KANUMA

What it is

Kanuma is sebelipase alfa rce supplied as a single-use vial containing 20 mg of sebelipase alfa rce (2 mg/mL). It is a concentrated solution for intravenous infusion. Sebelipase alfa rce is a recombinant human lysosomal acid lipase produced by recombinant DNA technology and purified from the egg white of genetically engineered chickens.

Approved indications

— Long-term enzyme replacement therapy in patients of all ages with lysosomal acid lipase deficiency (LAL-D).

Dosing overview

For infants presenting with rapidly progressive LAL-D within the first 6 months of life, the recommended starting dose is 1 mg/kg or 3 mg/kg administered as an intravenous infusion once weekly depending on the clinical status of the patient. A dose escalation to 3 mg/kg should be considered in case of suboptimal clinical response, and a further dose escalation up to 5 mg/kg may be considered in case of persistent suboptimal clinical response. For children and adults presenting with LAL-D, the recommended dose is 1 mg/kg administered every two weeks as an intravenous infusion. Dose escalation to 3 mg/kg once every two weeks could be considered based on suboptimal response to clinical and biochemical criteria. The total volume of the infusion should be administered over approximately 2 hours. Infusion over 1 hour may be considered for those patients receiving the 1 mg/kg dose after patient tolerability is established.

Key safety warnings

Hypersensitivity reactions, including anaphylactic reactions or anaphylaxis, have been reported in Kanuma-treated patients. In clinical trials, 5 of 125 (4%) patients treated with Kanuma, including 3 of 19 (16%) infants and 2 of 106 (2%) children and adults, experienced serious signs and symptoms consistent with anaphylaxis. Anaphylaxis has occurred as late as 1 year after treatment initiation. The majority of reactions occurred during or within 4 hours of the completion of the infusion. Appropriate medical support should be readily available when Kanuma is administered. If anaphylaxis occurs, immediately discontinue the infusion and initiate appropriate medical treatment. Sebelipase alfa rce is produced in the egg white of transgenic chickens. Consider the risks and benefits of treatment with Kanuma in patients with known life-threatening hypersensitivity to egg.

Contraindications

Kanuma is contraindicated in patients with a life-threatening hypersensitivity (anaphylactic reaction) to the active substance, to egg, or to any of the excipients, when attempts to rechallenge are unsuccessful.

Regulatory history

Kanuma was first listed on the Australian Register of Therapeutic Goods on 18 May 2017. The TGA approved Kanuma for long-term enzyme replacement therapy in patients of all ages with lysosomal acid lipase deficiency on 17 May 2017, based on the drug's demonstrated medical value and long-term safety as the first enzyme replacement therapy for this rare, life-threatening condition. In March 2022, the PBAC recommended that Kanuma not be listed for infantile onset lysosomal acid lipase deficiency.