BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases, is pleased to announce the Health Canada approval of MYALEPTA™ (metreleptin for injection).
As an adjunct to diet, MYALEPTA is indicated as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients with confirmed congenital generalised LD (Berardinelli-Seip syndrome) or acquired generalised LD (Lawrence syndrome) in adults and children two years of age and above. MYALEPTA is also indicated in adults and children 12 years of age and above with confirmed familial partial LD (PL) or acquired PL (Barraquer-Simons syndrome) with persistent significant metabolic disease for whom standard treatments have failed to achieve adequate metabolic control.
MYALEPTA was developed by Amryt Pharma, which was acquired in April 2023 by the Chiesi Group, an international, research focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare disease and specialty care.
“Patients living with lipodystrophy are often burdened with tremendous physical, psychological and emotional challenges and as a profession we have had little in our toolbox of treatments to help them,” said Dr. Robert A. Hegele, Endocrinologist, Professor of Medicine and Biochemistry, Western University. “The approval of MYALEPTA provides clinicians and patients with a treatment that directly impacts the underlying metabolic disorder, which will result in effective symptom control.”
“We are very pleased that Medison has taken the initiative needed to secure MYALEPTA’s approval in Canada, offering patients new hope with a treatment specifically targeting the underlying cause of this rare condition,” said Dr. Sonia Rehal, science educator and patient advocate, Lipodystrophy Canada Foundation. “Being a lipodystrophy patient myself and having lost my mother and sister to complications from this devastating disorder, our work is helping to advocate on behalf of patients and caregivers.”
About Lipodystrophy
Lipodystrophy syndromes are a diversified group of rare, potentially life-threatening disorders that affect how the body accumulates and stores fat. These syndromes are categorized into two main forms: generalized is characterized by the absence or progressive loss of fat (adipose) tissue, and partial where the tissue loss is more limited, typically impacting certain areas like limbs or upper body. Lipodystrophy is also categorized by etiology with inherited and acquired forms.
The estimated prevalence of lipodystrophy syndromes is 1.3 to 4.7 cases per million worldwide.
Patients with lipodystrophy present with a broad range of symptoms, which can vary depending on the type of lipodystrophy and the extent of fat loss. The most common symptoms include organ abnormalities (e.g. hepatic steatosis, nephropathy and pancreatitis) and metabolic abnormalities (diabetes, insulin resistance and hypertriglyceridemia). These severe metabolic abnormalities can lead to organ damage and higher rates of mortality. Additionally, patients experience other comorbidities such as reproductive dysfunction, psychological distress and pain.
In March 2021, Medison, the creator and leader of the multi-regional commercial platform, extended its partnership with Amryt Pharma, adding Canada to their partnership in Israel. The multiregional partnership has continued, following Amryt Pharma’s acquisition by Chiesi Farmaceutici S.p.A. (“Chiesi”).
“At Medison, our business model is built on partnering with emerging biotech companies in the pursuit of bringing highly innovative therapies to patients in international markets,” said Gil Gurfinkel, CEO at Medison. “We are proud to have played an instrumental leadership role, leveraging our multi-regional platform assets, to expand the global access of MYALEPTA to patients in Canada.”
“We are very proud of the partnership that we have developed with Medison and applaud the team for their efforts to bring this innovative and important treatment to patients in Canada,” said Giacomo Chiesi, Head of Global Rare Diseases at the Chiesi Group. “At the Chiesi Group, we are committed to creating a difference in the lives of patients with rare diseases and in collaboration with the Medison team, we have been able to achieve this for those living with lipodystrophy.”
“Patients in Canada with lipodystrophy have been waiting for MYALEPTA to become available for many years and we are very pleased to be able to make this announcement today,” said Pamela Minden, Country Manager at Medison Pharma in Canada. “The commercialization strength of our team, leading to the achievement of MYALEPTA’s approval, has once-again demonstrated the value we bring to our partners in support of patients with unmet medical needs.”
About 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 generalized lipodystrophy (GL) – congenital or acquired – included a change from baseline to month 12 in HbA1c of -2.2 per cent and a percent change from baseline to month 12 in triglycerides of -32.1 per cent.
The observed primary efficacy results in the PL subgroup patients included a change from baseline to month 12 in HbA1c of -0.9 per cent and a percent change from baseline to month 12 in triglycerides of -37.4 per cent.
The most commonly reported adverse drug reactions (ADRs) were weight decreased (17%), hypoglycaemia (14%), and fatigue (7%).
About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people affected by rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system.
About Medison Pharma
Medison is a global pharma company focused on providing access to highly innovative therapies to patients in international markets. Medison is the first to create an international commercialization platform for highly innovative therapies, helping to save and improve lives by making the best available novel treatments accessible to patients in international markets. Medison has a track record of multi-territorial partnerships with leading pharmaceutical and biotech companies seeking to expand their global reach.
For additional information, please contact:
Chiesi Media Group Contract
Chiara Travagin
Rare Communications Manager
Tel: +39 348 8818985
Email: [email protected]
Canadian Media Inquiries:
Sky Striar, LifeSci Communications
Tel: 617-797-6672
Email: [email protected]