Novartis's Branaplam (LMI070) Receives the US FDA's Orphan Drug Designation for Huntington's Disease
Shots:
- The US FDA has granted ODD for branaplam in HD. In preclinical trials- branaplam demonstrated a reduction in levels of the mutant huntingtin protein. Additionally- the therapy showed a reduction in huntingtin mRNA in SMA patients
- Novartis expects to initiate the P-IIb study for branaplam in HD patients in 2021
- Branaplam (qw- PO) is an RNA splicing modulator- currently under investigation for the treatment of SMA
Ref: Novartis | Image: Novartis
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