European Commission Approves Kanuma for Lysosomal Acid Lipase Deficiency

The European Commission has approved Kanuma, an innovative enzyme replacement therapy (ERT), for patients of all ages with lysosomal acid lipase deficiency (LAL-D), a genetic and progressive ultra-rare metabolic disease. This approval marks a significant milestone for patients with LAL-D, who suffer from multi-organ damage and premature death. Kanuma has shown significant benefit in terms of survival, reducing liver damage and lipid accumulation, and improving liver function in clinical trials.

Key Takeaways:

• The European Commission has approved Kanuma for patients of all ages with LAL-D, a genetic and progressive ultra-rare metabolic disease.
• Clinical studies have shown significant benefit in terms of survival, with 67% of infant patients treated with Kanuma surviving beyond 12 months, compared to 0% in an untreated historical cohort.
• Treatment with Kanuma has resulted in normalization of ALT, reduction in liver fat content, and other markers of liver injury in pediatric and adult patients with LAL-D.
• The most serious adverse reactions experienced by 3% of patients in clinical studies were signs and symptoms consistent with anaphylaxis, including chest discomfort, conjunctival injection, dyspnea, rash, hyperemia, mild eyelid edema, rhinorrhea, respiratory distress, tachycardia, tachypnea, and urticaria.
• Kanuma is designed to address the underlying cause of LAL-D by reducing substrate accumulation in the lysosomes of cells throughout the body, including the liver.
• Alexion has commenced reimbursement processes with healthcare authorities in each of the major European countries, with the goal of making Kanuma available to patients as quickly as possible.
• Kanuma has been granted Breakthrough Therapy designation for LAL Deficiency presenting in infants by the U.S. Food and Drug Administration, and accepted the Kanuma BLA for Priority Review.
• A New Drug Application for Kanuma has been submitted to Japan's Ministry of Health, Labour and Welfare.

Statistics:

• 67% of infant patients treated with Kanuma survived beyond 12 months, compared to 0% in an untreated historical cohort.
• 3% of patients in clinical studies experienced signs and symptoms consistent with anaphylaxis.
• 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.
• Kanuma has been shown to reduce the markers of liver injury and lipid accumulation in patients with LAL-D.

Sources:

• Business Wire
• NewsRx