UAE – The Ministry of Health and Prevention (MoHAP) has granted accelerated approval of Roche’s gene therapy for the treatment of ambulatory children with Duchenne Muscular Dystrophy (DMD).

The UAE health regulator has approved Roche’s delandistrogene moxeparvovec for treating ambulatory children aged 4 through 5 with Duchenne Muscular Dystrophy.

Roche’s delandistrogene moxeparvovec is the first and only gene therapy approved for use in DMD patients.

With the fast-tracked approval from the MoHAP, Roche will begin commercialization activities in the United Arab Emirates.

This approval is especially significant for pediatric patients affected by the life-threatening, rare, genetic, muscle-wasting disease.

It has the potential to shed light on the urgent need for new therapies targeting the underlying cause of the disease.

In a press release, Dr. Mohamed ElShaarawy, General Manager of UAE Roche Pharmaceuticals, said: “Living by our purpose of ’doing now what patients need next’, we are working with urgency to support our partners in ensuring the UAE healthcare system is ready to bring this promising gene therapy to people with Duchenne and their families this year.”

Although life expectancy has increased due to improvements in the standard of care, DMD still leads to early death often before or during patients’ 30s due to respiratory impairment and cardiomyopathy.

It is worth highlighting that current treatments for Duchenne Muscular Dystrophy have limited effectiveness.

Hence, the MoHAP registration of delandistrogene moxeparvovec is the first regulatory approval outside the United States Food and Drug Administration (US FDA).

The FDA-approved gene therapy is designed to deliver a gene that makes a shortened functional form of the dystrophin protein to muscle cells with the aim of improving muscle function via a one-time administration.

It marks a step forward in illuminating a path toward enhanced therapeutic outcomes and a potential extension of survival since DMD has no known cure.

For this reason, the approval of delandistrogene moxeparvovec underpins the Ministry of Health and Prevention’s smooth and clear accelerated registration process.

On his part, Dr. Mohamed ElShaarawy stated: “Clinical data to date suggest that delandistrogene moxeparvovec has the potential to transform the lives of patients and families living with Duchenne Muscular Dystrophy.”

Roche, in collaboration with the MoHAP, will work closely to enhance access to the first-of-its-kind gene therapy for the treatment of DMD in pediatric patients in the UAE.

Roche plans to share the results of the pivotal ongoing Phase 3 EMBARK study by the end of 2023 as part of its ongoing partnership with health authorities to help patients live longer and better lives.

It could pave the way for drug discovery and the development of gene therapies that are not only suitable but available to the majority of people living with DMD.

From its UAE base, the approval of delandistrogene moxeparvovec underlines Roche’s commitment to pioneering Research & Development and innovation in the healthcare industry.

Roche’s delandistrogene moxeparvovec is expected to be available in all MoHAP-approved hospitals before the end of 2023.

This regulatory milestone means that DMD patients in the United Arab Emirates may be the first to receive the treatment outside the United States.

Astoundingly, the regulatory approval stands as a reflection of the UAE Government’s commitment to healthcare innovation and patient access to the most recent advancements in patient care.

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