Making the RARE voice heard
Myalepta®▼ Approved in Europe : the First Treatment Indicated for Patients with Ultra-Rare Disease, Lipodystrophy
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.”
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.”
Professor Sir Stephen O’Rahilly, Professor of Clinical Biochemistry and Medicine at Addenbrooke’s Hospital, Cambridge
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.”
Rebecca Sanders, Founder and Chair of Lipodystrophy UK
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).[i] The condition is associated with a number of critical metabolic problems, such as severe diabetes, and can cause severe organ damage if uncontrolled.[ii] 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
[i] Chiquette, E., et al. Estimating the prevalence of generalized and partial lipodystrophy: findings and challenges. Diabetes Metab Syndr Obes, 2017. 10: p.375-383.
[ii] Pope, E., et al., Childhood acquired lipodystrophy: A retrospective study. Journal of the American Academy of Dermatology, 2006. 55(6): p. 947-950.
Rare Revolution Editor