SMA Newsroom

Jul 23, 2021

Novartis stops development of branaplam for SMA

Novartis has made the decision to discontinue development of branaplam, an investigational oral, once-weekly RNA splicing modulator, for the treatment of SMA.

This was a difficult decision that was made as the result of rapid advancements in the SMA treatment landscape in recent years.

Knowing the progressive nature of this devastating, genetic disease, maintaining continuity of care for all children enrolled in the branaplam SMA clinical trial is Novartis' top priority. As this decision was not driven by the safety or efficacy of branaplam, the company encourages patients to continue branaplam treatment until an alternate solution is arranged for ongoing care. In partnership with trial investigators, Novartis will ensure treatment solutions are determined on a case-by-case basis, tailored to the individual needs of each child.

There are currently three approved treatments for SMA, including two RNA splicing modulators and a one-time gene therapy. The wide-spread availability of these treatments impacts the feasibility of conducting a registration-enabling clinical trial for branaplam in this small patient population. The company also recognises that branaplam would not offer a highly differentiated treatment solution for the SMA community. They are however continuing to advance the investigation of branaplam for Huntington’s Disease, a rare, genetic disease without any approved, disease-modifying therapies.

Novartis remains unwavering in its commitment to use science-based innovation to improve outcomes for SMA patients. The company thanks the parents who have children in this study for their courage and commitment. They understand that participating in any study is one made with both heart and mind so are truly thankful for the dedication. Novartis is also grateful to the whole SMA community for its continued support and generous advice.