Treatment for acquired hypothalamic obesity now available in Japan
Imcivree was approved by country's regulatory agency in August
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Imcivree is now commercially available in Japan for people with acquired hypothalamic obesity, becoming the first treatment available for this rare condition in the country.
Imcivree (setmelanotide) is now commercially available in Japan for people with acquired hypothalamic obesity (aHO), becoming the first treatment available for this rare condition in the country.
The launch follows the therapy’s approval by the country’s regulatory agency in August and its subsequent inclusion on the country’s National Health Insurance drug price list, which allows patients to pay only a small percentage of the medication cost.
“This launch marks an important advancement for people living with acquired HO in Japan, where there were no approved therapies for this disease until now,” Yann Mazabraud, executive vice president and head of international of Rhythm Pharmaceuticals, the therapy’s developer, said in a company press release. “We have been building our team in Japan in preparation for this launch and are excited about the opportunity to positively impact the lives of Japanese patients.”
Imcivree previously received orphan drug designation for treating aHO in Japan, where 5,000 to 8,000 people are estimated to live with the disease, according to Rhythm. Orphan drug designation is intended to accelerate the development of treatments for rare diseases by providing certain financial and regulatory incentives.
The therapy is also approved for people with aHO in the U.S., the European Union, the U.K., and Canada. It is also cleared for use to treat certain genetic forms of obesity, including Bardet-Biedl syndrome.
Imcivree activates key protein
aHO is caused by damage to the hypothalamus, a region of the brain that helps regulate hunger, appetite, and energy balance. This can result from a brain tumor, surgery, radiation treatment, or other injury.
As a result, people with aHO often experience excessive, difficult-to-control hunger, along with rapid and substantial weight gain. Because the condition disrupts the body’s mechanisms for regulating hunger and weight, conventional strategies such as diet and exercise are often insufficient.
Imcivree works by activating the melanocortin-4 receptor (MC4R), a protein involved in the brain’s signaling pathways that regulate hunger and energy balance. By restoring activity of this pathway, the treatment aims to reduce excessive hunger and promote weight loss.
The treatment’s approval was supported by results from the Phase 3 TRANSCEND clinical trial (NCT05774756), which enrolled 142 patients with aHO, ages 4 and older. These included 120 patients from the main study group, 12 participants from a Japanese study group, and 10 additional participants.
Participants received daily under-the-skin injections of either Imcivree or a placebo for one year. The trial met its primary goal, with Imcivree resulting in a 19.8% greater reduction in body mass index (BMI) than the placebo. BMI is calculated using a person’s weight and height and is used as a proxy for body fat.
A significantly higher proportion of participants receiving Imcivree achieved a clinically meaningful BMI reduction of at least 5% compared with those on the placebo (83% vs. 21%). The treatment was also associated with a significantly greater reduction in hunger scores, with average weekly hunger scores decreasing by 2.73 points with Imcivree, compared with 1.45 points with the placebo.
The company is also developing other treatments targeting the MC4R pathway, including RM-718, an injectable version for once-weekly dosing, and bivamelagon, an oral formulation to be taken daily. Clinical trial data so far in people with aHO have shown reductions in BMI with both treatments, while bivamelagon was also associated with reduced hunger.
