Inhibikase receives orphan drug designation for PML treatment
- FDA gave an orphan drug designation to imatanib, a tyrosine-kinase inhibitor commonly used to treat various cancers. Inhibikase Therapeutics has extensively tested this drug for the treatment of progressive multifocal leukoencephalopathy (PML).
- PML is a rare side effect associated with certain antibody-based treatments for autoimmune diseases. It also occurs in 1% to 3% of AIDS patients
- Imatanib is also the main ingredient in Gleevec, which was developed by Novartis and used for the treatment of chronic leukemia.
Progressive multifocal leukoencephalopathy (PML) is a side effect associated with at least 13 drugs. Drugs used as immunomodulators, such as Tysrabi for the treatment of multiple sclerosis, can weaken the body’s immune system to the point that patients become susceptible to the JC virus, which leads to a potentially fatal brain infection. Inhibikase Therapeutics has received an orphan drug designation for imatanib, which currently represents the only treatment for PML. Having a treatment option for PML can decrease the risk associated with treating patients with autoimmune diseases.