According to a story from PR Newswire, the US Food and Drug Administration (FDA) has recently approved a new treatment for Dravet syndrome, a rare disorder that causes seizures and other problems. This new treatment is called fenfluramine (marketed as Fintepla) and it has been cleared for use in patients aged 2 or older. The availability of this new treatment is good news for patients and families affected by this disease, which can have debilitating effects.
About Dravet Syndrome
Dravet syndrome is a form of severe epilepsy that usually begins by the sixth month of life. The disease is most characterized by frequent seizures that are sometimes triggered by fever or hot temperatures. In most cases, the syndrome is caused by mutations of the SCN1A gene. Most of the time, these mutations are not heritable and appear spontaneously. The predominant symptom of Dravet syndrome is seizures; varying types of seizures often occur as the disorder progresses, as well as ataxia, developmental delays, behavioral disorders that may resemble autism, and cognitive impairment. Seizures can be potentially lethal. Management strategies for Dravet syndrome may include a ketogenic diet, anticonvulsant medications, cognitive rehabilitation therapy, and cannabidiol. This rare disorder is estimated to appear anywhere from 1 in every 20,000 to 1 in every 40,000 births. To learn more about Dravet syndrome, click here.
The approval of Fintepla for this illness follows promising data from a clinical trial involving 202 patients living with Dravet syndrome. In this study, the primary endpoint was changes in the frequency of convulsive seizures. Within 3-4 weeks Fintepla was able to significantly bring down the frequency of seizures in comparison to the placebo group. The effect was consistent over the duration of the period for treatment.
While Fintepla is clearly a useful therapy for this syndrome, it does not come without risks, and it is linked to pulmonary arterial hypertension and valvular heart disease. These are serious illnesses and as a result of this risk Fintepla is only going to be available through a program of restricted distribution. This will include a risk evaluation and mitigation strategy. Patients are required to have heart monitoring prior, during, and after treatment has ceased.
Fintepla was approved under Priority Review protocols.