FDA Gives Inhibikase Therapeutics Orphan Drug Designation For PML Treatment
Inhibikase Therapeutics has announced that it has received orphan drug designation for its treatment of Progressive Multifocal Leukoencephalopathy, or PML. The company’s drug, imatinib, is used to combat the JC virus infection that PML patients in particular can be affected by.
PML is a lethal brain infection that often occurs in patients that have a suppressed immune system, as the brain is more easily infected with the John Cunningham (JC) virus when the immune system is suppressed. The JC virus lives in most people without causing any medical problems. However, when the immune system is suppressed, the JC virus can find its way to the brain and destroy critical brain cells. The destruction of these cells causes a loss in motor and cognitive functions. PML is rare — it affects patients with autoimmune diseases who are treated with small molecule and antibody drugs and affects between 1 and 3 percent of clinical AIDS patients.
Dr. Jeffrey B. English, the Director of Clinical Research at the MS Center of Atlanta, commented on the FDA’s decision to give orphan drug designation to Inhibikase. “Multiple Sclerosis (MS) can be a very disabling disease. To date, Tysabri is our most effective treatment,” he said. “Unfortunately, it carries a risk of a life threatening brain infection that can lead to PML. There are ways to screen for PML early, but we have no effective treatments for this disease,” commented Dr. English. “If there was a way to treat PML, this would open up a pathway for many more patients to receive Tysabri, the most effective treatment for MS.”
Milton H. Werner, PhD, President, and CEO of Inhibikase, said that the orphan drug designation will help the company continue to develop IkT-001Pro, the company’s lead product which uses imatinib to disrupt the JC virus’ ability to reproduce in the body. He added that IkT-001Pro could be used to treat PML should it arise as a side effect from any other future treatments that enter the market.
Dr. Timothy Coetzee, an advocate with the National MS Society, said that the drug being produced by Inhibikase was meeting a need for patients who have very limited treatment options.