Orphan designation for Novartis’ branaplam in Huntington’s disease

Novartis’ branaplam (LMI070) has gained Orphan Drug Designation in the US in Huntington’s disease (HD).

In preclinical models, branaplam has been shown to reduce levels of mutant huntingtin protein. In addition, during the investigation of branaplam in spinal muscular atrophy (SMA), it was also observed to reduce huntingtin messenger RNA (mRNA) in SMA patients.

A decrease of huntingtin mRNA is expected to result in reduction of huntingtin protein levels, the underlying cause of HD.

Based on these findings, Novartis intends to start a development program for branaplam to determine if it has the potential to be a transformative treatment for people living with this devastating condition.

Branaplam is currently under investigation for the treatment SMA.  It is dosed once weekly for the treatment of SMA, and the same dosing regimen may also be a possibility for HD.

Novartis plans to start the Phase IIb trial for branaplam in HD patients in 2021.

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