November 25, 2025 — Leads & Copy — The Muscular Dystrophy Association (MDA) is celebrating the U.S. Food and Drug Administration’s approval of Itvisma (onasemnogene abeparvovec-brve), a gene replacement therapy developed by Novartis, as a significant advancement for individuals and families affected by spinal muscular atrophy (SMA).
This approval marks Itvisma as the first and only gene replacement therapy accessible to a broader SMA population, including children aged two and older, teenagers, and adults with a confirmed mutation in the SMN1 gene. Itvisma is administered intrathecally, a formulation of Zolgensma, which was previously approved for infants with SMA. This new delivery method enables the safe and effective application of gene replacement therapy to older individuals for the first time, offering renewed hope to the SMA community.
According to Angela Lek, PhD, Chief Research Officer at the Muscular Dystrophy Association, the FDA’s decision is the result of decades of foundational SMA research and the combined efforts of scientists, clinicians, industry partners, and advocates. She noted that MDA has supported many of the discoveries that paved the way for this milestone, reinforcing the impact of collaboration, innovation, and the inclusion of families living with neuromuscular disease.
Dr. Barry J. Byrne, Chief Medical Advisor and Board Member at the Muscular Dystrophy Association and Director of the Powell Gene Therapy Center at the University of Florida, highlighted the importance of this moment for both the SMA community and the field of neuromuscular medicine. He mentioned that Itvisma’s approval broadens therapeutic options for patients within the MDA Care Center Network, who previously had limited access to gene therapy, and demonstrates the potential of precision genetic treatments for neuromuscular diseases.
For nearly 75 years, MDA has supported research across more than 300 neuromuscular diseases, including SMA, investing nearly $51 million in SMA research since its inception. This includes $2 million invested in six active SMA research grants from 2020 to 2025.
Mindy Henderson, Vice President of Disability Outreach & Empowerment at the Muscular Dystrophy Association, stated that the approval of Itvisma signifies both scientific progress and renewed hope for people and families living with SMA. She emphasized MDA’s commitment to making the previously impossible, possible.
SMA is characterized as a rare genetic neuromuscular disease marked by progressive muscle weakness and atrophy due to motor neuron loss in the spinal cord. Itvisma addresses the underlying genetic cause of SMA by delivering a functional copy of the SMN1 gene directly to motor neurons. This allows for the production of SMN protein, essential for motor neuron survival and function.
The Muscular Dystrophy Association provides ongoing support for families and medical professionals. The MDA Resource Center can be contacted by phone at 1-833-ASK-MDA1 (1-833-275-6321) or by email at ResourceCenter@mdausa.org. Media inquiries should be directed to press@mdausa.org.
Mary Fiance, National Vice President, Strategic Communications at the Muscular Dystrophy Association, can be reached through press@mdausa.org.
To learn more about the Muscular Dystrophy Association, visit MDA75.org.
Source: Muscular Dystrophy Association
