According to a press release published on Globe Newswire, the biopharmaceutical company Saniona has announced recently that its investigational therapy Tesomet has recently earned Orphan Drug designation from the US Food and Drug Administration (FDA). This designation is for the treatment of hypothalamic obesity, a rare form of obesity. The drug has also previously earned the designation for Prader-Willi syndrome, which is a rare genetic disorder that can cause obesity.

About Hypothalamic Obesity

Hypothalamic obesity is a rare condition in which obesity occurs as a result of damage to the hypothalamus, a region of the brain that plays a role in the endocrine system and controls functions such as hunger, hormones, sleep, and body temperature. The cause of damage to the hypothalamus may include physical trauma, a birth defect, surgery, brain swelling, or a tumor. As a result, this condition is usually acquired and not genetically inherited. The primary symptom is obesity, triggered by extreme hunger and a low metabolic rate. If the pituitary gland is affected, delayed puberty and infertility may occur. Seizures, ADHD, and other psychiatric conditions may be present. Treatment is symptomatic and may include diabetes medications, nutritional and lifestyle counseling, and gastric surgery. There is no cure at this time. To learn more about hypothalamic obesity, click here. 

About Orphan Drug Designation

Orphan Drug designation is generally reserved for therapies that are being developed for diseases that are considered rare. This is defined as any illness that affects less than 200,000 people in the US. To qualify, the therapy must either have the potential to satisfy a currently unmet medical need or display potential advantages in efficacy and/or safety over currently available treatments. This designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a period of market exclusivity lasting seven years if the drug is ultimately approved by the FDA.

About Tesomet

Tesomet is the first medicine to ever receive this designation for the treatment of hypothalamic obesity, making it a significant moment for the patient community. The drug has been evaluated for the disease in a phase II clinical trial. Adult patients in the trial saw significant weight loss while using the medicine with few adverse effects. A phase IIb study is planned to begin later this year.