3 The technology


Metreleptin (Myalepta, Amryt) is an analogue of the human hormone leptin, which is secreted into the circulation from adipocytes. Metreleptin has a UK marketing authorisation under 'exceptional circumstances' as 'an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients:

  • with confirmed congenital generalised LD (Berardinelli–Seip syndrome) or acquired generalised LD (Lawrence syndrome) in adults and children 2 years of age and above

  • with confirmed familial partial LD or acquired partial LD (Barraquer–Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control'.


Metreleptin is administered daily by subcutaneous injection. For people weighing 40 kg or less, the starting daily dose is 0.06 mg/kg. Dose adjustments of 0.02 mg/kg are allowed up to a maximum daily dose of 0.13 mg/kg. For men and women weighing over 40 kg, the starting daily dose is 2.5 mg and 5 mg respectively. For people weighing over 40 kg, dose adjustments of 1.25 mg to 2.5 mg are allowed up to a maximum daily dose of 10 mg.


The most common treatment-emergent adverse events in metreleptin studies included weight loss, hypoglycaemia, fatigue, injection site reactions, neutralising antibodies, decreased appetite, nausea, headache, abdominal pain, menorrhagia and alopecia. For full details of adverse reactions and contraindications, see the summary of product characteristics.


The price of metreleptin per 11.3 mg vial (up to a 10 mg dose) is £2,335.00; per 5.8 mg vial (up to a 5 mg dose) is £1,167.50 and per 3 mg vial (up to a 2.8 mg dose) is £583.75 (excluding VAT; company's evidence submission). The company has a commercial arrangement (simple discount patient access scheme). This makes metreleptin available to the NHS with a discount. The size of the discount is commercial in confidence. It is the company's responsibility to let relevant NHS organisations know details of the discount.

  • National Institute for Health and Care Excellence (NICE)