WASHINGTON (dpa-AFX) - The U.S. Food and Drug Administration has approved gene therapy for the treatment of Spinal Muscular Atrophy.
Itvisma (onasemnogene abeparvovec-brve), an adeno-associated virus vector-based gene therapy, has been approved to treat adults and pediatric patients 2 years and older with confirmed mutation in the survival motor neuron 1 (SMN1) gene.
'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 Vinay Prasad, the FDA's Chief Medical and Scientific Officer 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.'
Spinal Muscular Atrophy, or 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 approximately 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.
Itvisma demonstrated substantial evidence of effectiveness for the treatment of SMA in children abone 2 years with a confirmed mutation in the SMN1 gene based on primary evidence of effectiveness from the adequate and well controlled Phase 3 study, FDA said.
The direct administration of Itvisma into the cerebrospinal fluid surrounding the spinal cord (site of action) allows for delivery to motor neurons with a lower dose of vector, without the need to adjust for the patient's body weight. This provides a treatment with rapid onset and direct targeting of the genetic root cause of SMA. By addressing the root cause of SMA, Itvisma restores SMN protein production and halts further disease progression.
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