Policy on Expanded Access to Investigational Drugs
Rhythm is committed to developing safe and effective therapies for the treatment of rare melanocortin-4 receptor (MC4R) pathway diseases. Setmelanotide is an investigational MC4R agonist being evaluated in clinical trials for the treatment of obesity and insatiable hunger or hyperphagia associated with MC4R pathway diseases.
We believe the most appropriate way to deliver on our commitment to patients is through participation in our clinical trials. Clinical trials, and the subsequent approval of a therapy, provide the best way to provide long-term access to as many patients as possible. If you have questions, we encourage you to speak with your physician and potentially participate in one of Rhythm’s clinical trials. You can learn more about our clinical trials on our website or www.clinicaltrials.gov
Expanded access program for setmelanotide
Expanded access is the use of an investigational therapy to treat patients who are unable to participate in clinical trials and have serious or immediately life-threatening diseases or conditions that have no comparable or satisfactory alternative treatment options. Following the completion of a Phase 3 clinical trial meeting key clinical endpoints, Rhythm may consider making setmelanotide available through potential expanded access programs (EAP). If a treating physician believes setmelanotide, through the EAP, may be the only therapeutic option for a patient, the physician can request additional information about the EAP by emailing:
ExpandedAccess@rhythmtx.com. Rhythm will acknowledge receipt of such requests within five (5) business days.
While Rhythm has ongoing clinical trials to evaluate setmelanotide in several additional diseases of obesity, we recognize that not all patients will be eligible for these clinical trials or our expanded access program. As more information and late-stage clinical data on the safety and efficacy of our investigational therapies become available, we will review our policy on expanded access regularly.