Rare Daily Staff

The European Commission granted approval to Novelion Therapeutics’ Myalepta as a replacement therapy to treat the complications of leptin deficiency with certain forms of lipodystrophy who have failed to achieve adequate metabolic control with standard treatment.

The EC granted approval of Myalepta as an adjunct to diet for patients with leptin deficiency as a result of confirmed congenital generalized lipodystrophy, (Berardinelli-Seip syndrome) or acquired generalized lipodystrophy (Lawrence syndrome) in adults and children 2 years of age and above; or with confirmed familial partial lipodystrophy or acquired partial lipodystrophy (Barraquer-Simons syndrome).

Lipodystrophy syndromes are ultra-rare disorders characterized by the irreversible loss of adipose tissue. Lipodystrophy can be genetic or acquired after an immune condition and occurs in childhood or adolescence. In patients with lipodystrophy syndromes, levels of leptin are often very low. Leptin is a naturally occurring hormone produced in adipose tissue and is an important regulator of energy homoeostasis, fat and glucose metabolism, reproductive capacity, and other diverse physiological functions.

Myalepta, a recombinant analog of human leptin, is the first and only licensed treatment in Europe indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy patients. Myalepta won orphan drug designation in Europe in 2012. The U.S. Food and Drug Administration approved Myalepta in February 2014 as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired general lipodystrophy, but it is not approved in the U.S. for partial lipodystrophy.

The company said that pricing and reimbursement negotiations with healthcare authorities have commenced and will be pursued on a country-by-country basis.

“The approval of Myalepta in the EU is a significant advancement for patients with generalized and partial lipodystrophy and a significant milestone for our company,” said Jeff Hackman, interim chief executive officer and chief operating officer Novelion. “In preparation for launch, we have worked closely with regulatory authorities, patient organizations, and healthcare professionals across Europe to ensure that generalized LD and partial LD patients in need have access to Myalepta as quickly as possible, subject to securing pricing and reimbursement approvals where required.”

August 1, 2018
Photo: Jeff Hackman, interim CEO of Novelion