VANCOUVER, British Columbia, Jan. 23, 2017 (GLOBE NEWSWIRE) -- Novelion Therapeutics Inc. (NASDAQ:NVLN) (TSX:NVLN), a biopharmaceutical company dedicated to developing new standards of care for individuals living with rare diseases, today 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.
U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION
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. LIMITATIONS OF USE: 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. For more detailed information, please see additional Important Safety Information and the Prescribing Information for MYALEPT.
Forward Looking Statements:
Certain statements in this press release constitute “forward-looking statements” and constitute “forward-looking information” within the meaning of applicable securities laws, including statements regarding the approval, orphan drug exclusivity and the market potential of metreleptin in the EU, and the prevalence of the proposed metreleptin indications. Forward-looking statements are based on estimates and assumptions made by Novelion in light of current conditions and expected future developments, as well as other factors that Novelion believes are appropriate in the circumstances, including but not limited to, our financial position and execution of our business strategy, receipt of regulatory approvals, indication prevalence, and product competition, market acceptance, sales, pricing, reimbursement and side effects. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond our control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. Many such risks, uncertainties and other factors are taken into account as part of our assumptions underlying these forward-looking statements and include, among others, the following: the risks that metreleptin may not be approved by the EMA at all or for the desired indications; that, if approved, metreleptin may not obtain reimbursement approvals on a timely basis or at all or at the levels necessary to support commercialization; that the prevalence estimates for GL and a subset of PL may be inaccurate; the risk that orphan drug, data and marketing exclusivity or the patent protection for metreleptin may not be sufficient or may be invalidated; and the other risks common to seeking regulatory approval for, developing and commercializing orphan drugs.
For additional disclosure regarding these and other risks we face, see the disclosure contained in the "Risk Factors" section of Aegerion's Quarterly Report on Form 10-Q filed on November 4, 2016, Novelion’s Annual Report on Form 10-K filed on February 25, 2016 (and amended on April 29, 2016) and Quarterly Report on Form 10-Q filed on November 1, 2016 and each company’s other public filings with the SEC, available on the SEC's website at www.sec.gov. Except as required by law, we undertake no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.
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