NICE approves lipid disorder drug Myalepta for NHS use

pharmafile | January 20, 2021 | News story | Research and Development Amryt Pharma, NICE 

NICE has approved Amryt’s rare lipid disorder drug Myalepta (metreleptin), currently the only leptin replacement therapy available, for use within the NHS.

The drug will be used as an adjunct to diet as replacement therapy for the treatment of leptin deficiency in patients with congenital or acquired general lipodystrophy (GL) in adults and children two years of age and above, and familial or acquired partial lipodystrophy (PL) in adults and children 12 years of age and above.

GL and PL are rare disorders that affect how the body stores and uses fat. People with lipodystrophy may have little or no body fat, and can suffer from serious metabolic abnormalities including severe insulin resistance, diabetes, hypertriglyceridemia, and fatty liver disease. Lipodystrophy can be inherited, or acquired without a known genetic cause.

Responding to the approval, Rebecca Sanders, CEO of Lipodystrophy UK, commented: “Lipodystrophy UK are ecstatic that NICE has approved metreleptin for the treatment of partial and generalised lipodystrophy. We have worked tirelessly with all stakeholders involved in this appraisal to ensure the voice of patients and their carers was heard.

“We are grateful to the clinical experts at the National Specialist Treatment Centre at Addenbrooke’s Hospital, Amryt, and NICE for their hard work and collaborative spirit throughout the appraisal. Lipodystrophy UK will continue to advocate for our community to ensure better treatment and care for everyone affected by lipodystrophy.”

Professor Sir Stephen O’Rahilly, Director of the MRC Metabolic Diseases Unit at the University of Cambridge, and Honorary Consultant Physician at Addenbrooke’s Hospital in Cambridge, also said: “Patients with lipodystrophy can suffer severe metabolic complications as a result of being deficient in the fat cell hormone, leptin. I am delighted that metreleptin, currently the only form of leptin replacement therapy, will now be available as a treatment for appropriate NHS patients with lipodystrophy.

“The National Severe Insulin Resistance Service at Addenbrooke’s Hospital, Cambridge, where many of these patients are supported, has worked tirelessly with Amryt and NICE to assist them in reaching a fair and equitable solution to providing this medication to the patients who need it. Without the commitment and effort of people with lipodystrophy, working through their support group Lipodystrophy UK, none of this would have been possible. Today is a vindication of all their hard work.”

Darcy Jimenez

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