Lipodystrophy, Congenital Generalized

The safety and efficacy of the asset in treating metabolic disorders linked to lipodystrophy syndromes were evaluated in an open-label, single-arm study. Credit: MikeSaran / shutterstock.com. Health Canada has approved Chiesi Group unit Chiesi Global Rare Diseases’ MYALEPTA (metreleptin for injection) to treat individuals with lipodystrophy, an ultra-rare condition. MYALEPTA is a replacement therapy adjunct to diet to address complications of leptin deficiency in patients with congenital or acquired forms of the disease. MYALEPTA can be used in adults and children aged two and above with confirmed congenital generalised lipodystrophy (Berardinelli-Seip syndrome) or acquired generalised lipodystrophy (Lawrence syndrome). It is also approved for patients aged 12 years and above with familial partial lipodystrophy (PL) or acquired PL (Barraquer-Simons syndrome). The treatment is intended for patients with metabolic disease who have not achieved adequate metabolic control with conventional treatments. MYALEPTA is originally developed by Amryt Pharma . Chiesi Group gained access to MYALEPTA through the acquisition of Amryt in April 2023. See Also: UK MHRA approves GSK’s momelotinib for myelofibrosis anaemia Germany’s Ministry of Health refutes accusations of worsening market conditions The safety and efficacy of the asset in treating metabolic disorders linked to lipodystrophy syndromes were evaluated in a long-term, open-label, single-arm study. The research was conducted under the supervision of the US National Institutes of Health. Weight decrease, hypoglycaemia and fatigue were the most commonly observed adverse drug reactions linked to the treatment. Chiesi Group Global Rare Diseases head Giacomo Chiesi stated: “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. “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.”

BOSTON, Feb. 05, 2024 (GLOBE NEWSWIRE) -- 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 Lipodystrophyi 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 MYALEPTAii,iii 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%). Important and complete safety information about MYALEPTA can be found by accessing the Product Monograph here. Important Safety Information Indication MYALEPTA (metreleptin for injection) is indicated as an adjunct to diet 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 2 years of age and above with confirmed familial partial LD (PL) or acquired PL (Barraquer-Simons syndrome), in adults and children 12 years of age and above with persistent significant metabolic disease for whom standard treatments have failed to achieve adequate metabolic control. Contraindications MYALEPTA is contraindicated in patients who are hypersensitive to this drug or to any ingredient in the formulation, including any non-medicinal ingredient, or component of the container. MYALEPTA is contraindicated in patients with general obesity not associated with confirmed generalised leptin deficiency, or confirmed partial lipodystrophy, or with HIV-related lipodystrophy. Most Serious Warnings and Precautions Anti-metreleptin antibodies with neutralizing activity have been identified in patients treated with MYALEPTA. The consequences are not well characterized but could include inhibition of endogenous leptin action and loss of MYALEPTA efficacy. Worsening metabolic control and/or severe infection have been reported. Test for anti-metreleptin antibodies with neutralizing activity in patients with severe infections or loss of efficacy during MYALEPTA treatment. T-cell lymphoma has been reported in patients with acquired generalised lipodystrophy, both treated and not treated with MYALEPTA. Carefully consider the benefits and risks of treatment with Myalepta in patients with significant hematologic abnormalities and/or acquired lipodystrophy. Hypoglycaemia risk with concomitant use of MYALEPTA with insulin and other antidiabetic medication. Large dose reductions of baseline insulin requirements may be needed in the first 2 weeks of treatment. Dose adjustments of other antidiabetic medications may also be needed to minimise the risk of hypoglycaemia. Closely monitor blood glucose in patients on concomitant insulin therapy, especially those on high doses, or insulin secretagogues and combination treatment. Hypoglycemia management should be reviewed and reinforced with patients when initiating MYALEPTA therapy. Acute pancreatitis: Discontinuation of MYALEPTA may result in worsening hypertriglyceridaemia and associated pancreatitis, particularly in patients with risk factors for pancreatitis (e.g. history of pancreatitis, severe hypertriglyceridaemia). If a patient develops pancreatitis whilst being treated with MYALEPTA, it is advised that MYALEPTA be continued uninterrupted, as stopping treatment abruptly may exacerbate the condition. When discontinuing MYALEPTA, tapering of the dose over a two-week period is recommended in conjunction with a low-fat diet. Autoimmune disorder progression / flares, including severe autoimmune hepatitis, have been observed in some patients treated with MYALEPTA. Leptin plays a role in immune system homeostasis. Acquired lipodystrophies are associated with autoimmune disorders including autoimmune hepatitis and membranoproliferative glomerulonephritis. A causal relationship between MYALEPTA treatment and the development and/or progression of autoimmune disease has not been established. Other relevant warnings and precautions Hypersensitivity reactions immediately after using MYALEPTA Benzyl alcohol toxicity in pediatric patients when reconstituted with BWFI. MYALEPTA contains no preservative when reconstituted with sterile Water for Injection (SWFI). Driving and operating machinery Pregnant women, women of child-bearing potential, nursing women Medication dosing errors in pediatric patients with small volumes of dosing Pediatrics – limited data in children aged 0 to 2 years with generalised LD and children aged 0 to 12 years with partial LD Geriatrics - limited information in patients of 65 years For more information Please consult the MYALEPTA Product Monograph at for important information relating to adverse reactions, drug interactions and dosing information that has not been discussed in this piece. The Product Monograph is also available by calling 1-800-696-1341. 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. For more information visit . About Chiesi Group Chiesi is an international, research-focused biopharmaceuticals group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi’s commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we’re part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With over 85 years of experience, Chiesi is headquartered in Parma (Italy), operates in 31 countries, and counts more than 6,500 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. 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. To learn more visit . For additional information, please contact: Chiesi Media Group Contract Chiara Travagin Rare Communications Manager Tel: +39 348 8818985 Email: c.travagin@chiesi.com Canadian Media Inquiries: Sky Striar, LifeSci Communications Tel: 617-797-6672 Email: sstriar@lifescicomms.com i Akinci, B., Oral, E., et. al. Comorbidities and Survival in Patients with Lipodystrophy: An International Chart Review Study. J. Clin. Endocrinol. Metab. 2019, 104(11):5120-5135. ii Brown, R., Oral, E., et. al. Long-term effectiveness and safety of metreleptin in the treatment of patients with generalized lipodystrophy. Endocrine. 2018 Jun;60(3):479-489. iii MYALEPTA Product Monograph.

Pictured: FDA signage outside its office in Maryland/iStock, hapabapa The FDA on Tuesday approved Chiesi Global Rare Diseases’ topical drug birch triterpenes, now to be marketed as Filsuvez, for the treatment of junctional or dystrophic epidermolysis bullosa in patients six months of age and older. Tuesday’s regulatory win makes Filsuvez the first approved treatment for wounds related to junctional epidermolysis bullosa (EB), a rare and moderate-to-severe form of the skin disease that often develops starting from infancy, according to Chiesi. Brett Kopelan, executive director of the non-profit debra of America, in a statement called Filsuvez’s approval an “important milestone” for patients living with junctional EB, adding that Filsuvez can also provide “those living with EB a safe and effective treatment option for the most prominent and difficult symptom of EB, open wounds that may not heal.” The FDA’s decision is backed by data from the Phase III EASE study, a double-blinded, randomized and vehicle-controlled study that demonstrated accelerated wound healing in patients treated with Filsuvez, along with a reduction in overall wound burden and lower pain associated with changing wound dressings. Filsuvez is a topical herbal gel mixture, containing dry extracts from two bark species—Betula pendula Roth and Betula pubescens Ehrh—as well as hybrids of both species. Its exact mechanism of action is currently unknown but based on its clinical efficacy and safety pro won the EU’s marketing authorization in June 2022. The topical treatment was originally developed by Amryt Pharma, which Chiesi bought in January 2023 for $1.25 billion upfront. The deal also came with a contingent payment of up to $225 million if Filsuvez met certain milestones—including winning an FDA approval. The Amryt acquisition also gave Chiesi two other rare disease assets: Myalept (metreleptin) for congenital or acquired generalized lipodystrophy and Mycapssa (octreotide) for acromegaly. The FDA previously rejected Filsuvez. In February 2022, the regulator handed Amryt a Complete Response Letter, asking for additional confirmatory data to prove the topical treatment’s efficacy. The biotech pushed back against the rejection and in June 2022 filed a formal dispute resolution request with the FDA. With Filsuvez’s approval on Tuesday, it joins Krystal Biotech’s Vyjuvek (beremagene geperpavec-svdt), which won the FDA’s go-ahead in May 2023 for dystrophic EB. Vyjuvek is the first-ever topical and redosable gene therapy to hit the market. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. He can be reached at tristan@tristanmanalac.com or tristan.manalac@biospace.com.