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Novelion Therapeutics, a biopharmaceutical company dedicated to developing new standards of care for individuals living with rare diseases, has announced that the European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) 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). The MAA was filed in December 2016. An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in December of 2017, and decision is expected in the first quarter of 2018.
"The MAA filing acceptance marks important progress in our priority initiative to deliver Myalepta to adult and pediatric GL and a subset of PL patients in Europe," said John Orloff, M.D., executive vice president, head of research and development for Novelion Therapeutics. "There are more than 100 patients on therapy via a pre-approval compassionate use program in certain markets in Europe, the majority of whom are within the proposed indications. Additionally, there are a meaningful number of patients who currently have limited options for treating complications of these rare forms of lipodystrophy. We look forward to working with European regulatory authorities with the objective of achieving marketing authorization and bringing this needed therapy to patients."
Metreleptin was granted orphan drug designation by the European Commission in July 2012, and, if approved, will carry 10 years of orphan drug 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 a subsidiary, will seek to market metreleptin in the EU, if approved, under the tradename Myalepta.
Myalept (metreleptin) for injection is a 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. The safety and effectiveness of Myalept for the treatment of complications of partial lipodystrophy or for the treatment of liver disease, including nonalcoholic steatohepatitis (NASH), have not been established.
Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with Myalept. T-cell lymphoma has been reported in patients with acquired generalized lipodystrophy, both treated and not treated with Myalept.
Ema accepts novelion, therapeutics, maa for metreleptin
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