A drug for the treatment of Leber hereditary neuropathy (LHON) was approved for reimbursement in Ireland in May 2019. The drug, which is called Idebenone (commercial name Raxone) is now available for a subset of people living with LHON who meet certain criteria. One of the main eligibility criteria is “onset of visual loss in the most recently affected eye of five years or less”. This means that if someone began to lose vision in their second eye more than five years ago, they are not eligible to receive the drug. You can read more about the reimbursement of Idebenone here.
In November 2018 the European Commission approved the gene therapy Luxturna (voretigene neparvovec-rzyl). Luxturna has been approved for treatment of children and adults with vision loss caused by mutations in both copies of the RPE65 gene and who have sufficient viable retinal cells. Conditions caused by this type of mutation include very rare forms of Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP). This approval means that Luxturna can now be marketed in all European Union and European Economic Area countries. The next stage involves a separate application for approval at a national level in each country. The pharmaceutical company, Novartis, will now make applications to national reimbursement agencies in a number of countries, with other countries to follow. The gene therapy is expected to be made available to the first patients in these countries in 2019/2020. You can read more about the approval of Luxturna here.
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