Business Wire

Aegerion’s Myalepta®▼ Approved in Europe : the First Treatment Indicated for Patients with Ultra-Rare Disease, Lipodystrophy

Share

Aegerion Pharmaceuticals today announced the granting of a marketing authorisation in Europe for Myalepta (metreleptin) by the European Commission. Myalepta is a treatment for the ultra-rare condition, lipodystrophy, and has been approved as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy patients. With this authorisation, Myalepta becomes the first and only licensed medication to treat the underlying leptin deficiency at the heart of this ultra-rare condition.

Professor Sir Stephen O’Rahilly, Professor of Clinical Biochemistry and Medicine at Addenbrooke’s Hospital, Cambridge, said, “Lipodystrophy is a rare condition which, due to its complex nature, requires highly specialised treatment. Patients with lipodystrophy have previously relied on lifestyle changes and medications, like insulin injections to manage the condition’s associated complications. Today’s milestone marks a significant change in the way lipodystrophy is treated. For the first time, we will be able to offer patients in Europe a treatment option that addresses one of the fundamental aspects of the condition itself.”

Rebecca Sanders, Founder and Chair of Lipodystrophy UK, said, “Lipodystrophy presents multiple ongoing challenges, both physical and psychological, to individuals living with the condition. Current available treatment options have been limited, address only some of the complications of the condition and are not specialised for lipodystrophy. Myalepta offers a long-term solution for patients and we are delighted that there is now a treatment available specifically to treat the complications of leptin deficiency in lipodystrophy which will give patients a much needed option to help improve both their disease and their quality of life.”

Lipodystrophy is an ultra-rare and incurable disease which impacts a person’s ability to store fat in their body, and can either be generalised (GL) or partial (PL).1 The condition is associated with a number of critical metabolic problems, such as severe diabetes, and can cause severe organ damage if uncontrolled.2 Globally, lipodystrophy affects approximately between 1 and 4 people per million.1

Paul Greenland, President EMEA at Aegerion Pharmaceuticals, said, “We are thrilled that patients with lipodystrophy in Europe will now have a specific treatment option. This also represents an exciting time for Aegerion as Myalepta becomes available in one of the largest global pharmaceutical markets. We will work collaboratively with patient organisations, health care professional and health services across Europe to enable access to as many patients as possible.”

#ENDS#

NOTES TO EDITORS

Data supporting approval of Myalepta

The safety and efficacy of Myalepta for the treatment of metabolic disorders, associated with lipodystrophy syndromes in pediatric and adult patients, were evaluated in a long-term, open-label, single-arm study conducted under the auspices of the National Institutes of Health (NIH) in the United States.

The observed primary efficacy results in patients with generalised lipodystrophy were:

  • A mean change from baseline to month 12 in HbA1c* of -2.2%; and
  • A mean percent change from baseline to month 12 in triglyceridesof -32.1%.3

In addition, 41% (16 of 39) of GL patients on insulin at baseline were insulin-free following treatment, 22% (7 of 32) of GL patients on oral anti-diabetic agents at baseline were able to discontinue, and 24% (8 of 34) of GL patients on lipid-lowering therapies at baseline were able to discontinue use of these therapies during Myalepta treatment.4

The observed primary efficacy results in the PL subgroup patients were:

  • A mean change from baseline to month 12 in HbA1c of -0.9%; and
  • A mean percent change from baseline to month 12 in triglycerides of -37.4%.3

The most frequently occurring adverse reactions from the clinical studies were hypoglycaemia (14%) and weight decreased (17%). Other common side effects (occurring inbetween 1 to 10% of patients) were decreased appetitite, headache, abdominal pain, nausea, alopecia, menorrhagia, fatigue, injection site bruising/erythema/reactions, neutralising antibody formation**.3

About lipodystrophy

Lipodystrophy is an ultra-rare condition that is characterised by the irreversible loss of adipose tissue.1 In patients with lipodystrophy, the lack of adipose tissue results in a loss of leptin production. Leptin is a naturally occurring hormone produced by subcutaneous fat and is an important regulator of energy homoeostasis, fat and glucose metabolism, reproductive capacity, and other diverse physiological functions.5

Lipodystrophy can be genetic or acquired after an immune condition and occurs in childhood or adolescence.6 Generalised lipodystrophy is a complete lack of fat affecting the whole body.7 Partial lipodystrophy is typically associated with a lack of fat in the arms, legs, head, and trunk regions while fat can accumulate in other areas of the body, including the neck, face and intra-abdominal regions affecting vital organs like the liver and heart.7

About Aegerion Pharmaceuticals

Aegerion Pharmaceuticals is a Novelion Therapeutics Inc. company: a biopharmaceutical company dedicated to developing and commercialising innovative new therapies for individuals living with rare and orphan diseases. Aegerion was acquired by Novelion Therapeutics Inc. in November 2016. The company’s European headquarters are situated in Windsor, UK.

This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. Adverse events can be reported to Aegerion at

00800 23437466 or at medinfo.emea@aegerion.com

* HbA1c is a standard test used to measure an individual’s average blood sugar levels

Triglycerides are a type of fat in the blood

** Refer to the SmPC for a full list of adverse reactions (provided with this press release)

References

1 Chiquette, E., et al. Estimating the prevalence of generalized and partial lipodystrophy: findings and challenges. Diabetes Metab Syndr Obes, 2017. 10: p.375-383.

2 Pope, E., et al., Childhood acquired lipodystrophy: A retrospective study. Journal of the American Academy of Dermatology, 2006. 55(6): p. 947-950.

3 SmPC EMEA. Myalepta 11.3 mg powder for solution for injection. (provided with this document).

4 Brown, RJ., et al., Long-term effectiveness and safety of metreleptin in the treatment of patients with generalized lipodystrophy. Endocrine, 2018. 60(3): p.479-489.

5 Kelesidis, T., et al., Narrative Review: The Role of Leptin in Human Physiology: Emerging Clinical Applications. Ann Intern Med, 2010. 152(2): p.93–100.

6 Brown, R.J., et al., The Diagnosis and Management of Lipodystrophy Syndromes: A Multi-Society Practice Guideline. J Clin Endocrinol Metab, 2016. 101(12): p. 4500-4511.

7 Fiorenza, C.G., et al., Lipodystrophy: Pathophysiology and Advances in Treatment. Nat Rev Endocrinol, 2011. 7(3): p. 137-150.

Contact information

Aegerion Pharmaceuticals
Charlotte Dawkins
Consultant, Patient Advocacy and Communications
07932 722950
charlotte.dawkins@aegerion.com
or
Harriet Reynolds
Senior Account Manager
Reynolds-MacKenzie
07384 438631
HarrietR@ReynoldsMackenzie.com

About Business Wire

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

LTI to Acquire Advanced Analytics Firm Lymbyc17.7.2019 10:07:00 EESTPress release

Larsen & Toubro Infotech Ltd. (NSE: LTI, BSE: 540005), a global technology consulting and digital solutions company, has signed a definitive agreement to acquire Lymbyc, a specialist AI, machine learning, and advanced analytics company. The acquisition further strengthens LTI’s fast-growing digital and analytics offerings. Founded in 2012, Lymbyc is headquartered in Bengaluru, India and has customers in the USA, UK, Africa, and India. The company is well recognized for its unique capabilities in analytics and data-sciences space. The core of Lymbyc’s capabilities are centered around a proprietary product, Leni, which solves for the entire spectrum of descriptive to predictive business insights, leveraging deep learning, Natural Language Processing, data visualization and predictive analytics. Leni is a virtual analyst that allows users to conversationally access information and insights. With growing dependence on data, global enterprises need to be better at data discovery, agile anal

Unicaja Banco signs strategic partnership with Ebury Partners17.7.2019 09:30:00 EESTPress release

Ebury, one of the UK and Europe’s largest fintechs, is pleased to announce it has entered into a partnership with Spain’s seventh largest bank, Unicaja Banco. The agreement will enable Unicaja to offer international payments and cash management services through Ebury’s global transaction banking platform to its 400,000 SME customers and 3 million retail customers. Using Ebury’s regulated and future-proofed capabilities, Unicaja will significantly enhance its offering, particularly to its SME customer-base. Unicaja´s customers can now make international payments in more than 100 currencies, collect over 40 currencies across Europe, APAC, Africa and South America, use dedicated currency accounts in over 20 countries, and access flexible foreign exchange risk management tools for over 130 currencies, via one platform 24/7, from any location. The partnership demonstrates Ebury’s ability to help banks around the world by enabling them to offer their clients an innovative global transaction

Traxens Closes €20M Series C Financing Round With Key Strategic Partners17.7.2019 09:30:00 EESTPress release

Traxens, expert in providing high-value data and services for the supply chain industry, today announces that it has closed a Series C funding round worth close to €20M ($22.7M), led by the Itochu Corporation, Bpifrance and Supernova Invest (Crédit Agricole Innovations et Territoires Fund). This financing will enable the company to launch a global large-scale sea-land fleet of IoT tracking solutions on vessels operated by its partner shipping companies, including CMA CGM and MSC. Traxens expects to have 100,000 containers (both dry and reefer containers) equipped with its solution by end of 2020, making it one of the major players in the IoT for smart containers industry. This number is set to grow even higher after Traxens announced in June 2019 that Maersk will become a shareholder and customer with an initial order for up to 50,000 containers. Traxens’ Internet-of-Things solution is based on a patented, breakthrough technology that enables access to the most comprehensive, precise a

Gilead Sciences to Release Second Quarter 2019 Financial Results on Tuesday, July 30, 201916.7.2019 23:05:00 EESTPress release

Gilead Sciences, Inc. (Nasdaq: GILD) announced today that its second quarter 2019 financial results will be released on Tuesday, July 30, after the market closes. At 4:30 p.m. Eastern Time, Gilead’s management will host a conference call to discuss the company’s financial results for the second quarter 2019 and provide a business update. The live webcast of the call can be accessed at the company’s Investors page at http://investors.gilead.com/. Please connect to the company’s website at least 15 minutes prior to the start of the call to ensure adequate time for any software download that may be required to listen to the webcast. Alternatively, please call 877-359-9508 (U.S.) or 224-357-2393 (international) and dial the conference ID 8696029 to access the call. Telephone replay will be available approximately two hours after the call through 8:00 p.m. Eastern Time, August 1, 2019. To access the replay, please call 855-859-2056 (U.S.) or 404-537-3406 (international) and dial the confere

MSCI Schedules Investor Conference Call to Review Second Quarter 2019 Results16.7.2019 17:49:00 EESTPress release

MSCI Inc. (NYSE: MSCI), a leading provider of critical decision support tools and services for the global investment community, announced today it will release its results for second quarter 2019 on Thursday, August 1, 2019. MSCI's senior management will review the second quarter 2019 results on Thursday, August 1, 2019 at 11:00 AM Eastern Time. To listen to the live event, visit the events and presentations section of MSCI's Investor Relations homepage, http://ir.msci.com/events.cfm, or dial 1-877-376-9931 conference ID: 7743929 within the United States. International callers dial 1-720-405-2251 conference ID: 7743929. The earnings release and related investor presentation used during the conference call will be made available on MSCI's Investor Relations homepage. An audio recording of the conference call will be available on our Investor Relations website, http://ir.msci.com/events.cfm, beginning approximately two hours after the conclusion of the live event. Through August 4, 2019,

Keio Plaza Hotel Tokyo Offers “Tea Ceremony Experience and Luxurious Accommodations for Families” Package16.7.2019 17:05:00 EESTPress release

Keio Plaza Hotel Tokyo (KPH), one of Japan’s most prestigious international hotels located in Shinjuku, Tokyo, will begin offering a special summer accommodation package entitled “Tea Ceremony Experience and Luxurious Accommodations for Families” from July 20 to September 1, 2019. This accommodation package has been specially designed to keep families with children entertained by providing the unique cultural experience of a Japanese tea ceremony as fond memories of their summer vacation at our Hotel. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190716005325/en/ With the accommodation package, guests with children could experience unique cultural Japanese tea ceremony to learn how to make a green tea and its history. (Photo: Business Wire) The rooms offered in this accommodation package are the very popular “Two Queen Beds Rooms” located on the “Premier Grand” club floors with use of the Club Lounge included. The rooms are

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom