Amryt Pharma PLC (LON:AMYT) (NASDAQ:AMYT) noted that support groups for people suffering from the metabolic leptin deficiency disorder lipodystrophy said they were 'ecstatic' after its treatment for the condition, Myalepta was approved for the use by the NHS.
NICE, the UK’s pharma regulator, has approved Myalepta for reimbursement as an adjunct to diet as replacement therapy for two forms of the condition, generalised (GL) and partial lipodystrophy (PL).
In a statement, Joe Wiley, Amryt Pharma’s chief executive, said: “We are very excited to report NICE’s approval which will now give all England and Wales based patients suffering from lipodystrophy the opportunity to access Myalepta.
“Today’s news represents further progress in our efforts to grow the geographic reach of our commercial products in both existing and new territories.”
Professor Sir Stephen O'Rahilly, a consultant physician, at Addenbrooke's Hospital, Cambridge commented: “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 [Myalepta], currently the only form of leptin replacement therapy, will now be available as a treatment for appropriate NHS patients with lipodystrophy."
Rebecca Sanders, chief executive of Lipodystrophy UK, added: “Lipodystrophy UK are ecstatic that NICE has approved metreleptin for the treatment of partial and generalised lipodystrophy."
Generalised (GL) and partial lipodystrophy (PL) are rare disorders characterised by loss or lack of adipose tissue resulting in the deficiency of the hormone leptin, produced by fat cells.
The disorders are associated with severe metabolic abnormalities including severe insulin resistance, diabetes, hypertriglyceridemia and fatty liver disease.