Segesterone Acetate and Ethinyl Estradiol
Kanuma (sebelipase alfa) is a prescription enzyme replacement therapy designed to treat Lysosomal Acid Lipase (LAL) deficiency, a rare genetic disorder where the body cannot properly break down and recycle fats. It is approved for use in both infants and children/adults with this condition, helping to reduce disease-related complications and improve outcomes.
Kanuma is an enzyme replacement therapy used to treat LAL deficiency, a rare genetic disorder affecting fat metabolism.
Key facts at a glance
- RxNorm
- 1726980
- Generic
- Kanuma
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Kanuma is indicated for the treatment of Lysosomal Acid Lipase (LAL) deficiency, which includes the severe infantile form (Wolman disease) and the less severe later-onset form (cholesteryl ester storage disease). It helps manage the accumulation of fats in various organs and tissues caused by this enzyme deficiency.
Kanuma contains sebelipase alfa, a recombinant form of the human lysosomal acid lipase enzyme. It works by replacing the deficient or defective LAL enzyme in patients with LAL deficiency, allowing for the proper breakdown of fats (cholesteryl esters and triglycerides) within cells and reducing the harmful accumulation of these substances in organs and tissues.
Kanuma is administered via intravenous infusion, typically every other week. As it is a protein-based therapy, there is a risk of infusion-related reactions, and patients should be monitored during administration. It is specifically indicated for patients with confirmed LAL deficiency based on genetic and/or biochemical testing. Common side effects may include diarrhea, vomiting, fever, and nasal congestion.
Official drug label
WellAlly may earn a referral fee from linked resources at no extra cost to you. Editorial content is never influenced by commercial relationships.
This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy