According to a story from Global Genes, the US Food and Drug Administration (FDA) has said that it will not take the pharmaceutical company Biohaven’s recent New Drug Application under review. The application is for a drug called troriluzole as a treatment for spinocerebellar ataxia, a rare disease. The agency’s reasoning for refusing to review the application is because the drug did not satisfy the primary endpoint in its phase 3 clinical trial.
About Spinocerebellar Ataxia
Spinocerebellar ataxia (SCA), also known as spinocerebellar atrophy, is a progressive, degenerative disease that affects the cerebellum, a portion of the brain that is critical for movement control and coordination. These heritable disorders can often be fatal. There are several different types of spinocerebellar ataxia which are linked to a variety of genetic mutations; many types are caused by repeats of the CAG gene. Symptoms of these disorders include ataxia (abnormal gait), seizures, poor hand coordination, speech difficulties, peripheral neuropathy, chorea, and cognitive impairments. Unfortunately, there is no cure for spinocerebellar ataxia and treatment is mostly focused on relieving symptoms. Physical and occupational therapy can help patients maintain their mobility. Gene therapy could be a potential future option for treating the disorder. There is a dire need for more effective treatments for this disorder. To learn more about spinocerebellar ataxia, click here.
The End of the Road?
Without decisive results from a clinical study, the application does not permit a ‘substantive’ evaluation of the drug. The company has 30 days to meet with the FDA to discuss possible next steps or other options.
CEO and chairman of Biohaven, Vlad Coric, alleges that the refusal means that the agency won’t be able to evaluate all of the available data. Around 200 people living with spinocerebellar ataxia type 3 have been treated with the drug for as long as three years. He also believes that these patients have remained relatively stable in comparison to natural history cohorts, who have seen further disease progression.
“We believe the NDA package is compelling and shows that treatment with troriluzole leads to clinically meaningful treatment benefits including significantly delaying disease progression and reduction in falls.” – Vlad Coric, CEO, Biohaven
