New hope for European patients with ultra-rare disease

US biotech firm Aegerion Pharmaceuticals has won European approval for Myalepta (metreleptin), a treatment for an ultra-rare condition called lipodystrophy, becoming the first and only licensed medication to treat the underlying problem of leptin deficiency.

The therapy has been approved as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy patients. Globally, lipodystrophy affects approximately between 1 and 4 people per million.

Stephen O’Rahilly, at Addenbrooke’s Hospital in Cambridge, UK, said: “Patients with lipodystrophy have previously relied on lifestyle changes and medications, like insulin injections to manage the condition’s associated complications.”