Patients of all ages now have access to care for the rare genetic disease.
The Food and Drug Administration (FDA) has announced approval for Itvisma (onasemnogene abeparvovec-brve), which can now be used for adult and pediatric patients 2 years and older with spinal muscular atrophy (SMA). SMA is a neurodegenerative disorder caused by a mutation to the SMN1 gene, which leads to muscle atrophy and possibly paralysis, and death.
The gene replacement therapy is administered by injection into the patient’s cerebrospinal fluid surrounding the spinal cord, allowing for “rapid onset and direct targeting of the genetic root cause of SMA.” Itvisma is the only gene replacement therapy for patients over 2 years of age with SMA.
FDA’s Chief Medical and Scientific Officer and Director of the Center for Biologics Evaluation and Research, Vinay Prasad, said that this 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. 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.”
As the Lord Leads, Pray with Us…
- For Commissioner Makary to be led by the Lord as he heads the FDA.
- For FDA officials as they assess therapies to help individuals with rare diseases.
- For healing for patients of all ages affected by various types of muscular dystrophy.
Sources: Food and Drug Administration, Novartis
