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US FDA Expands Breakthrough Therapy Designation to Rhythm's Setmelanotide.

M2 PHARMA-May 15, 2017-US FDA Expands Breakthrough Therapy Designation to Rhythm's Setmelanotide

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- The US Food and Drug Administration has expanded a previously granted Breakthrough Therapy Designation to US-based biopharmaceutical company Rhythm's for setmelanotide novel melanocortin-4 receptor agonist, the company said.

According to the company, this expanded BTD is for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway (the MC4 pathway), which includes both pro-opiomelanocortin and leptin receptor deficiency obesity.

The FDA had previously granted BTD to setmelanotide for the treatment of POMC deficiency obesity.

Rhythm is developing setmelanotide for the treatment of obesity caused by genetic deficiencies in the MC4 pathway, a key biological pathway in humans that regulates weight by increasing energy expenditure and reducing appetite.

The company has completed positive Phase 2 clinical trials in both POMC deficiency obesity and LepR deficiency obesity in which patients treated with setmelanotide experienced significant weight loss and substantial reductions in hunger. Setmelanotide was well tolerated in these trials.

Rhythm recently initiated a Phase 3 clinical trial of setmelanotide in POMC deficiency obesity.

The FDA grants BTD to expedite the development and review of therapeutics to treat serious or life-threatening conditions for which preliminary clinical evidence indicates that the therapy may demonstrate substantial improvement on at least one clinically significant endpoint over existing therapies.

This designation conveys all FDA Fast Track program features, such as eligibility for rolling new drug application submissions and priority review (if supported by clinical data at the time of NDA).

Additionally, this designation provides more intensive involvement of FDA staff in a proactive, collaborative, cross-disciplinary review process.

Setmelanotide is a potent, first-in-class MC4R agonist in development for the treatment of obesity caused by genetic deficiencies in the MC4 pathway, a key biological pathway in humans that regulates weight by increasing energy expenditure and reducing appetite.

The critical role of the MC4 pathway in weight regulation was validated with the discovery that single genetic defects along this pathway result in early-onset and severe obesity.

In 2016, The New England Journal of Medicine reported results from a setmelanotide Phase 2 trial in POMC deficiency obesity that demonstrated substantial weight loss in two adult patients, the company said.

Both POMC and LepR deficiency obesity are rare genetic disorders associated with severe, early-onset obesity and unrelenting hyperphagia.

Rhythm recently initiated a Phase 3 clinical trial of setmelanotide in POMC deficiency obesity.

The company is focused on developing peptide therapeutics for the treatment of rare genetic deficiencies that result in life-threatening metabolic disorders.

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Publication:M2 Pharma
Date:May 15, 2017
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