™ is the third treatment approved to treat SMA in Europe in adults and children two months and older. It received European Union authorisation in 2021 for the treatment of SMA Type 1, Type 2, or Type 3 patients with four or fewer copies of the SMN2 gene.

It is an oral medicine that is designed to increase SMN protein levels in the central nervous system (CNS) and throughout the body. This SMN-enhancing therapy is a small molecule that targets the SMN2 gene and helps to increase the amount of stable SMN protein made by this gene. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and other functions such as swallowing, speaking, breathing and movement.

The HSE has included risdiplam as part of the Medicines Management Programme and has Managed Access Protocols for the product. Please, see the link below for the managed access protocol for risdiplam.

Share Now:

Categories: Information

1. Muscular Dystrophies

  • Becker muscular dystrophy
  • Duchenne muscular dystrophy
  • Manifesting carrier of Duchenne
  • Congenital muscular dystrophy
  •     •  General
  •     •  MDC1A (merosin-deficient congenital muscular dystrophy)
  •     •  Rigid spine syndrome (RSS)
  •     •  Ullrich congenital muscular dystrophies
  •     •  Bethlem myopathy
  • Emery-Dreifuss muscular dystrophy
  • Facioscapulohumeral muscular dystrophy
  • Limb-girdle types of muscular dystrophy (LGMD)
  •     •  General
  •     •  LGMD 1B (also known as Laminopathy)
  •     •  LGMD 1C (also known as Caveolinopathy)
  •     •  LGMD 2A (also known as Calpainopathy)
  •     •  LGMD 2B (also known as Dysferlinopathy)
  •     •  LGMD 2I
  • Ocular myopathies including ocularopharangeal muscular dystrophy

2. Myotonic Disorders

  • Congenital Myotonic Dystrophy
  • Myotonia
  • Myotonic Dystrophy

3. Congenital Myopathies

  • Central Core Myopathy
  • Congenital Fibre-type Disproportion Myopathy
  • Minicore (Multicore) myopathy
  • Myotubular or Centronuclear myopathy
  • Nemaline myopathy

4. Mitochondrial Myopathies

  • Mitochondrial Myopathies

5. Metabolic Disorders

  • Metabolic disorders (general)
  • McArdle’s Disease
  • Pompe’s Disease

6. Periodic Paralyses

  • Periodic Paralyses

7. Autoimmune Myositis

  • Polymyositis, Dermatomyositis and Sarcoid myopathy
  • Juvenile dermatomyositis
  • Inclusion body myositis

8. Spinal Muscular Atrophies

  • Severe (Type I)
  • Intermediate (Type II)
  • Mild (Type III)
  • Adult spinal muscular atrophy

9. Hereditary Motor and Sensory Neuropathies

  • (Also known as Charcot-Marie-Tooth or Peroneal muscular atrophy)

10. Disorders of the Neuromuscular Junction

  • Congenital myasthenic syndromes
  • Myasthenia Gravis

11. Friedreich’s Ataxia

  • Friedreich’s Ataxia

12. Other (Please Specify)

13. Unspecified