Health Canada Approves Chiesi’s Myalepta for Lipodystrophy
February 5, 2024
Rare Daily Staff
Health Canada has approved Chiesi Global Rare Diseases Myalepta as an adjunct to diet to treat the complications of leptin deficiency in the rare, metabolic condition lipodystrophy.
The approval of Myalepta as a replacement therapy is for patients with confirmed congenital generalized lipodystrophy (LD) (Berardinelli-Seip syndrome) or acquired generalized 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.
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 tissue, and partial where the tissue loss is more limited, typically impacting certain areas like limbs or upper body. People with the condition 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 and metabolic abnormalities. 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.
Myalepta, a recombinant analog of human leptin. The U.S. Food and Drug Administration approved Myalepta in February 2014 as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired general lipodystrophy. It won approval in Europe in 2018. Amryt Pharma, which Chiesi Group acquired in 2023, developed 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 in the United States.
The observed primary efficacy results in patients with generalized lipodystrophy–congenital or acquired –included a change from baseline to month 12 in HbA1c of -2.2 percent and a percent change from baseline to month 12 in triglycerides of -32.1 percent.
The observed primary efficacy results in the familial partial LD 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 were weight decreased (17 percent), hypoglycemia (14percent), and fatigue (7 percent).
“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 Robert Hegele, endocrinologist and professor of medicine and biochemistry at 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.”
Photo: Robert Hegele, endocrinologist and professor of medicine and biochemistry at Western University
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