Listen to this articleThe basics:
- FDA approves Novartis‘ Itvisma as first gene replacement therapy for SMA patients age 2+
- Treatment targets the SMN1 gene mutation, the root cause of spinal muscular atrophy
- One-time intrathecal dose may improve motor function and reduce chronic therapy needs
Novartis has received approval from the U.S. Food and Drug Administration for Itvisma, a treatment for spinal muscular atrophy.
The Swiss drugmaker with U.S. headquarters in East Hanover announced the green light Nov. 24.
Itvisma – or onasemnogene abeparvovec-brve – is intended for the treatment of patients 2 years and older living with SMA with a confirmed mutation in the survival motor neuron 1 (SMN1) gene.
The pharmaceutical company noted that it is the first gene replacement therapy available for these patients.
Dr. John Day is professor of neurology and pediatrics, director of the Division of Neuromuscular Medicine at Stanford University School of Medicine, and co-director of Stanford’s Neuro IGNITE Center. He applauded the federal nod.
“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,” Day said in Novartis’ announcement. “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.”
How does it work?
SMA is a rare, genetic neuromuscular disease caused by a mutated or missing SMN1 gene. That gene is responsible for producing most of the SMN protein a body needs for muscle function, including breathing, swallowing and basic movement, according to Novartis.
Without treatment, patients may experience progressive, debilitating muscle weakness.
Novartis estimated that about 9,000 people live with the disease in the U.S. Meanwhile, the SMA Foundation puts that range at 10,000 to 25,000 children and adults.
Itvisma addresses the genetic root cause of SMA with a one-time dose, Novartis explained. By replacing the SMN1 gene, Itvisma can improve motor function. Additionally, it may potentially reduce the need for chronically administered treatment associated with other available therapies.
Itvisma is administered directly to the central nervous system by a single intrathecal injection.
‘The power of gene therapies’
“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 officer as well as 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,” Prasad added.
Novartis’ FDA approval follows closely other major announcements from the company, such as plans to build a $771 million a flagship manufacturing hub in North Carolina as well as its $12 billion acquisition of San Diego-based biopharma Avidity Biosciences.
