One of Cure SMA’s top priorities is to relay the SMA community’s experiences, preferences, and unmet needs to the United States Food and Drug Administration (FDA). This enables the FDA to make patient-centered decisions about new SMA drugs.
This is a critical time for patient input on SMA treatment, as new therapies and variations of approved disease-modifying treatments are currently being considered by the FDA.
Last year, Cure SMA provided an update on the state of spinal muscular atrophy (SMA). Our outreach highlighted the benefit of current SMA treatments and reported the significant unmet needs faced by children and adults with SMA. Since then, Cure SMA has collected direct experiences from the SMA community documenting treatment impact, healthcare barriers, and unmet needs. We are now pleased to share with FDA our latest annual State of SMA report, our latest publication on unmet needs among adults living with SMA, and first-person perspectives from the SMA community with FDA. To see the full FDA package click here.
The three FDA approved therapeutics represent landmark advances in the treatment of SMA. We appreciate the FDA’s long-standing commitment to and engagement with the SMA community. We know that the patient voice is critical to drug development and will continue to share community data with FDA.
For more information about the investigational drugs under FDA review see:
FDA Accepts Application for Higher-Dose Nusinersen
Scholar Rock Submits BLA for Apitegromab
Novartis submits FDA application for OAV101 IT, an intrathecal gene therapy candidate for SMA.