Molecola in corso di sperimentazione per la SMA sarà sperimentata anche nell'Huntington

Treatment for SMA could be used for Huntington’s disease: new trial planned for next year


On October 21st, the pharmaceutical company Novartis has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug status to branaplaman experimental drug developed by the company for the treatment of spinal muscular atrophy (SMA), for treatment of Huntington’s disease.

Branaplam is currently tested for the treatment of SMA, a rare neuromuscular disease and it works by interfering with how genetic messages are processed in cells and can increase the levels of a protein called SMN2. Boosting levels of SMN2 helps SMA patients. 

Banaplam could also have beneficial effects in the treatment of Huntington's disease, reducing the levels of the huntingtin gene message and the huntingtin protein.

For this reason, Novartis plans to start a phase II / b trial in 20021 in order to test the efficacy of branaplam in treating Huntington's disease. The huntingtin-lowering therapies currently in clinical trials need to be given through spinal injections (Generation HD1) or brain surgery (ATM-130 gene therapy). Unlike these, branaplam is a small molecule drug, which means it can be taken as a tablet.

On this topic, Ferdinando Squitieri stated: "Even if the drug might represent a precious resource that can be administered orally, supposedly, in this experimental phase, it will not yet be possible, to completely eliminate the need to perform an intrathecal lumbar puncture to test the effects in the CSF" 


Text from HD Buzz Read more 

What are Orphan Drugs 

Novartis Official Press Release