FDA grants rare pediatric disease designation to relutrigine for Dravet
Developer Praxis expects to launch trial by June involving Dravet patients
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to relutrigine, a treatment candidate for Dravet syndrome that’s being developed by Praxis Precision Medicines.
The therapy aims to reduce seizures in children with epileptic disorders such as Dravet.
“We are thrilled to have been granted rare pediatric disease designation for relutrigine in Dravet syndrome,” Marcio Souza, president and CEO of Praxis, said in a company press release.
The FDA gives this designation to experimental therapies designed to treat rare disorders that affect children. The designation aims to provide incentives for companies developing treatments for rare diseases — given that the small number of patients affected means there might not otherwise be a profitable market. A major perk of rare pediatric disease designation is that, if the therapy is ultimately approved, the developer gets a voucher that can be used to get a faster FDA review of another experimental treatment. These vouchers can be used or sold, often for millions of dollars.
“This milestone reflects our commitment to addressing critical unmet needs in rare neurological disorders and underscores the potential of relutrigine as a meaningful new option for patients and families affected by this debilitating developmental and epileptic encephalopathy,” Souza said.
Rare pediatric disease designation provides perks to drug developers
Dravet syndrome is a developmental and epileptic encephalopathy, or DEE — a broad term encompassing brain disorders that cause seizures and problems with development. Dravet is caused mainly by mutations in the gene SCN1A, which provides instructions to build a protein that forms a subunit of a sodium channel.
Seizures are marked by abnormal bursts of electrical activity in the brain. Brain cells send electrical signals by moving electrically charged salt particles, known as ions. One of the most important ions involved in nerves’ electrical signaling is sodium.
Relutrigine is designed to reduce the movement of sodium ions in nerve cells, thereby lessening the abnormal electrical activity that drives seizures. Praxis is now running a study called EMBOLD that’s testing the therapy in people with DEEs caused by mutations in the genes SCN2A or SCN8A. Early data have suggested that relutrigine is better than a placebo at preventing seizures in individuals with these mutations.
The [rare pediatric disease] designation builds upon encouraging clinical data demonstrating relutrigine’s ability to reduce seizure frequency and improve overall seizure control, highlighting our dedication to advancing innovative therapies that can significantly impact quality of life for those with limited treatment options.
Praxis also plans to start another clinical trial, called EMERALD, in the first half of this year. EMERALD will enroll patients with varying types of DEEs, including Dravet syndrome.
“The [rare pediatric disease] designation builds upon encouraging clinical data demonstrating relutrigine’s ability to reduce seizure frequency and improve overall seizure control, highlighting our dedication to advancing innovative therapies that can significantly impact quality of life for those with limited treatment options,” Souza said.
As a potential treatment for DEEs associated with SCN2A or SCN8A mutations, relutrigine has previously been granted orphan drug designations by the FDA and authorities in Europe. Like rare pediatric disease designation, orphan drug designation aims to give extra economic incentives for companies investing in developing treatments for rare diseases.
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