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
Om Nasdaq GlobeNewswire
One Liberty Plaza - 165 Broadway
NY 10006 New York
+1 212 401 8700http://www.nasdaqomx.com
NASDAQ (NASDAQ: NDAQ) is a leading provider of trading, exchange technology, information and public company services across six continents.
Følg saker fra Nasdaq GlobeNewswire
Registrer deg med din epostadresse under for å få de nyeste sakene fra Nasdaq GlobeNewswire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra Nasdaq GlobeNewswire
Primal Pictures Partners with Physitrack to Create a Unique Patient Education Solution25.6.2018 18:00 | Pressemelding
LONDON, June 25, 2018 (GLOBE NEWSWIRE) -- Today, Primal Pictures, pioneer of the world's most medically-accurate and detailed 3D models of human anatomy, and Physitrack, the market-leading patient engagement and Telehealth solution provider, announce their partnership on a unique anatomically augmented home exercise and patient education feature. The feature inside Physitrack, which is powered by Primal Pictures, will allow healthcare practitioners to better educate their patients, and therefore help patients to improve their health outcomes. "Even the best physical therapy regime will be unsuccessful without proper patient education," explained Henrik Molin, CEO and co-founder of Physitrack. "That's why Physitrack developed what has become the industry's gold standard in patient engagement and outcome tracking. With Primal Pictures, we saw the opportunity to take our solution to a new level by helping patients make the connection between their physical therapy regime and the anatomy i
EPAM's InfoNgen(TM) Debuts With Highest Score Possible in Seven Criteria in AI-Based Text Analytics Platforms Report by Independent Research Firm25.6.2018 16:58 | Pressemelding
InfoNgen, categorized as one of the top eight providers that matter most, is a fully integrated, end-to-end turnkey solution that helps enterprises become more insights-driven NEWTOWN, Pa., June 25, 2018 (GLOBE NEWSWIRE) -- EPAM Systems, Inc. (NYSE:EPAM), a leading global provider of digital platform engineering and software development services, today announced that it has been named a Contender in the report "The Forrester Wave(TM): AI-Based Text Analytics Platforms, Q2 2018" for its product InfoNgen(TM). InfoNgen ranked among the highest scores in the enterprise features, RPA functionality, training data for machine learning, focus on cloud and SaaS, and overall revenue criteria. According to the report, "[EPAM's] InfoNgen text analytics product is a good example of how modern consulting firms and systems integrators differentiate - they productize solutions." "EPAM's inclusion in this report, along with leaders like IBM and SAS, demonstrates, in our opinion, our ability to extend e
Align Technology Reaches One Millionth Invisalign(R) Patient in the EMEA Region25.6.2018 16:00 | Pressemelding
Milestone Reflects Accelerating Adoption of Invisalign Treatment Among Adults and Teens in Europe SAN JOSE, Calif. and AMSTERDAM, The Netherlands, June 25, 2018 (GLOBE NEWSWIRE) -- Align Technology, Inc. (Nasdaq:ALGN), today announced that it shipped its one millionth Invisalign case for the EMEA region in April, 2018. This significant milestone for the company and the more than 34 thousand Invisalign-trained orthodontists and general dentists in the region reflects accelerating adoption of Invisalign clear aligner therapy in Europe, Middle East and Africa. "I am immensely proud to announce that we have reached yet another significant milestone for EMEA with our one millionth Invisalign patient in the region. The demand and adoption rate for Invisalign clear aligners in EMEA has accelerated rapidly during recent years. I would like to thank our doctors for their trust and confidence in treating patients with the Invisalign system, and acknowledge the more than one million patients in E
Novelion Therapeutics Announces Presentation of Positive Metreleptin Data at American Diabetes Association Scientific Sessions25.6.2018 14:00 | Pressemelding
Data Show Leptin Replacement Therapy Sustainably Decreased Weight Over Time in Patients with Low Baseline Leptin Levels Company plans to initiate Phase 2 proof of concept study in hypoleptinemic metabolic disorder (HMD) by year-end VANCOUVER, British Columbia, June 25, 2018 (GLOBE NEWSWIRE) -- Novelion Therapeutics Inc. (NASDAQ:NVLN), a biopharmaceutical company dedicated to developing and commercializing therapies for individuals living with rare diseases, today announced that clinical data from a metreleptin study assessing weight loss in overweight and obese adults with low leptin levels will be featured in a poster presentation at the American Diabetes Association's (ADA) 78th Scientific Sessions, being held June 22-26, 2018 in Orlando. The study, titled "Efficacy of Metreleptin for Weight Loss in Overweight and Obese Adults With Low Leptin Levels," was conducted in adults with low baseline leptin, defined as <16 ng/mL in females and <5 ng/mL in males, and Body Mass Index (BMI) lev
ERYTECH Confirms Strategic Focus of Eryaspase on Solid Tumors and Ceases Development in Acute Lymphoblastic Leukemia25.6.2018 00:06 | Pressemelding
Conference call on Monday, June 25th at 2:30 pm CET/8:30 am EDT Focuses development efforts on solid tumor indications Confirms planned launch of company-sponsored randomized Phase 2 trial in first-line pancreatic cancer, in addition to ongoing launch of Phase 3 trial in second-line pancreatic cancer. Expands eryaspase production capacity in the United States and France Plans to cease development in acute lymphoblastic leukemia (ALL), including withdrawal of European Marketing Authorization Application (MAA) LYON, France and CAMBRIDGE Mass., June 24, 2018 (GLOBE NEWSWIRE) -- ERYTECH Pharma (Euronext:ERYP) (Nasdaq:ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, today announced that it will focus its development efforts for its product candidate eryaspase on the potential treatment of selected solid tumor indications. The company also announced that it plans to cease its development pro
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 | Pressemelding
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
I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.Besøk vårt presserom