The U.S. Food and Drug Administration (FDA) has approved Rhythm Pharmaceuticals’ RYTM supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide), a melanocortin-4 receptor (MC4R) agonist, for patients with Bardet-Biedl syndrome (BBS).
The regulatory approval was supported by data readout from pivotal Phase 3 clinical trial, which was the largest and longest interventional clinical trial in BBS, the trial met its primary endpoint and all key secondary endpoints.
David Meeker, M.D., Chair, President and Chief Executive Officer, stated, "This FDA approval represents a significant milestone for Rhythm, validating our strategy of developing IMCIVREE for people with hyperphagia and severe obesity caused by rare MC4R-pathway diseases and allowing us to provide our precision therapy to an established community of patients living with BBS and their families who are eagerly awaiting a new treatment option.”
Bardet-Biedl syndrome (BBS) is a rare genetic disease, that generates insatiable hunger, also known as hyperphagia, and severe obesity beginning early in life.
Rhythm also announced that the regulatory agency has issued a complete response letter (CRL) for the supplemental New Drug Application (sNDA) for setmelanotide in Alström syndrome. The company plans to reevaluate potential paths forward in Alström syndrome in the United States.
Commenting on the CRL issued by the FDA, Dr. Meeker, said, "We appreciate the FDA's careful review of our sNDA for IMCIVREE for the treatment of Alström syndrome, but are disappointed in this outcome, We are tremendously grateful to the Alström syndrome patients, caregivers and physicians who participated in our clinical development efforts and look forward to providing an update regarding our path forward.”
Rhythm Pharmaceuticals shares are trading down 10 percent at $3.51 in the after hours session.
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