Gene-based medicine for treatment of inherited blindness in UAE

Dubai - The approval of the UAE for this treatment is the third globally at a health authorities level.

Dr. Mohamed Salim Al Olama, Under-Secretary of the Ministry of Health and Prevention, MoHAP, has revealed the registration of "Luxturna", an innovative gene-based medicine for the treatment of blindness caused by inherited retinal disease.
The medicine's registration comes as part of MoHAP's innovative mechanism for its accelerators to evaluate and approve the world's breakthrough drugs in accordance with the 'Fast Track/Accelerate Process' system.
Al Olama, who is also the Vice Chairman of the Higher Committee for Drug Pricing and Registration, said that the the registration of Luxturna comes in line with the Ministerial Decree No. 28 of 2018 which outlines the mechanism for registering breakthrough and rare medicines.
"It's also part of the UAE's ongoing support for the global initiative, 'Vision 2020: The Right to Sight', launched by the World Health Organisation, WHO, to eliminate avoidable blindness by the year 2020," he added.
"We are witnessing a significant turning point with the expectation of experts that gene-based therapies will be expanded to include other incurable diseases. For that reason, the UAE has approved this new treatment as it paves the way towards future prospects for gene-based therapies which would help patients recover from a lot of complex diseases," Al Olama noted.
The approval of the UAE for this treatment is the third globally at a health authorities level. This comes in line with MoHAP's strategy to build quality and safety for therapeutic, healthcare and pharmaceutical systems according to international standards, as well as the UAE Vision 2021.
For his part, Dr. Mohamed Ezz El Din, Country Manager, Novartis, said, "We received some good news that MoHAP completed the registration process of Luxturna which is the first gene-based therapy in the UAE. We are looking forward to further strengthening our presence in the region to ensure that patients could get this global therapy that may change their lives.
"For those born with a mutation in both copies of the RPE65 gene, nearly 60 percent of patients have severe forms of the disease, with severe visual impairment occurring shortly after birth. RPE65 inherited retinal disease is a rare one, as mutations in both copies of the RPE65 gene affect approximately one in 200,000 people, however, mutations in both copies of the RPE65 gene can lead to blindness. Hence the importance of this innovative medicine, Luxturna, that restores sight and improves vision," added Dr. Ezz El Din.

What is gene therapy?