Novelion Therapeutics Inc., a biopharmaceutical company, announced that its subsidiary has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) seeking approval for metreleptin as replacement therapy to treat complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (GL) and in a subset of patients with partial lipodystrophy (PL).
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.
Mary Szela, chief executive officer of Novelion 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."
"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," said John Orloff, M.D., EVP, head of research and development for Novelion Therapeutics. "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 paediatric 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 US, metreleptin is approved under the trade name Myalepta 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 Myalepta Risk Evaluation and Mitigation Strategy (REMS) programme.
"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," said David Araújo-Vilar, M.D., Ph.D., Universidade de Santiago de Compostela, Spain. "We look forward to the possibility of new options becoming available to advance the care of these patients."
Novelion Therapeutics is a biopharmaceutical company dedicated to developing new standards of care for individuals living with rare diseases.