Ovid Therapeutics, a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to TAK-935/OV935 for the treatment of Dravet syndrome, a severe and rare form of childhood epilepsy that typically presents during the first year of life. Takeda Pharmaceutical Company Limited and Ovid formed a global collaboration to develop and commercialize TAK-935/OV935 for the treatment of developmental and epileptic encephalopathies in January 2017.
Dravet syndrome is classified as a developmental and epileptic encephalopathy, a group of rare epilepsies that cause significant morbidities and can worsen over time. Children with Dravet syndrome experience frequent seizures, loss of muscle control, cognitive deficits and, in approximately 10 percent of cases, death before the age of 12 years. Moreover, in those who survive into adulthood, their long-term intellectual development and seizure outcomes are typically extremely poor.
“We are pleased by the FDA’s decision to grant orphan drug designation to TAK-935/OV935 for the treatment of Dravet syndrome, a severe and debilitating disease,” said Dr. Emiliangelo Ratti, head of Takeda’s Neuroscience Therapeutic Area Unit. “This designation is a significant step forward in researching a potential treatment option for people living with Dravet syndrome for whom therapeutic options are severely limited, and an important milestone for this investigational molecule.”
TAK-935/OV935 is a potent, highly-selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H). It is believed that CH24H is involved in over-activation of the glutamatergic pathway, which has been shown to play a role in the initiation and spread of seizure activity. To Ovid and Takeda’s knowledge, TAK-935/OV935 is the only molecule with this mechanism of action in clinical development.
“We believe that TAK-935/OV935, with its novel mechanism of action, has the potential to be an innovative treatment for people with rare epilepsies, such as Dravet syndrome,” said Matthew During M.D., DSc, FACP, FRACP, president and chief scientific officer of Ovid Therapeutics. “We have rapidly advanced this program into a Phase 1b/2a clinical trial and anticipate data in 2018. We look forward to continuing our work with Takeda to bring this potentially transformative therapy to patients.”
Orphan drug designation is intended to facilitate and expedite drug development for rare diseases for which there are no current treatments available. It also provides substantial benefits to the sponsor, including the potential for tax credits for clinical development costs, study-design assistance, and several years of market exclusivity for the product upon regulatory approval.
About Dravet Syndrome
Dravet syndrome is a severe form of childhood epilepsy that typically presents during the first year of life. It is believed to be largely caused by mutations in the SCN1A gene. Children experience frequent seizures, loss of muscle control, cognitive deficits and, in approximately 10 percent of cases, death before the age of 12 years. While some patients may survive into adulthood, their long-term intellectual development and seizure outcomes are typically extremely poor. The incidence of Dravet syndrome in the United States ranges from 1 in 15,700 to 1 in 20,900 births. Patients are frequently treated with combinations of classic anti-epileptic drugs, none of which are particularly effective. No drugs have been approved specifically for the treatment of Dravet syndrome in the United States and only one drug, the anticonvulsant stiripentol, has been approved in Europe.
Dravet syndrome is one of several disorders which together are designated as developmental and epileptic encephalopathies. This group includes epilepsy syndromes associated with severe cognitive and behavioral disturbances. The International League Against Epilepsy (ILAE) defines an epileptic encephalopathy as a condition in which “the epileptiform EEG abnormalities themselves are believed to contribute to a progressive disturbance in cerebral function.”
These epilepsies cause significant morbidities for patients beyond what might be expected from the known underlying pathology alone and can worsen over time. Developmental and epileptic encephalopathies typically present early in life and are often associated with severe cognitive and developmental impairment in addition to frequent treatment-resistant seizures throughout the person’s lifetime. These disorders vary in age of onset, developmental outcomes, etiologies, neuropsychological deficits, electroencephalographic (EEG) patterns, seizure types and prognosis.