The UAE has approved the use of a gene therapy to advance the treatment of adults and children with spinal muscular atrophy.
Itvisma, developed by Swiss pharmaceutical company Novartis, works to replace the defective SMN1 gene responsible for the debilitating disease. Delivered in a one-time fixed dose, it aims to improve quality of life and reduce reliance on long-term treatments.
The Emirates Drug Establishment granted regulatory approval following clinical trials that demonstrated sustained improvements in patients' motor abilities.
The UAE is only the second country to authorise Itvisma's use – for adults and children aged two and above – after it was approved by the US Food and Drug Administration in November.
What is spinal muscular atrophy?
SMA affects motor neurons, which are nerve cells that send impulses to the muscles, and causes limb weakness.
Some people with the condition are unable or find it difficult to walk. SMA may also make swallowing and breathing difficult.
Children who have the most severe form, type 1, and who are not treated cannot sit unsupported and typically die before their second birthday, according to the US National Institutes of Health. Symptoms of type 1 begin to manifest within a baby's first six months.
Type 2 causes less severe symptoms but may prevent a child from walking.
Two other forms, type 3 and type 4, affect older children and adults, with milder but still significant symptoms.
Harnessing medical advances
“Granting the regulatory approval for Itvisma is a significant step demonstrating the UAE's commitment to providing patients with the latest advanced genetic therapies, particularly those with rare genetic diseases like spinal muscular atrophy,” said Dr Fatima Al Kaabi, director-general of the Emirates Drug Establishment,
“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 time frames. We are committed to ensuring patients receive rapid access to cutting-edge treatment options that improve their quality of life.”
Mohamed Ezz Eldin, head of GCC countries at Novartis, said authorisation of Itvisma in the UAE will help support patients and their families.
It is not known when the gene therapy will become available and how much it will cost.
Delivering a lifeline
Existing treatments for SMA are costly, pricing many patients out of critical care.
Itvisma is a new version of Novartis' existing gene therapy, Zolgensma, which provides a working SMN gene to cells, enabling them to produce the SMN protein.
It is also administered only once but has been described as the world’s most expensive drug as the single treatment costs a reported $2 million (Dh7.35 million).
Also available are gene-based treatments that have to be administered several times at a cost of several hundred thousand dollars a year per patient.
In July, a Syrian girl with spinal muscular atrophy received Dh7 million gene therapy paid for by Sheikh Mohammed bin Rashid, Vice President and Ruler of Dubai, at Dubai Health's Al Jalila Children’s Hospital.
Two-year-old Yaqeen Kankar received treatment two weeks after her family made a video appeal on social media for help to save her.
The hospital has completed more than 100 treatments for patients with SMA, said Dr Mohamed Al Awadhi, executive director of the Dubai Health Women and Children’s Campus.
