MYALEPTA® (metreleptin) Receives Positive CHMP Opinion in Patients with Generalized and Partial Lipodystrophy
Europe represents potentially the largest market for metreleptin with expected authorization in both generalized and partial lipodystrophy
VANCOUVER, British Columbia, June 01, 2018 (GLOBE NEWSWIRE) -- Novelion Therapeutics Inc. (NASDAQ:NVLN), a biopharmaceutical company dedicated to developing new standards of care for individuals living with rare diseases, today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the granting of marketing authorization, under exceptional circumstances, for MYALEPTA.
The CHMP recommends granting marketing authorization for MYALEPTA, as an adjunct to diet, as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients with confirmed congenital generalized LD (Berardinelli-Seip syndrome); or acquired generalized LD (Lawrence syndrome) in adults and children 2 years of age and above; or with confirmed familial partial LD or acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control.
The CHMP's positive opinion will now be reviewed by the European Commission (EC), which has the authority to grant approval of this indication. The Company anticipates a decision in mid-2018. If approved by the EC, MYALEPTA, which has orphan drug designation in Europe, will be the first and only licensed treatment in the EU as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in LD patients.
Murray Stewart, DM, FCRP, Executive Vice President, Head of Research & Development said, "Lipodystrophy is a serious, rare condition, characterized by loss of fat tissue. In some patients it is inherited, while in others it may be acquired. This loss of fat tissue causes a deficit in the hormone leptin leading to possible metabolic complications such as hypertriglyceridemia, insulin resistance and hyperglycemia. This decision by the CHMP is welcomed by the physician community who is in need of treatment options for its generalized and partial lipodystrophy patients."
Chief Operating Officer Jeffrey Hackman said, "The positive CHMP opinion is a substantial milestone for our company, and even more importantly, for generalized and partial lipodystrophy patients. Europe is a significant market with a meaningful number of generalized and partial lipodystrophy patients already being treated through the metreleptin early access program. Assuming EC approval, we look forward to the opportunity to work with physicians, payers and patient organizations to convert these patients to commercial patients and to reach even more generalized and partial lipodystrophy patients in need."
In accordance with the receipt of a marketing authorization under exceptional circumstances, certain risk minimization measures and post-authorization obligations will be required, including proposed studies which will further the understanding of MYALEPTA's impact on patients with generalized and partial lipodystrophy.
LD syndromes are ultra-rare disorders characterized by the irreversible loss of adipose tissue. In patients with lipodystrophy syndromes, levels of leptin are often very low. Leptin is a naturally occurring hormone produced in adipose tissue and is an important regulator of energy homoeostasis, fat and glucose metabolism, reproductive capacity, and other diverse physiological functions.
With generalized lipodystrophy, the loss of fat affects the whole body. With partial lipodystrophy, the loss of fat typically occurs in the arms, legs, head, and trunk regions, while accumulation of fat may occur in other areas of the body, including the neck, face, and intra-abdominal regions.
MYALEPT, the U.S. brand name of metreleptin, is approved in the U.S. to treat generalized lipodystrophy, but is not approved to treat partial lipodystrophy.
FOR IMPORTANT SAFETY INFORMATION, INCLUDING BOXED WARNING IN THE U.S., PLEASE VISIT WWW.MYALEPT.COM
About Novelion Therapeutics
Novelion Therapeutics is a biopharmaceutical company dedicated to developing new standards of care for individuals living with rare diseases. The company seeks to advance its portfolio of rare disease therapies by investing in science and clinical development. Novelion has a commercial portfolio of products through its indirect subsidiary, Aegerion Pharmaceuticals, Inc.
Certain statements in this press release constitute "forward-looking statements" of Novelion within the meaning of applicable laws and regulations and constitute "forward-looking information" within the meaning of applicable securities laws. Any statements contained herein which do not describe historical facts, including statements regarding expectations and beliefs about the EC's approval decision, including timing, the size of the market for MYALEPTA in the EU, and converting early access patients to commercial patients and adding new MYALEPTA patients in the EU, are forward-looking statements which involve risks and uncertainties that could cause actual results to differ materially from those discussed in such forward-looking statements. Such risks and uncertainties include, among others, those risks identified in our filings with the U.S. Securities and Exchange Commission (the "SEC"), including under the heading "Risk Factors" in our Annual Report on Form 10-K filed on March 16, 2018, and subsequent filings, with the SEC, available on the SEC's website at www.sec.gov. Any such risks and uncertainties could materially and adversely affect our results of operations and cash flows, which would, in turn, have a significant and adverse impact on our stock price. We caution you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. 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.
Investors and others should note that we communicate with our investors and the public using our company website, www.novelion.com, including, but not limited to, company disclosures, investor presentations and FAQs, SEC filings, press releases, public conference call transcripts and webcast transcripts. The information that we post on this website could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.
Amanda Murphy, Director, Investor Relations & Corporate Communications
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Novelion Therapeutics Inc. via Globenewswire
Följ NASDAQ OMX
Abonnera på våra pressmeddelanden.
Senaste pressmeddelandena från NASDAQ OMX
CORRECTING and REPLACING -- PRESS ALERT & INVITATION: Arbitrade to Hold a Press Conference on June 28th From Bermuda - Its New Global Headquarters23.6.2018 14:00 | Pressmeddelande
NEW YORK, June 23, 2018 (GLOBE NEWSWIRE) -- In a press release issued under the same headline earlier today by Arbitrade, please note that the correct date of the event is Thursday, June 28, 2018 at 8:00 a.m. EDT. The corrected release follows: This is an invitation to all media following Arbitrade and/or covering the cryptocurrency markets. You are invited to join Arbitrade's Chairman, Len Schutzman, and management who will discuss their progress toward establishing Arbitrade as a world-class cryptocurrency exchange and coin company. They will also discuss their move and incorporation into Bermuda, one of the world's best regulated jurisdictions and a platform from which Arbitrade will reach out to several international markets. It will be a lively discussion that will also include details of the many economic and social advantages and new jobs that companies, like Arbitrade, will bring to countries in which they settle and do business around the world. Details of how to access the pr
15th Consecutive Nasdaq Closing Cross Sets Record for Number of Shares Traded During 2018 Russell US Indexes Reconstitution22.6.2018 23:19 | Pressmeddelande
NEW YORK, June 22, 2018 (GLOBE NEWSWIRE) -- Nasdaq (Nasdaq:NDAQ) today announced the Nasdaq Closing Cross had a record day as it was used for the 15th consecutive year to rebalance Nasdaq-listed securities in the entire family of Russell U.S. Indexes, part of leading global index provider FTSE Russell, during their annual reconstitution. A record 1.188 billion shares representing $39.26 billion were executed in the Closing Cross in 0.935 seconds across some 2,591 Nasdaq-listed stocks. This compares to 2017, when Nasdaq executed 972 million shares representing $28.9 billion executed in 0.861 seconds across 2,499 Nasdaq-listed stocks. "Today, we saw a record-breaking performance for the Nasdaq Closing Cross, which benefits issuers and investors alike with transparent and efficient price discovery," said Tom Wittman, Head of Global Equities and Executive Vice President of Global Trading and Market Services at Nasdaq. "The rebalance is an important event to ensure that the Russell U.S. ind
PRESS ALERT & INVITATION: Arbitrade to Hold a Press Conference on June 28th From Bermuda - Its New Global Headquarters22.6.2018 22:29 | Pressmeddelande
NEW YORK, June 22, 2018 (GLOBE NEWSWIRE) -- This is an invitation to all media following Arbitrade and/or covering the cryptocurrency markets. You are invited to join Arbitrade's Chairman, Len Schutzman, and management who will discuss their progress toward establishing Arbitrade as a world-class cryptocurrency exchange and coin company. They will also discuss their move and incorporation into Bermuda, one of the world's best regulated jurisdictions and a platform from which Arbitrade will reach out to several international markets. It will be a lively discussion that will also include details of the many economic and social advantages and new jobs that companies, like Arbitrade, will bring to countries in which they settle and do business around the world. Details of how to access the press briefing webcast are as follows: Please register now for the upcoming Arbitrade Conference Call: Date: Wednesday, June 27, 2018 Time: 8:00 a.m. EST We recommend that you connect to the meeting at l
Northland Power's Hai Long Offshore Wind Project Awarded Additional 744 Megawatts in Taiwan Auction22.6.2018 14:15 | Pressmeddelande
TORONTO, June 22, 2018 (GLOBE NEWSWIRE) -- Northland Power Inc. ("Northland") (TSX:NPI) today announced that the Taiwan Bureau of Energy ("BOE") has awarded 232 megawatts ("MW") to the Hai Long 2 offshore wind farm ("Hai Long 2") and 512 MW to the Hai Long 3 offshore wind farm ("Hai Long 3") under Taiwan's offshore wind auction program. These awards are separate from and in addition to the 300 MW in 2024 that Hai Long 2 was allocated by the Taiwan BOE on April 30, 2018 under Taiwan's Feed-in-Tariff ("FIT") program. Northland and its partner Yushan Energy Co. Ltd. ("Yushan Energy") own 60% and 40%, respectively, of Hai Long 2 and Hai Long 3. This is another significant step for Northland in Taiwan, with a second and third project progressing to connect to Taiwan's grid in 2025, subject to securing 20-year power purchase agreements. Taken together with the FIT award, the Hai Long 2 and Hai Long 3 offshore wind farms now total 1,044 MW of offshore wind capacity in Taiwan. Northland is bui
Williams Scotsman to Acquire ModSpace22.6.2018 13:09 | Pressmeddelande
Creates an industry-leading specialty rental services provider with over $1 billion of combined revenue and over 160,000 rental units across North America Leverages, and further strengthens, Williams Scotsman's scalable operating platform to capture an estimated $60 million of cost synergies Combines the best of both companies' go-to-market strategies, benefiting our customers and accelerating the expansion of Williams Scotsman's turnkey "Ready To Work" solutions across a broader asset base and enhanced branch network Total enterprise value of approximately $1.1 billion represents 6.6x ModSpace's Adjusted EBITDA for the twelve month period ended March 31, 2018, inclusive of forecast cost synergies and the expected value of acquired tax attributes1 ,2 BALTIMORE, June 22, 2018 (GLOBE NEWSWIRE) -- WillScot Corporation (NASDAQ:WSC) ("Williams Scotsman") the leading specialty rental services provider of innovative modular space and portable storage solutions across North America, today anno
GMAC Launches 'Study in China' Initiative in Partnership with 11 Leading Business Schools in China22.6.2018 06:01 | Pressmeddelande
BEIJING and HONG KONG, June 22, 2018 (GLOBE NEWSWIRE) -- On June 22, the Graduate Management Admission Council (GMAC) joins hands with 11 leading business schools in China to launch the "Study in China" initiative. This partnership marks the opening of an interactive platform for international students across 20 Belt and Road (B&R) regions to explore opportunities in China and to pursue higher education in business-related fields. The "Study in China" initiative aims to meet rapidly growing international student demand and to champion China as a world-class study destination and emerging global hub for business education. The "Study in China" online portal (www.studyinchinamba.com) officially launched today allows students to discover more about studying and living in China. The newly designed English-language website connects prospective students with partner business schools and offers access to an abundance of information to help find the perfect fit for their China education experi
I vårt pressrum kan du läsa de senaste pressmeddelandena, få tillgång till pressmaterial och hitta kontaktinformation.Besök vårt pressrum