POMBILITI

What it is

POMBILITI is cipaglucosidase alfa 105 mg/7 mL powder for injection vial. Cipaglucosidase alfa is a hydrolytic glycogen-specific enzyme, produced by recombinant DNA methodology derived from Chinese Hamster Ovary (CHO) cell line using perfusion methodology, resulting in cellularly (CHO)-derived N-glycans. This genetic technology deliberately creates a recombinant enzyme with a very similar structure to the natural form of human acid α-glucosidase (rhGAA) enzyme. Cipaglucosidase alfa degrades glycogen by catalysing the hydrolysis of α-1,4- and α-1,6-glycosidic linkages of lysosomal glycogen.

Approved indications

— Long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).

Dosing overview

The recommended dose of POMBILITI is 20 mg/kg body weight administered every other week as an intravenous solution for IV administration. Infusion of the 20 mg/kg dose is normally administered over the course of 4 hours if tolerated. The initial infusion rate should be no more than 1 mg/kg/hr for 30 minutes. The infusion rate may be increased by 2 mg/kg/hr every 30 minutes after the patient's tolerance to the infusion rate is established, until a maximum rate of 7 mg/kg/hr is reached. POMBILITI must be used in combination with miglustat 65 mg capsules.

Key safety warnings

Serious anaphylaxis and infusion-associated reactions (IARs) have occurred in some patients during infusion and following infusion with POMBILITI. If anaphylaxis or severe allergic reactions occur, infusion should be immediately paused, and appropriate medical treatment should be initiated. The current medical standards for emergency treatment of anaphylactic reactions are to be observed and cardiopulmonary resuscitation equipment should be readily available. If a patient experiences anaphylaxis or severe allergic reactions in the home setting, and if the patient continues therapy, their next infusions must occur in a clinical setting, equipped to deal with such medical emergencies. Patients with acute underlying respiratory illness or compromised cardiac and/or respiratory function may be at risk of serious exacerbation of their cardiac or respiratory compromise during infusions. Appropriate medical support and monitoring measures should be readily available during POMBILITI infusion. Immune complex-related reactions have been reported with other enzyme replacement therapies in patients who had high IgG antibody titres, including severe cutaneous reactions and nephrotic syndrome. A potential class effect cannot be excluded.

Contraindications

— Patients with a history of life-threatening infusion-associated reactions (eg, anaphylaxis and severe cutaneous reactions) to the active substance, or to any of the excipients, when rechallenge was unsuccessful. — Patients with a contraindication to miglustat.

Regulatory history

POMBILITI cipaglucosidase alfa 105 mg powder for injection vial was first registered on the ARTG on 17 February 2025. The approval date was 10 February 2025. POMBILITI was approved as a long-term enzyme replacement therapy, in combination with miglustat, for the treatment of adults with late-onset Pompe disease, based on a favourable efficacy and safety profile. This medicinal product is subject to additional monitoring in Australia. This will allow quick identification of new safety information.