Novartis discontinues development of SMA drug branaplam

Earlier this week, Novartis announced its decision to discontinue development of branaplam, an investigational drug for the treatment of spinal muscular atrophy (SMA). This is disappointing news, but the company notes its decision is driven by the fact that there are already three licensed treatments for SMA.

Novartis’s own gene therapy, known as Zolgensma, is available in the UK on the NHS, while Biogen’s Spinraza is also available to many people with SMA in the UK. And NICE is currently carrying out an appraisal of the third drug, called Evrysdi. Although this third drug has not yet been approved, we hope that its manufacturer, Roche, and NHS England’s commercial arm reach an agreed price that will allow it to be recommended.

It is always regrettable when a potential treatment is not taken forward. But in this case, Novartis suggests that "branaplam would not offer a highly differentiated treatment solution for the SMA community". This is because it works in a similar way to both Spinraza and Evrysdi, making it difficult to persuade healthcare providers to pay for it.