Ultra Orphan Assessment

The Scottish Medicines Consortium (SMC) has completed its initial assessment of the evidence for this medicine using the ultra-orphan framework.

voretigene neparvovec (Luxturna)

Indication under review: For the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

Key points:

  • Inherited retinal dystrophy due to RPE65 mutations leads to progressive blindness and there are no available treatments.
  • In a clinical trial, voretigene neparvovec improved functional vision at one year compared with no treatment, measured by a multi-luminance mobility test.
  • Uncontrolled follow-up data support that treatment effect is maintained at four years. Whilst it is biologically plausible that the treatment effect will continue, it is not known if effectiveness is sustained in the long term.
  • A retrospectively validated visual function questionnaire showed improved activities of daily living, but there is uncertainty over how this relates to actual quality of life.
  • A model-based economic evaluation projected a substantial gain in quality-adjusted life years compared to best supportive care. However, there were uncertainties particularly surrounding utility values and also how long the treatment effect lasts.
  • Despite a Patient Access Scheme (PAS), the treatment’s cost in relation to its health benefits remains high.

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From July 2020 voretigene neparvovec can be prescribed within the ultra-orphan pathway while further evidence on its effectiveness is generated.  After 3 years the company will provide an updated submission for reassessment to allow a decision on its routine use in NHSScotland.

Medicine details

Medicine name:
voretigene neparvovec (Luxturna)
SMC ID:
SMC2228
Indication:

For the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

Pharmaceutical company
Novartis Pharmaceuticals UK Ltd
BNF chapter
Eye
Submission type
Ultra-orphan initial assessment
Date Published
10 February 2020
Additional notes

A new approach for assessing medicines for extremely rare conditions started in October 2018. Further information is available on our ultra-orphan webpage and in the Scottish Government’s guidance.