Novel Drug Granted Breakthrough Therapy for Hypothalamic Obesity
The U.S. Food and Drug Administration (FDA) has designated setmelanotide, a brand-new medication developed by Rhythm Pharmaceuticals, as a breakthrough therapy for the management of hypothalamic obesity.
The FDA's designation of a drug as a "breakthrough therapy" is meant to expedite the development and review of medicines that are intended to treat serious conditions with unmet medical needs and for which there is preliminary clinical evidence that suggests the medication may significantly outperform existing therapies on one or more clinically significant endpoints.
MC4R pathway, which regulates physiological processes including hunger and weight management, is part of the hypothalamus area of the brain, which is damaged in hypothalamic obesity, a rare, acquired form of extreme obesity.
It usually occurs after the development or surgical removal of a craniopharyngioma, an astrocytoma, or another uncommon brain tumour. In the first six to twelve months after tumour resection, patients have fast weight gain, a decrease in energy expenditure, and an increase in hunger, leading to severe obesity.
According to Rhythm, there are roughly 500 new cases of acquired hypothalamic obesity every year, affecting between 5,000 and 10,000 people in the United States.
Based on the outcomes of a Phase 2, 16-week clinical investigation with setmelanotide, the designation of "Breakthrough Treatment" was given.
Preliminary data indicates that all 11 evaluable patients experienced a BMI decrease of more than 5%, with a mean reduction in BMI of 17.2%, at 16 weeks on setmelanotide medication, according to Rhythm's announcement of positive interim trial results in July 2022.
