Saniona (OMX: SANION), is a clinical stage biopharmaceutical company focused on rare diseases. Today they announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Tesomet for the treatment of hypothalamic obesity (HO).
Tesomet is the first and only investigational treatment for HO to receive orphan drug designation. Saniona is preparing to initiate two Phase 2b studies of Tesomet in the second half of this year, one in HO and the other in Prader-Willi syndrome (PWS), for which Tesomet has already received orphan drug status.
“There are currently no FDA-approved medicines for hypothalamic obesity. 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,” said Rudolf Baumgartner, M.D., Chief Medical Officer and Head of Clinical Development at Saniona AB.
Amy Wood, Executive Director of the Raymond A. Wood Foundation and parent of a child living with hypothalamic obesity, commented, “The recognition of the first orphan drug designation in hypothalamic obesity is a critical milestone for the HO community. 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 a special status granted by the FDA to medicines and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. The number of people living with HO is estimated to be between 10,000 and 25,000 in the U.S. and between 16,000 and 40,000 in Europe. Receiving orphan designation qualifies Saniona for certain development benefits, including tax credits, elimination of certain FDA license application fees, and seven years of market exclusivity in the U.S. following approval.
Saniona AB previously evaluated Tesomet in a 24-week, double-blind, randomized, placebo-controlled initial Phase 2 trial in HO. Adults receiving Tesomet demonstrated statistically significant reductions in body weight and improvements in waist circumference and glycemic control. These improvements were maintained during an additional 24-week open-label extension. Tesomet was reported to be generally well-tolerated, and most adverse events were mild. The most common adverse events included sleep disorders, dizziness, dry mouth and headache. No clinically significant differences in heart rate or blood pressure were observed between the Tesomet-treated patients and the placebo-treated patients.
In preparation for the initiation of the Phase 2b study of Tesomet in HO in the second half of 2021, Saniona has selected the clinical research organization (CRO) that will support the clinical trial and is in the process of assessing and selecting clinical trial sites in the U.S. and globally. Saniona has also selected the contract manufacturer to produce Tesomet for Phase 2b and Phase 3 clinical trials. Additionally, Saniona AB has initiated multiple partnerships with the HO advocacy community to incorporate caregiver and patient feedback into the clinical trial process and to provide the community with education on clinical trials.
Saniona AB is also evaluating Tesomet for the treatment of Prader-Willi syndrome (PWS) and plans to begin a Phase 2b trial in this indication in the second half of this year. The FDA granted orphan drug designation to Tesomet for the treatment of PWS in March 2021.