Approval in US of first gene therapy for Duchenne muscular dystrophy – ELEVIDYS

July 18th, 2023

On 22 June, the US Food and Drug Administration (FDA) granted accelerated approval to Sarepta Therapeutics’ gene therapy SRP-9001 (also known as delandistrogene moxeparvovec-rokl) for Duchenne muscular dystrophy (DMD). Approval was granted for the treatment of children aged between four and five years, with Duchenne muscular dystrophy who can walk unassisted.

The FDA’s accelerated approval programme is designed to speed up the availability of treatments for serious conditions that demonstrate promising early results.

While accelerated approval allows patients in the US to access the therapy sooner, it also requires ongoing monitoring and additional studies to confirm its benefits. Continued approval is dependent upon results from these ongoing and future studies. This approach strikes a balance between addressing unmet medical needs and ensuring patient safety. The gene therapy has been marketed as ELEVIDYS and is not suitable for individuals with any deletion in exon 8 and/or exon 9 in the DMD gene.

The FDA’s decision will not directly affect people living with Duchenne muscular dystrophy outside of the United States. MDI are in contact with Roche – Sarepta’s partner who are responsible for distribution of this drug outside US– who will keep us updated about their plans for getting the treatment to people in Ireland.

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Categories: Research

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