The last we heard of Rhythm Pharmaceuticals they were modifying their drug treatment for weight loss following feedback from the FDA. This seemed quite the setback at the time. However, it appears they’ve been successful. Not just once, but twice.
Rhythm Pharmaceuticals has announces FDA Approval of IMCIVREE® (setmelanotide) for use in patients with Bardet-Biedl Syndrome.
Bardet-Biedl Syndrome is something we’ve touched on very briefly as it’s among one of many others that is also affected by the MC4r receptor. It is a rare genetic disorder with highly variable symptoms which may include retinal degeneration, obesity, reduced kidney function, polydactyly (extra digits of the hands or feet) among other features.
This is the first FDA-approved therapy that targets impairment in the MC4R pathway – a root cause of early-onset, severe obesity and hyperphagia associated with Bardet-Biedl syndrome.
IMCIVREE is now available for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to Bardet-Biedl syndrome.
FDA approval represents significant milestones not only for Rhythm Pharmaceuticals but for everyone associated with MC4r.
Off the back of the approval for IMCIVREE Rhythm Pharmaceuticals we’re able to put a strategy in place for people with hyperphagia (an abnormally great desire for food; excessive eating) and severe obesity caused by rare MC4R-pathway diseases and is now available for patients in Germany; after receiving marketing authorization from the European Commission last year.
The German Federal Joint Committee (G-BA) earlier this year voted to exclude IMCIVREE from its lifestyle exemption list for POMC or LEPR deficiency obesity. In Germany, drugs classified as lifestyle drugs such as those that are designed to help hair loss, weight loss or help people stop smoking are not eligible for reimbursement.
IMCIVREE, which is the first precision medicine designed to induce weight loss and control of hunger has receive an exemption and is now eligible for national coverage and reimbursement.
Rhythm Pharmaceuticals have had a good few weeks. I’m excited for them, I’m excited for the MC4r community. When I was diagnosed there was little to no hope of a treatment and now, there is a drug on the market to help people. Yes, it’s still early days but I’m so happy for the people whose lives will change for the better. Weight can have such a big impact on a persons mental health and for the people who need it, this is a light at the end of the tunnel.