Emirates Pharmaceuticals approves gene therapy for patients with spinal muscular atrophy

The Emirates Medicines Corporation announced the approval of the drug “Etvizma”, which is a gene therapy based on an adenoviral vector, which is used to treat spinal muscular atrophy in adults and children, starting from the age of two years and above, in the medically qualified category. With this decision, the UAE becomes the second country in the world to grant approval for this treatment, which strengthens its leadership in accelerating the arrival of advanced medical innovations to patients in the region.
“Etvisma” treatment works to target the genetic cause of spinal muscular atrophy by replacing the gene responsible for the disease, which contributes to improving patients’ motor functions and reducing their dependence on long-term treatments.
This accreditation comes within the framework of the Emirates Pharmaceutical Corporation’s ongoing efforts to provide advanced, high-value treatments and accelerate the arrival of pharmaceutical innovations to the Emirati community, in line with the country’s trends towards developing a pioneering health sector based on efficiency, innovation and sustainability.
Dr. Fatima Al Kaabi, Director General of the Emirates Drug Corporation, said: Issuing regulatory approval for the drug “Etvizma” represents a pivotal step that reflects the UAE’s commitment to providing the latest advanced genetic treatments for patients, especially those with rare genetic diseases such as spinal muscular atrophy. This decision embodies the efficiency of the health system in the country and its ability to evaluate and approve innovative medicines according to the highest scientific standards and within effective periods of time, ensuring that patients have rapid access to pioneering treatment options that enhance their quality of life.
Al-Kaabi explained that the approval of the drug is based on the results of clinical studies that demonstrated a clear improvement in patients’ motor abilities and the sustainability of these benefits in the long term, in addition to the positive safety profile that the treatment showed during the clinical evaluation stages.
She added: “The fact that the UAE grants regulatory approval for this treatment reflects its leadership in adopting and approving advanced pharmaceutical technologies, and enhances international confidence in the ability of its regulatory model to achieve an accurate equation that combines speed, accuracy and transparency, ensuring that innovative treatments reach patients in a timely manner.”
She concluded by saying: “The Emirates Pharmaceutical Corporation will continue to support medical innovation and adopt modern treatments that improve the quality of life, in line with the country’s vision to build a pioneering health sector based on knowledge, efficiency and sustainability.”
Regional Director of Novartis in the GCC region, Mohamed Ezz El-Din, confirmed that the approval of the drug “Etvisma” in the UAE comes primarily to serve patients and their families. He pointed out that the close cooperation with the Emirates Drug Foundation and partners aims to ensure rapid and equitable access to treatment, allowing people with spinal muscular atrophy who were previously unable to benefit from single-dose gene therapy a new opportunity to obtain this type of medical intervention.
He added that the UAE’s support to accelerate the adoption of innovative treatments contributes to changing the disease course of spinal muscular atrophy, stressing the company’s pride in its role in this medical progress. He pointed out that this achievement reflects Novartis’ commitment to the field of neurology, based on its extensive experience in treating spinal muscular atrophy and multiple sclerosis, and coinciding with its expansion in the fields of neuroimmunology, neurodegenerative disorders, and diseases of the neuromuscular system.
The regulatory approval of the drug “Etvizma” comes within the framework of the Emirates Drug Corporation’s strategy aimed at strengthening the country’s position as a regional center for pharmaceutical innovation, by providing advanced gene therapies at the highest level of quality and safety. The organization employs a modern organizational model based on careful governance, transparency, and global partnerships, to support research and development and adopt the latest technologies and integrated care.
This approach contributes to ensuring that patients have efficient access to advanced medicines, and enhances the country’s readiness in the healthcare sector, in line with the UAE’s vision of building a pioneering and sustainable health system.”