Vanda Pharmaceuticals Inc.’s therapy for patients with a rare developmental disorder has earned a regulatory green light, the District biotech said late Monday.
The company said the Food and Drug Administration has approved Hetlioz, its existing drug for non-24-hour sleep-wake disorder, as a treatment for nighttime sleep disturbances in Smith-Magenis Syndrome.
The agency’s go-ahead applies to a liquid formulation for children and a capsule for adults with SMS, for which no approved treatment has existed up to this point. The disease — marked by intellectual disability, delayed speech, sleep disturbances and behavioral problems — affects roughly 15,000 people nationwide.
The approval represents a major victory for these patients as well as for Vanda (NASDAQ: VNDA), which has scuffled with the FDA on multiple matters over the past couple of years. It’s also Hetlioz’s first additional indication since its initial 2014 approval.
“The FDA approval of Hetlioz for the treatment of nighttime sleep disturbances in SMS would not have been accomplished without the heroic efforts of SMS patients and the efforts of their families and advocates supporting the recruitment, design and conduct of the study,” Dr. Mihael Polymeropoulos, president and CEO of Vanda, said in a statement. “We remain committed to providing this much needed therapy to patients with SMS.”
Hetlioz capsules “will be immediately available” and the liquid form for kids is expected to start selling in the first quarter of 2021.
Vanda’s share price was trading up 9.64% at $13.53 early Wednesday afternoon.
The approval comes after the FDA accepted Vanda’s application for priority review in August, nearly five months after the regulatory body refused to accept that application because of issues the parties subsequently resolved. It also follows the FDA’s rejection last year of Vanda’s application for Hetlioz as a treatment for jet-lag disorder, citing deficiencies in its application.
Still, Vanda marches ahead with a recent FDA approval of its investigational new drug application for a candidate for allergic conjunctivitis, or allergy-related eye inflammation. Its phase 2 study is on track to start enrollment by the end of this year and produce results in 2021.
The company is separately testing drug candidate tradipitant as a potential Covid-19 treatment, based on preliminary results that show the candidate may speed up improvement in patients with pneumonia relating to the coronavirus. Vanda has also resumed patient enrollment in its study of tradipitant as a treatment for gastroparesis after a pandemic-related pause, with eyes toward filing a new drug application in the second half of 2021.
The D.C. biotech reported a 9% increase in net product sales for the first nine months of 2020 despite the pandemic, with $180.5 million in revenue for the first three quarters of the year. Hetlioz accounted for $116.5 million, a 12% increase from the first three quarters of 2019. Vanda closed Q3 with $348.5 million in cash, cash equivalents and marketable securities.
