FDA gives Rhythm the green light for setmelanotide, a drug aimed at reducing obesity in certain genetic disorders
A little over a year after completing successful pivotal trials, Rhythm Pharmaceuticals $RYTM has its first drug approval on its hands.
The Boston-based biotech announced Friday that the FDA gave setmelanotide the thumbs-up for three rare genetic disorders that result in obesity in patients six and older. It’s the agency’s first such approval, Rhythm said, with the indicated deficiencies being the POMC, PCSK1 and LEPR genes. Rhythm will market the drug as Imcivree, and plans to have it on the shelves in the first quarter of 2021.
Investors embraced the news as Rhythm shares shot up more than 20% in Friday’s half-day of trading.
Rhythm highlighted that genetic testing is required to confirm the genetic disorders and the drug is not indicated for suspected or benign variant cases of POMC, PCSK1, or LEPR deficiency. It’s also not approved for other obesity-related genetic disorders or general obesity.
Setmelanotide, given by daily injection, is an MC4 receptor agonist, aiming to regulate a pathway that controls hunger, energy expenditure and consequently body weight. The drug is designed to restore impaired upstream activity that arises from genetic diseases. Patients with the disorder often show signs at a young age, struggling with extreme hunger that results in obesity.
“To manage this obesity and control disruptive food-seeking behavior, caregivers often lock cabinets and refrigerators and significantly limit social activities,” said Jennifer Miller in a statement, a pediatric endocrinologist involved with the trials. “This FDA approval marks an important turning point, providing a much-needed therapy and supporting the use of genetic testing.”
Friday’s approval was based on two Phase III trials, one studying POMC and PCSK1 deficiencies and the other looking at LEPR deficiency. In the former, 8 of 10 patients saw greater than 10% weight loss, and the average reduction in body weight was 25.4%. For the LEPR study, 5 of 11 patients saw greater than 10% weight loss after a year of treatment, with the average reduction clocking in at 12.5%.
Though those numbers are small for pivotal trials in general, Rhythm said they were the largest conducted to date in these specific genetic disorders. The trials were designed to include a four-week placebo withdrawal period after 12 weeks of treatment to evaluate whether patients would revert back to their original state. Patients’ on average gained back 11 pounds during the withdrawal period, but once restarting on the drug, Rhythm said at the time the trends reversed.
Imcivree has helped Rhythm chart a steady course since it was founded as a private company back in 2008. The company ultimately went public in 2017 after the drug posted positive Phase II data a year earlier, and now with the approval in hand Rhythm is shooting for the green light in Europe.
The company is currently conducting another Phase III for the drug in Bardet-Biedl or Alström syndrome, with topline data expected in early 2021 at the latest. Rhythm was also issued a priority review voucher and has not stated their plans for it as of yet.