- Kanuma is the First Approved Treatment for Patients Suffering from LAL-D, a Life-threatening
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"Today's approval is a crucial milestone for patients with LAL-D, a grave condition that can have devastating consequences for patients of all ages," said Vassili Valayannopoulos, M.D., Ph.D., investigator in the Kanuma pivotal studies, Hôpital
LAL-D is a genetic, chronic and progressive metabolic disease in which infants, children and adults suffer multi-organ damage and premature death. It is an ultra-rare disease, which is defined as a disease that affects fewer than 20 patients per one million of the general population.1 Patients with LAL-D often experience a rapid onset of life-threatening disease manifestations, and similar to other liver diseases, many patients may be asymptomatic until they experience a severe consequence of the disease. LAL-D is caused by genetic mutations that result in a marked decrease or loss in LAL enzyme activity in the lysosomes across multiple body tissues, leading to the chronic build-up of cholesteryl esters and triglycerides in the liver, blood vessel walls and other tissues.2,3
"We are pleased that the
Kanuma is a highly innovative enzyme replacement therapy (ERT) designed to address the underlying cause of LAL-D. The approval of Kanuma applies to all 28 EU member states as well as
Clinical Data
The approval of Kanuma in the EU was based on data from two clinical studies and a supporting open-label extension study comprising infant, pediatric, and adult patients with LAL-D. Study results showed significant benefit in terms of survival (67%, or 6 out of 9) in patients with the infant form of LAL-D beyond 12 months, compared with 0 out of 21 patients in an untreated historical cohort. Infant patients treated with Kanuma also had improvements in liver parameters, including ALT and AST, as well as weight gain within the first several weeks of treatment. In pediatric and adult patients with LAL-D, treatment with Kanuma resulted in normalization of ALT, reduction in liver fat content and other markers of liver injury compared to placebo, as well as significant improvements in lipid accumulation as measured by LDL-C and HDL-C. In patients who received Kanuma during the double-blind period and subsequently entered the open-label extension period, reductions in ALT levels were maintained and further improvements were seen in LDL-C and HDL-C.
The most serious adverse reactions experienced by 3% of patients in clinical trials were signs and symptoms consistent with anaphylaxis. Signs and symptoms included chest discomfort, conjunctival injection, dyspnea, generalized and itchy rash, hyperemia, mild eyelid edema, rhinorrhea, severe respiratory distress, tachycardia, tachypnea and urticaria.
About Lysosomal Acid Lipase Deficiency (LAL-D)
LAL-D is a genetic, chronic and progressive ultra-rare metabolic disease associated with devastating morbidities and premature mortality. In patients with LAL-D, genetic mutations result in decreased activity of the LAL enzyme. This leads to marked accumulation of cholesteryl esters and triglycerides in vital organs, blood vessels, and other tissues, resulting in progressive and multi-organ damage including fibrosis, cirrhosis, liver failure, accelerated atherosclerosis, cardiovascular disease, and other devastating consequences.2,3
LAL-D affects patients of all ages with clinical manifestations from infancy through adulthood and may have sudden and unpredictable clinical complications. Infants experience profound growth failure, liver fibrosis, and cirrhosis with a median age of death at 3.7 months.4 In an observational study, approximately 50% of children and adults with LAL-D progressed to fibrosis, cirrhosis, or liver transplant in 3 years.5 The median age of onset of LAL-D is 5.8 years and the disease can be diagnosed with a simple blood test.6,7
About Kanuma™ (sebelipase alfa)
Kanuma™ (sebelipase alfa) is an innovative enzyme replacement therapy designed to address the underlying cause of lysosomal acid lipase deficiency (LAL-D) by aiming to reduce substrate accumulation in the lysosomes of cells throughout the body, including the liver, to prevent vital organ damage and premature death.
The
Important Safety Information
Hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with sebelipase alfa therefore, appropriate medical support must be readily available when sebelipase alfa is administered. If severe reactions occur, the sebelipase alfa infusion should be immediately stopped and appropriate medical treatment should be initiated. For patients who have experienced allergic reactions during infusion, caution should be exercised upon re-administration.
The most serious adverse reactions experienced by 3% of patients in clinical studies were signs and symptoms consistent with anaphylaxis. Signs and symptoms included chest discomfort, conjunctival injection, dyspnoea, generalised and itchy rash, hyperaemia, mild eyelid oedema, rhinorrhoea, severe respiratory distress, tachycardia, tachypnoea and urticaria.
About
[ALXN-G]
Forward-Looking Statements
This news release contains forward-looking statements, including statements related to potential medical benefits of Kanuma™ (sebelipase alfa) for lysosomal acid lipase deficiency (LAL-D). Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ from those expected, including, for example, decisions of regulatory authorities regarding marketing approval or material limitations on the marketing of Kanuma for LAL-D, delays in arranging satisfactory manufacturing capabilities and establishing commercial infrastructure for Kanuma for LAL-D, the possibility that results of clinical trials are not predictive of safety and efficacy results of Kanuma in broader or different patient populations, the risk that third party payors (including governmental agencies) will not reimburse for the use of Kanuma at acceptable rates or at all, the risk that estimates regarding the number of patients with Kanuma and observations regarding the natural history of patients with Kanuma are inaccurate, and a variety of other risks set forth from time to time in
References
1. REGULATION (EU) No 536/2014 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of
2. Bernstein DL, et al. Chloesteryl ester storage disease: review of the findings in 135 reported patients with an underdiagnosed disease. J Hepatol. 2013;58:1230-43. doi:10.1016/j.jhep.2013.02.014.
3. Reiner Z, et al. Lysosomal acid lipase deficiency - an under-recognized cause of dyslipidemia and liver dysfunction. Atherosclerosis. 2014;235:21-30. doi:10.1016/j.atherosclerosis.2014.04.003.
4. Jones S et al. Severe and rapid disease course in the natural history of infants with lysosomal acid lipase deficiency. Mol Genet Metab. 2014 Feb;111(2):S57-58.
5. Data on file,
6. Burton et al. Clinical Features of Lysosomal Acid Lipase Deficiency - a Longitudinal Assessment of 48 Children and Adults. J Pediatr Gastroenterol Nutr. 2015
7. Hamilton J, et al. A new method for the measurement of lysosomal acid lipase in dried blood spots using the inhibitor Lalistat 2.
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