GlobeNewswire

MYALEPTA® (metreleptin) Receives Positive CHMP Opinion in Patients with Generalized and Partial Lipodystrophy

Dela

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.

About 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. 

Forward-Looking Statements

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.

CONTACT:

Amanda Murphy, Director, Investor Relations & Corporate Communications
Novelion Therapeutics
857-242-5024
amanda.murphy@novelion.com




This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
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

GlobeNewswire



Följ GlobeNewswire

Abonnera på våra pressmeddelanden.

Senaste pressmeddelandena från GlobeNewswire

Karolinska Development's portfolio company Pharmanest changes name and signs licensing agreement15.10.2018 13:25Pressmeddelande

STOCKHOLM, October 15, 2018. Karolinska Development's portfolio company Phamanest changes its name to Palette Life Sciences. The company also announces a global licensing agreement with Nestlé Skin Health. Palette Life Sciences (former Pharmanest) has announced a licensing agreement with Nestlé Skin Health. The agreement gives Palette Life Sciences the worldwide commercialization and development rights for three products: Deflux®, Solesta® and BarrigelTM. The products are all based on the NASHATM (Non Animal Stabilized Hyaluronic Acid) technology. Palette Life Sciences will focus immediately on sales of Deflux and Solesta and will begin preparing for worldwide commercialization of Barrigel. Nestlé Skin Health will continue to manufacture the products on behalf of Palette Life Sciences. Palette Life Science will continue the development of SHACT under the name LidbreeTM. Lidbree will be submitted for European regulatory review later this year. Karolinska Development is a passive investo

Karolinska Development's portfolio company Pharmanest changes name and signs licensing agreement15.10.2018 13:25Pressmeddelande

STOCKHOLM, October 15, 2018. Karolinska Development's portfolio company Phamanest changes its name to Palette Life Sciences. The company also announces a global licensing agreement with Nestlé Skin Health. Palette Life Sciences (former Pharmanest) has announced a licensing agreement with Nestlé Skin Health. The agreement gives Palette Life Sciences the worldwide commercialization and development rights for three products: Deflux®, Solesta® and BarrigelTM. The products are all based on the NASHATM (Non Animal Stabilized Hyaluronic Acid) technology. Palette Life Sciences will focus immediately on sales of Deflux and Solesta and will begin preparing for worldwide commercialization of Barrigel. Nestlé Skin Health will continue to manufacture the products on behalf of Palette Life Sciences. Palette Life Science will continue the development of SHACT under the name LidbreeTM. Lidbree will be submitted for European regulatory review later this year. Karolinska Development is a passive investo

ICONOVO TECKNAR AVTAL MED MCMASTER UNIVERSITY AVSEENDE UTVECKLING AV VACCIN MOT TUBERKULOS15.10.2018 09:00Pressmeddelande

Det innovativa, Lundabaserade medicinteknikföretaget Iconovo AB (publ) meddelar i dag att bolaget har skrivit samarbetsavtal med McMaster University i Kanada. Avtalet avser utveckling av en torrpulverberedning av inhalerbart vaccin mot tuberkulos som ska användas i Iconovos unika engångsinhalator ICOone. Iconovo kommer att ingå i ett tvärvetenskapligt forsknings- och utvecklingsprojekt som syftar till att ta fram nästa generations virusbaserade vaccin i pulverform som är stabilt i rumstemperatur. Att kunna transportera vaccin utan krav på kylförvaring är en förutsättning för att snabbt och effektivt nå ut till de behövande. I det projekt som nu påbörjas kommer Iconovos unika engångsinhalator ICOone att användas som testplattform för att underlätta en framtida kommersialisering och storskalig tillverkning. Projektet beräknas pågå i tre år och finansieras bland annat av den kanadensiska staten (CIHR/NSERC). Tuberkulos förekommer i hela världen och är en av de tio vanligaste dödsorsakerna

Stillfront Group AB: Babil Games lanserar Arab Gamers' League4.10.2018 10:02Pressmeddelande

PRESSMEDDELANDE 4 oktober 2018 Babil Games, en del av Stillfront Group, ökar affärstillväxten och lanserar Arab Gamers' League, AGL Babil Games, ett dotterbolag till Stillfront Group, inleder partnerskap med kreativa byrån Grape Creations i Dubai och lanserar Arab Gamers' League (AGL), en ny studio som fokuserar på att lansera så kallade 'social casual games' till MENA-regionen. Babil Games kontrollerar 51% av AGL och Grape Creations övriga 49%. Babil Games är ansvarig för verksamheten och sourcing av spel medan Grape Creations ansvarar för marknadsföring och distribution. "Vi är mycket glada över vårt inledda partnerskap med Grape Creations gällande lanseringen av Arab Gamers' League. AGLs fokus är att lansera nästa generations 'social casual games' till MENA-regionen", säger MJ Fahmi, VD för Babil Games. Babil Games kommer att fortsätta att fokusera på MMO-strategispel. Antalet anställda i Babil Games har fördubblats under det senaste året och studion kommer att flytta till ett nytt

Stillfront Group AB: Babil Games launches Arab Gamers' League4.10.2018 10:02Pressmeddelande

PRESS RELEASE 4 October 2018 Babil Games, part of the Stillfront Group, accelerates business growth and launches Arab Gamers' League, AGL Babil Games, a subsidiary of Stillfront Group, is partnering with Dubai based creative agency Grape Creations to launch Arab Gamers' League (AGL), a new studio focused on bringing social casual games to the MENA-region. Babil Games controls 51% of AGL and Grape Creations the other 49%. Babil Games is head of the operations and the sourcing of the games while Grape Creations is responsible for marketing and distribution. "We are pleased to have Grape Creations as our partner for the launch of Arab Gamers League. In AGL we will focus on bringing the next generation of social casual games to the MENA region", says MJ Fahmi, CEO of Babil Games. Babil Games will continue to focus on strategy MMO games. The number of employees in Babil Games has doubled during the year and the studio will relocate to a new larger office in Amman, Jordan. "The expansion is

Hoylu AB: UNITED STATES AIR FORCE SELECTS HOYLU FOR COLLABORATION AND INNOVATION SOLUTIONS28.9.2018 08:30Pressmeddelande

Malmo, Sweden, September 28, 2018 - Hoylu, a leading enterprise collaboration company announced today that the United States Air Force has selected Hoylu Software and multiple large format HoyluWall systems as a digital workspace tool for workspace innovation and collaboration. Hoylu software will be used at multiple sites to create joined environments within the division. The order will be delivered in Q4 2018 and represents a deal value of SEK 3,670,000 in product and software revenue. The Air Force will use the system for training and education purposes, with the ability to connect users together and collaborate in real-time. The order represents a growing market for Hoylu software and services within the government sector. It is expected that Hoylu's solutions will continue to expand within this industry as innovative collaboration solutions are in demand. Hoylu's solutions and software offer new and exciting ways to learn and collaborate smarter, faster and more efficiently. Creat

I vårt pressrum kan du läsa de senaste pressmeddelandena, få tillgång till pressmaterial och hitta kontaktinformation.

Besök vårt pressrum