UAE approves Itvisma gene therapy: A leap in medical innovation

The Emirates Drug Establishment (EDE) has formally authorised Itvisma (onasemnogene abeparvovec), marking a new chapter for gene therapy treatment of spinal muscular atrophy (SMA) for both adults and children from the age of two.

This approval places the UAE immediately behind a single other nation globally, underscoring a drive to accelerate patient availability of ground-breaking treatments. The move further consolidates the country’s prominence as a pharmaceutical development centre within the region.

Functioning at the root cause, Itvisma substitutes the faulty gene behind SMA. This strategy aims to enhance patients’ motor skills and lessen their dependency on protracted therapies.

Dr Fatima Al Kaabi, Director General of EDE, offered a direct perspective: "Granting the regulatory approval for Itvisma is a significant step demonstrating the UAE's commitment to providing patients with advanced genetic therapies, particularly those with rare diseases like spinal muscular atrophy.

“This decision showcases the efficiency of the country's health system and its capability to evaluate and approve innovative drugs according to the highest scientific standards within effective timeframes. We are committed to ensuring patients receive rapid access to cutting-edge treatment options that improve their quality of life," Al Kaabi said.