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news.The European Medicines Agency (EMA) has accepted for review Novelion Therapeutics' 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).
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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.
Novelion Therapeutics executive vice president, research and development head John Orloff said: "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.
"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.