According to a story from PR Newswire, the cell therapy company Cellevolve Bio, Inc., recently announced that the US Food and Drug Administration (FDA) has advanced the development of its investigational therapy CE-VST01-JC. This therapy is being developed as a treatment for progressive multifocal leukoencephalopathy (PML). The agency has cleared the company’s Investigational New Drug (IND) application for the drug and CE-VSY01-JC also earned Orphan Drug designation.
About Progressive Multifocal Encephalopathy (PML)
Progressive multifocal leukoencephalopathy is a rare and frequently lethal viral illness resulting in steadily worsening damage to the white matter of the brain. The disease almost always occurs in people with a severe form of immunodeficiency or who are otherwise immunocompromised. Progressive multifocal encephalopathy is caused by the JC virus, which is normally harmless in people with fully functional immune systems. Symptoms include loss of coordination, progressive weakness, personality changes, difficulties with speech and vision, and possibly others depending on which brain regions are impacted. Treatment is limited but primarily focuses on enhancing the function of the immune system. Highly active antiretroviral therapy may be used in patients with HIV. Some other drugs, such as cytarabine, cidofovir, interleukin-2, and mefloquine have been used with limited success. Up to half of people diagnosed with the disease will die within the first few months of diagnosis; many survivors are left with permanent neurological impairment. To learn more about progressive multifocal leukoencephalopathy, click here.
The IND clearance will allow the company to proceed with its phase 2 clinical trial. The company aims for this trial to be the largest cell therapy study in this rare disease and hopes to enroll 60 patients in total.
About Orphan Drug Designation
The Orphan Drug designation is also a significant milestone for CE-VSY01-JC. This designation is reserved for therapies intended to treat diseases that are considered rare, which is any illness that affects under 200,000 people in the US. Additionally, the therapy must aim to meet a serious unmet medical need or possess potential advantages over current treatment options. Orphan Drug designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a seven-year period of market exclusivity if the drug ultimately gains approval from the FDA.
