EMA has recommended granting a marketing authorisation in the European Union (EU) for Imcivree (setmelanotide) to support weight loss and -management in patients from 6 years of age with obesity caused by the following rare genetic disorders: pro-opiomelanocortin (POMC) deficiency – including proprotein convertase subtilisin/kexin type 1 (PCSK1) deficiency – and leptin receptor (LEPR) deficiency in adults and children. These conditions must be confirmed by genetic testing before a patient can start treatment with Imcivree.

POMC, PCSK1 or LEPR deficiency affect the melanocortin 4 receptor (MC4R) pathway which is responsible for transmission of signals about when to eat and when to stop eating. Patients with these deficiencies continuously feel hungry and become severely obese early in life – often beginning in infancy. These conditions are extremely rare. Fewer than 50 patients with POMC deficiency, 90 with LEPR deficiency and 50 with PCSK1 deficiency have been reported worldwide thus far.

Setmelanotide, the active substance contained in Imcivree, works by activating a pathway in the brain that controls appetite and feeling full. This helps patients eating less and losing weight.

The opinion of EMA’s human medicines committee (CHMP) is mainly based on two studies which evaluated 21 participants. During the course of the studies 80% of POMC patients and 45.5% of LEPR patients achieved at least 10% weight loss after approximately one year of treatment.

The most common side effects observed in clinical trials were reactions at the site of the injection, darkening of the skin, nausea and headache.

The opinion adopted by the CHMP is an intermediary step on Imcivree’s path to patient access. The recommendation will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.

Notes: 

  1. The applicant for Imcivree is Rhythm Pharmaceuticals Limited from Ireland.
  2. Imcivree was granted eligibility to PRIME on 28 June 2018 for treatment of obesity and control of hunger associated with deficiency disorders of the MC4R pathway.
  3. Imcivree was designated as an orphan medicinal product on 14 July 2016.
  4. Following this positive CHMP opinion, the Committee for Orphan Medicinal Products (COMP) will assess whether the orphan designation should be maintained.

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