Kanuma is a novel medication approved for treatment of lysosomal acid lipase deficiency (LAL-D), a very rare genetic condition. LAL-D happens when the person inherits a gene that results in a deficiency of lysosomal acid lipase, which works by breaking down fats in the body.
Infants born with LAL-D accumulate fat in the intestines, leading to malabsorption resulting to stunted growth and failure to thrive. As the patient grows older, fat accumulates in the liver resulting in liver failure.
Sebelipase Alfa replaces lysosomal acid lipase missing in cells to improve overall symptoms.
As of the time of this writing, Sebelipase Alfa is a relatively new medicine. It was approved as an orphan drug on December 2015 in the United States.
Sebelipase Alfa requires a prescription.
2 What to know before using
Before using Kanuma, you must know all about the risks and complications associated with it.
Sebelipase Alfa is not indicated for patients with allergies to chicken eggs. Sebelipase Alfa is synthesized from chicken eggs and contains a significant amount of egg proteins. If you are allergic to chicken eggs, you cannot use Sebelipase Alfa.
Sebelipase Alfa can be given to children and infants aged at least one month old.
The safety of Sebelipase Alfa in pregnant and breastfeeding patients is not well understood.
Make sure to tell your doctor if you are pregnant or breastfeeding before receiving Sebelipase Alfa.
To use Kanuma properly, you must follow all instructions given by your doctor.
Sebelipase Alfa reduces liver enzymes within 2 weeks, while it takes around 8 weeks for it to lower LDL levels and triglycerides.
Sebelipase Alfa is an injectable drug, and it is given by infusing it into the vein (intravenous route).
Sebelipase Alfa is usually infused once every other week. Sebelipase Alfa must be infused very slowly, and infusion must be done over at least 1 or 2 hours.
During infusion of Sebelipase Alfa, the patient is closely observed to check for any hypersensitivity reactions.
If you miss an appointment for injection of Sebelipase Alfa, call the doctor right away.
Sebelipase Alfa is given by health professionals, so overdosing is very unlikely.
4 Precautions to take
Before using Kanuma, there are some precautions you must take.
Call your health providers if you experienced any symptoms during and after infusion of Sebelipase Alfa. If you experienced moderate allergic reactions, the doctor might have to halt infusion temporarily and give it later at a slower rate.
The doctor may order you to undergo regular blood and lab tests while having treatment with Sebelipase Alfa.
This helps the doctor know if the medicine is working and to detect early signs of adverse effects. Make sure to stick to clinic and lab appointments.
If you became pregnant or breastfeed while receiving Sebelipase Alfa, call the doctor right away.
5 Potential side effects
As with many medications, there are several potential side effects associated with Kanuma.
Call the doctor right away if you experienced serious side effects such as:
Severe allergic reaction such as hives, difficulty breathing, hoarseness, skin itching, swelling of the mouth, lips or tongue
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