Emirates Drug Establishment Approves Itvisma Gene Therapy in UAE

The Emirates Drug Establishment approved Itvisma, a one dose gene therapy developed by Novartis, for adults and children aged two and above with spinal muscular atrophy in the UAE. The treatment replaces the defective SMN1 gene linked to the condition and is delivered as a single fixed dose to reduce reliance on long term regimens and improve quality of life. The authorisation follows clinical trials that showed sustained improvements in patients’ motor abilities. The UAE is the second country to clear Itvisma for use after approval by the US Food and Drug Administration in November. Spinal muscular atrophy affects motor neurons, causing muscle weakness that can make walking, swallowing and breathing difficult. Type 1 begins within the first six months of life; untreated children cannot sit unsupported and typically die before their second birthday, according to the US National Institutes of Health. Type 2 presents less severe symptoms and may prevent walking, while Types 3 and 4 affect older children and adults with milder but significant symptoms. Itvisma is described as a new version of Novartis’ gene therapy Zolgensma, which provides a working SMN gene to enable SMN protein production. Zolgensma is administered once and has been described as the world’s most expensive drug, with a single treatment reported at USD 2 million (AED 7.35 million). Other gene based therapies require repeated dosing at several hundred thousand dollars per year. Availability and pricing for Itvisma in the UAE have not been confirmed. In July, a two year old Syrian girl received AED 7 million gene therapy funded by Sheikh Mohammed bin Rashid at Al Jalila Children’s Hospital, which has completed more than 100 treatments for patients with spinal muscular atrophy, according to the facility.