Ministry of Health and Prevention Registers an Emerging Gene Therapy Luxturna for Treatment of Inherited Blindness

Published Monday, 24 June 2019
Ministry of Health and Prevention Registers an Emerging Gene Therapy Luxturna for Treatment of Inherited Blindness

His Excellency Dr. Mohamed Salim Al Olama, the Undersecretary of the Ministry of Health and Prevention (MoHAP) and Vice Chairman of the Higher Committee for Drug Pricing and Registration, has revealed the registration of “Luxturna”, an innovative gene-based medicine for the treatment of blindness caused by inherited retinal disease.

The medicine 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 stated that the registration of this innovative drug comes in line with the Ministerial Decree No. 28/2018 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 Organization (WHO) to eliminate avoidable blindness by the year 2020,” he added.

New Hope for Patients

“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 global one at 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.

Accelerating Registration Path Offers Additional Solutions for Patients

Al Olama praised the keenness of global pharmaceutical companies to provide the UAE with the files of innovative medicines at the same time of submitting them to the FDA and EMEA with a view to obtaining the international accreditation. These files include a review of clinical studies, bioequivalence and stability studies.

He pointed to the leadership role of the UAE in protecting the intellectual property of innovative and rare medicine and promoting innovation in the pharmaceutical field as a key factor to discover viable solutions for complex and incurable diseases. This comes as part of MoHAP’s strategy aiming to provide a vital legislative and governance framework, as well as distinctive regulatory and supervisory services for the healthcare sector in accordance with the UAE Vision 2021. 

“Thanks to the directives of our wise leadership and our flexible legislative environment, the UAE has consolidated its position in the global pharmaceutical market. This matter encouraged the global pharmaceutical companies to take the UAE as a marketing hub for their new innovative products. This will offer additional solutions for patients in the country and the region, besides fostering the medical tourism in the UAE,” said Al Olama.

Sustainable Medications to Replace Conventional Remedies 

He further shed light on MoHAP’s strategy based on sustainable solutions. It aims to provide one-time treatment for incurable diseases. This will help health care providers to save substantial costs over the long term, compared to conventional remedies which patients need for a long time.

Ongoing Partnership with Novartis

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% 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 1 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.

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