Novelion Therapeutics Inc., a biopharmaceutical company dedicated to developing new standards of care for individuals living with rare diseases, announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the granting of marketing authorization, under exceptional circumstances, for Myalepta.
The CHMP recommends granting marketing authorization for Myalepta, as an adjunct to diet, as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients with confirmed congenital generalized LD (Berardinelli-Seip syndrome); or acquired generalized LD (Lawrence syndrome) in adults and children 2 years of age and above; or 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.
The CHMP's positive opinion will now be reviewed by the European Commission (EC), which has the authority to grant approval of this indication. The company anticipates a decision in mid-2018. If approved by the EC, Myalepta, which has orphan drug designation in Europe, will be the first and only licensed treatment in the EU as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in LD patients.
Murray Stewart, DM, FCRP, executive vice president, head of research & development said, "Lipodystrophy is a serious, rare condition, characterized by loss of fat tissue. In some patients it is inherited, while in others it may be acquired. This loss of fat tissue causes a deficit in the hormone leptin leading to possible metabolic complications such as hypertriglyceridemia, insulin resistance and hyperglycemia. This decision by the CHMP is welcomed by the physician community who is in need of treatment options for its generalized and partial lipodystrophy patients."
Jeffrey Hackman, chief operating officer said, "The positive CHMP opinion is a substantial milestone for our company, and even more importantly, for generalized and partial lipodystrophy patients. Europe is a significant market with a meaningful number of generalized and partial lipodystrophy patients already being treated through the metreleptin early access programme. Assuming EC approval, we look forward to the opportunity to work with physicians, payers and patient organizations to convert these patients to commercial patients and to reach even more generalized and partial lipodystrophy patients in need."
In accordance with the receipt of a marketing authorization under exceptional circumstances, certain risk minimization measures and post-authorization obligations will be required, including proposed studies which will further the understanding of Myalepta's impact on patients with generalized and partial lipodystrophy.
LD syndromes are ultra-rare disorders characterized by the irreversible loss of adipose tissue. 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.
With generalized lipodystrophy, the loss of fat affects the whole body. With partial lipodystrophy, the loss of fat typically occurs in the arms, legs, head, and trunk regions, while accumulation of fat may occur in other areas of the body, including the neck, face, and intra-abdominal regions.
Myalept, the US brand name of metreleptin, is approved in the US to treat generalized lipodystrophy, but is not approved to treat partial lipodystrophy.