Novartis has secured orphan drug designation from the US Food and Drug Administration (FDA) for its branaplam (LMI070) to treat Huntington’s disease (HD).
Branaplam has been demonstrated to decrease levels of mutant huntingtin protein in preclinical models, said Novartis.
Based on the results, Novartis aims to begin a development programme for branaplam to determine if it can treat people living with HD.
HD is a rare and inherited neurodegenerative disease that may result in progressive disability and death. It is characterised by progressive decline in motor, cognitive and psychiatric symptoms, which generally appear between the ages of 30 to 50, and worsen over a 15-20-year period.
According to the company, around 70,000 people across Europe and the US were clinically diagnosed with HD.
At present, branaplam is being assessed to treat spinal muscular atrophy (SMA), which is a rare and progressive genetic disease characterised by loss of motor neurons that are responsible for muscle function.
It was also observed to decrease huntingtin messenger RNA (mRNA) in SMA patients during the investigation of branaplam in SMA, said the company.
Branaplam is a once-weekly, orally administered and small molecule RNA splicing modulator, which is currently under assessment to treat SMA.
Novartis is planning to begin the phase IIb trial for branaplam in HD patients in 2021.
The approval allows Kesimpta to be used as an injection for subcutaneous use to treat RMS, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults.