FDA Grants Orphan Drug Designation to Tesomet In Hypothalamic Obesity
Officials with the FDA have granted orphan drug designation to Tesomet (Saniona) for the treatment of hypothalamic obesity (HO). According to a press release from Saniona, Tesomet is the first and only investigational treatment for HO to receive orphan drug designation.
“There are currently no FDA-approved medicines for hypothalamic obesity,” said Rudolf Baumgartner, MD, chief medical officer and head of clinical development at Saniona, in the release. “Despite the devastating weight gain and hunger this rare disease can cause, there has been relatively little drug development specifically for HO. Saniona is proud to be pioneering a regulatory path forward for people living with HO, and we are thrilled to have received the first-ever FDA orphan drug designation in HO. We look forward to initiating our Phase 2b trial of Tesomet for HO in the second half of this year.”
HO is a rare disorder caused by injury to the hypothalamus, most commonly sustained during surgery to remove a craniopharyngioma, a type of noncancerous tumor. HO is characterized by rapid, excessive, and intractable weight gain that persists even when food intake is restricted. Patients with HO may have hyperphagia and may display abnormal food-seeking behavior, such as stealing food.
Tesomet was evaluated for use in patients with HO in a 24-week, double-blind, randomized, placebo-controlled initial phase 2 trial. According to the investigators, adults receiving Tesomet had statistically significant decreases in body weight, improvements in glycemic control, and improvements in waist circumference. These improvements were maintained throughout a 24-week open-label extension to the study. Most adverse events were mild, with the most common being sleep disorders, dizziness, dry mouth, and headache.
“The recognition of the first orphan drug designation in hypothalamic obesity is a critical milestone for the HO community,” said Amy Wood, executive director of the Raymond A. Wood Foundation and parent of a child living with hypothalamic obesity, in the release. “HO places a tremendous burden on caregivers and families; it causes severe weight gain and constant hunger no matter how much a person eats, forcing us to lock up food and avoid social situations where food is served. We are incredibly grateful that both the FDA and Saniona recognize the seriousness of this disorder, and we hope this orphan drug designation is the first step towards having an innovative treatment.”
Orphan drug designation is granted by the FDA for drugs intended to treat rare diseases that affect fewer than 200,000 people in the United States. Currently, the number of people living with HO in the United States is estimated to be between 10,000 and 25,000.
Saniona receives U.S. FDA orphan drug designation for Tesomet in hypothalamic obesity [news release]. Saniona; July 26, 2021. Accessed July 26, 2021. https://news.saniona.com/news-releases/news-release-details/saniona-receives-us-fda-orphan-drug-designation-tesomet