Kanuma

Last updated on RxList: 2/16/2021
Kanuma Side Effects Center

Medical Editor: John P. Cunha, DO, FACOEP

What Is Kanuma?

Kanuma (sebelipase alfa) injection is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.

What Are Side Effects of Kanuma?

Common side effects of Kanuma include diarrhea, vomiting, nausea, fever, runny or stuffy nose, low levels of iron in the blood (anemia), cough, sore throat, hives, headache, weakness, constipation, and nausea.

Dosage for Kanuma

The dose of Kanuma injection is 20 mg/10 mL (2 mg/mL) solution in single-use vials.

What Drugs, Substances, or Supplements Interact with Kanuma?

Kanuma may interact with other drugs. Tell your doctor all medications and supplements you use.

Kanuma During Pregnancy and Breastfeeding

It is unknown if Kanuma would affect a fetus. Kanuma should be used during pregnancy only if prescribed. It is unknown if Kanuma passes into breast milk. Consult your doctor before breastfeeding.

Additional Information

Our Kanuma (sebelipase alfa) injection Side Effects Drug Center provides a comprehensive view of available drug information on the potential side effects when taking this medication.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Kanuma Consumer Information

Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Some side effects may occur during the injection. Tell your caregiver right away if you feel itchy, flushed, chilled or feverish, or if you have a runny nose, eye irritation, diarrhea, chest discomfort, or trouble breathing.

Call your doctor at once if you have:

  • severe skin rash, itching, or swelling;
  • agitation, irritability;
  • stomach pain; or
  • low red blood cells (anemia)--pale skin, feeling light-headed or short of breath, rapid heart rate, trouble concentrating.

Common side effects may include:

  • fever, weakness;
  • runny nose, sinus pain, sore throat, cough;
  • rash;
  • diarrhea, constipation;
  • nausea, vomiting; or
  • headache.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Read the entire detailed patient monograph for Kanuma (Kanuma Sebelipase Alfa)

Kanuma Professional Information

SIDE EFFECTS

Clinical Trials Experience

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice.

In clinical trials, a total of 106 patients received treatment with KANUMA. The data described below reflect exposure to KANUMA in 75 patients who received KANUMA at dosages up to 3 mg/kg once weekly in clinical trials:

  • Nine patients (5 males, 4 females) who had growth failure or other evidence of rapidly progressive LAL deficiency presenting within the first 6 months of life received KANUMA for up to 165 weeks (median 60 weeks) at escalating doses ranging between 0.35 mg/kg and 5 mg/kg once weekly [see Clinical Studies]. The recommended initial dosage for these patients is 1 mg/kg escalating to 3 mg/kg once weekly [see DOSAGE AND ADMINISTRATION].
  • 66 pediatric and adult patients with LAL deficiency aged 4 to 58 years (33 males, 33 females) received KANUMA 1 mg/kg every other week up to 36 weeks.

Table 2 summarizes the most common adverse reactions occurring in >30% of patients with rapidly progressive LAL deficiency presenting within the first 6 months of life receiving KANUMA.

Table 2: Most Common Adverse Reactions* in Patients with Rapidly Progressive LAL Deficiency Presenting within the First 6 Months of Life

Adverse ReactionsKANUMA
N=9 n (%)
Diarrhea6 (67)
Vomiting6 (67)
Fever5 (56)
Rhinitis5 (56)
Anemia4 (44)
Cough3 (33)
Nasopharyngitis3 (33)
Urticaria3 (33)
*Reported in more than 30% of patients receiving KANUMA

Other less common adverse reactions reported in patients with rapidly progressive disease presenting within the first 6 months of life who received KANUMA included hypotonia, decreased oxygen saturation, retching, sneezing, and tachycardia.

Table 3 summarizes the most common adverse reactions that occurred in ≥8% of pediatric and adult patients with LAL deficiency receiving KANUMA at a dosage of 1 mg/kg once every other week during the 20-week double-blind treatment period [see Clinical Studies].

Table 3: Most Common Adverse Reactions* in Pediatric and Adult Patients with LAL Deficiency

Adverse ReactionsKANUMA
N = 36
n (%)
Placebo
N = 30
n (%)
Headache10 (28)6 (20)
Fever9 (25)7 (23)
Oropharyngeal pain6 (17)1 (3)
Nasopharyngitis4 (11)3 (10)
Asthenia3 (8)1 (3)
Constipation3 (8)1 (3)
Nausea3 (8)2 (7)
*Reported in at least 8% of pediatric and adult patients receiving KANUMA and at a higher incidence than in patients receiving placebo

Other less common adverse reactions reported in pediatric and adult patients who received KANUMA included anxiety and chest discomfort.

Hyperlipidemia

Increases in circulating LDL-cholesterol (LDL-c) and triglycerides above pre-treatment values were observed in 29 of 36 (81%) and 21 of 36 (58%) patients, respectively, at 2 and 4 weeks following initiation of KANUMA [see CLINICAL PHARMACOLOGY]. The maximum mean percentage increase was 18% for LDL-c at Week 2 and 5% for triglycerides at Week 4.

Immunogenicity

As with all therapeutic proteins, there is potential for immunogenicity. Patients have developed anti-drug antibodies (ADA) to KANUMA. Immunogenicity assay results are highly dependent on the sensitivity and specificity of the assay and may be influenced by several factors such as: assay methodology, sample handling, timing of sample collection, concomitant medications, and underlying disease. For these reasons, comparison of the incidence of antibodies to KANUMA with the incidence of antibodies to other products may be misleading.

Patients With Rapidly Progressive LAL Deficiency Presenting Within The First 6 Months Of Life

Seven of the 9 infants with rapidly progressive disease had at least one post-treatment ADA assessment, and 4 of these 7 (57%) patients developed ADA during treatment with KANUMA. Two of the 4 ADA-positive patients were determined to be positive for neutralizing antibodies that inhibit in vitro enzyme activity and cellular uptake of the enzyme. At the time of initial ADA positivity, 3 patients were receiving a dosage of 1 mg/kg once weekly and 1 patient was receiving a dosage of 3 mg/kg once weekly. Three of the 4 ADA-positive patients had ADA titers monitored from the initiation of treatment, and developed measureable ADA titers within the first 2 months of exposure. One of the 4 ADA-positive patients had persistent ADA titers. ADA titers decreased to undetectable levels in the remaining 3 patients while receiving continued treatment at a dosage of 3 mg/kg once weekly.

Hypersensitivity reactions occurred in all 4 of the ADA-positive patients, whereas they occurred in only 1 of the 3 ADA-negative patients. None of the patients discontinued treatment. In 1 patient, decreased growth velocity in a setting of neutralizing antibodies to KANUMA was observed.

Pediatric And Adult Patients With LAL Deficiency

Five of 35 (14%) KANUMA-treated pediatric and adult patients who completed the 20-week double-blind period of study treatment developed ADA. All patients were receiving 1 mg/kg once every other week. All 5 ADA-positive patients first developed measurable ADA titers within the first 3 months of exposure. Two of the 5 ADA-positive patients had a measurable ADA titer at only one time point. In the 3 patients with measurable ADA titers at multiple time points, ADA titers decreased to undetectable levels during continued treatment. Two patients developed in vitro neutralizing antibodies during the open-label extension phase after 20 weeks and 52 weeks of treatment with KANUMA, respectively. There is no clear association between the development of ADA and decreased efficacy in pediatric and adult patients treated with KANUMA.

Read the entire FDA prescribing information for Kanuma (Kanuma Sebelipase Alfa)

© Kanuma Patient Information is supplied by Cerner Multum, Inc. and Kanuma Consumer information is supplied by First Databank, Inc., used under license and subject to their respective copyrights.

Health Solutions From Our Sponsors