Novartis (NVS) Gets Orphan Drug Tag for Huntington's Disease Drug

Novartis NVS announced that the FDA has granted orphan drug designation to branaplam (LMI070) in Huntington’s disease (HD).Huntington’s disease is a rare, inherited neurodegenerative disease that leads to progressive disability and death.

Notably, orphan drug designation is granted to drugs that are capable of treating rare diseases that affect less than 200,000 people in the United States. This tag also makes the company entitled to certain other benefits, including tax credits related to clinical trial expenses, an exemption from the FDA user fee and a designated period of market exclusivity following approval.

Branaplam (LMI070) is an orally-administered, small-molecule RNA splicing modulator that could potentially reduce the levels of mutant huntingtin protein.

In preclinical studies, branaplam has been shown to reduce levels of mutant huntingtin protein. Further, when branaplam was being evaluated in a study to treat spinal muscular atrophy (SMA), it was observed that it reduces huntingtin messenger RNA (mRNA) in SMA patients. A decrease in huntingtin mRNA is expected to result in the reduction of huntingtin protein levels, the underlying cause of HD. Based on these findings, the company plans 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. Novartis plans to start the phase IIb study onbranaplam in HD patients in 2021.

Branaplam is currently being evaluated for the treatment of SMA. SMA is a rare, progressive, genetic disease, characterized by the loss of motor neurons that are responsible for muscle function. The candidate is dosed once weekly for the treatment of SMA and the same dosing regimen might also be followed for HD.

Novartis has a strong oncology portfolio and continues to work on developing its immuno-oncology pipeline. However, Novartis is faced with intense competition by companies like Pfizer PFE, Merck MRK, Bristol-Myers Squibb BMY.

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