The Food and Drug Administration approved Itvisma (onasemnogene abeparvovec-brve) Nov. 25 for the treatment of spinal muscular atrophy (SMA) in adult and pediatric patients 2 years of age and older with confirmed mutation in the survival motor neuron 1 (SMN1) gene.
Itvisma is manufactured by Novartis Gene Therapies Inc. Novartis said Itvisma is the first and only gene replacement therapy available for this broad population. Itvisma is an adeno-associated virus (AAV) vector-based gene therapy.
Spinal muscular atrophy is a rare, genetic neuromuscular disease caused by a mutated or missing SMN1 gene, which is responsible for the production of a protein needed for muscle function, including breathing and swallowing. Spinal muscular atrophy is the leading genetic cause of infant deaths worldwide and about 9,000 people in the U.S. live with the condition.
“Today’s approval shows the power of gene therapies and offers treatment to patients across the SMA disease spectrum, including patients at various ages, SMA symptoms and motor functional levels,” said Dr. Vinay Prasad, the FDA’s chief medical and scientific oOfficer and director of the center for biologics evaluation and research.
“This exciting area of science continues to change the lives of patients and the FDA is committed to expediting the development of products for unmet medical needs.”
SMA is an autosomal-recessive neurodegenerative disorder caused by mutations in the SMN1 gene, characterized by irreversible and progressive motor neuron loss, leading to progressive muscle atrophy and weakness, and subsequent paralysis and death in the most severe cases. SMA has an incidence of about 4-10 per 10,000 live births. Prior to the availability of effective treatment, SMA was considered one of the leading causes of infant mortality due to genetic disease in the U.S.
“The FDA’s approval of intrathecal onasemnogene abeparvovec is a game-changing advance, expanding the use of transformational gene replacement therapy for SMA across age groups,” said Dr. John W. Day, professor of neurology and pediatrics, director, Division of Neuromuscular Medicine at Stanford University School of Medicine, and co-director of Stanford’s Neuro IGNITE Center. “This achievement is not only a significant step forward for SMA – it also signals new possibilities for the broader field of neurological disorders and genetic medicine.”
The FDA granted this application Fast Track, Breakthrough Therapy, and Priority Review designations. Itvisma also received Orphan Drug designation, which provides incentives to encourage the development of drugs for rare diseases.
