Novelion’s subsidiary seeks European approval for metreleptin

Metreleptin was granted orphan drug designation by the European Commission in July 2012, and if approved, carries 10 years of exclusivity in the EU.

The company estimates the prevalence of GL is approximately one in one million people, and the prevalence of the subset of PL being targeted for approval is approximately one half to one in one million people. The company, through its subsidiary, will seek to market metreleptin in the EU under the tradename MYALEPTA.

Novelion CEO Mary Szela said: “There are a meaningful number patients in Europe that could benefit from metreleptin based upon the proposed indications and, if approved by EMA and reimbursement approvals in key markets are secured, we look forward to the opportunity to bring this important therapy to these patients.

“In addition, currently there are more than 100 patients on therapy via a pre-approval compassionate use program in certain markets in Europe, the majority of which are within the proposed indications.

“In addition to our efforts to target GL and a subset of patients with PL, we are continuing to focus on the potential of developing metreleptin for a variety of indications across a range of rare and metabolic diseases associated with low leptin over the coming years.”

Novelion Therapeutics EVP, research and development head John Orloff, M.D. said: “The application seeking approval for MYALEPTA in Europe is an important step in advancing our commitment to provide breakthrough therapies for patients with rare diseases.

“There have been limited options for treating complications of these rare forms of lipodystrophy, and we look forward to collaborating with regulatory authorities to bring forward this much needed treatment for adult and pediatric GL and a subset of PL patients affected by these serious, rare diseases.”

Generalized lipodystrophy (GL) and partial lipodystrophy (PL) are ultra-rare disorders characterized by loss of adipose tissue. Because the hormone leptin is made by fat tissue, patients with GL and PL have low levels of leptin, which regulates food intake and energy balance.

Both GL and PL are associated with severe metabolic abnormalities, including hypertriglyceridemia, insulin resistance, and diabetes, which can result in life-threatening co-morbidities such as acute pancreatitis, inflammation and fatty deposits in the liver (steatohepatitis), and accelerated plaque accumulation in the arteries (atherosclerosis).

Leptin is a naturally occurring hormone and an important regulator of energy homeostasis, fat and glucose metabolism. Metreleptin is an analog of leptin made through recombinant DNA technology and has been studied as a replacement therapy, in addition to diet, to treat the complications of leptin deficiency in patients with generalized or partial lipodystrophy.

Open-label studies with metreleptin showed significant reductions in HbA1c (a measure of blood sugar control), fasting glucose, and triglycerides. In the U.S., metreleptin is approved under the trade name MYALEPT for the treatment of generalized lipodystrophy, and carries a boxed warning for the risks of anti-metreleptin antibodies with neutralizing activities and lymphoma.

It is available only through the MYALEPT Risk Evaluation and Mitigation Strategy (REMS) program.

David Araújo-Vilar, M.D., Ph.D. of the Universidade de Santiago de Compostela, Spain said: “Patients with generalized and partial forms of lipodystrophy have complex medical conditions with serious consequences. The options physicians have had to treat them have been significantly limited.

“We look forward to the possibility of new options becoming available to advance the care of these patients.”

MYALEPT (metreleptin for injection) is a recombinant human leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.