Cipaglucosidase Alfa: Uses, Side Effects, Dosage - Drugvu

ℹ️ Compiled from public regulatory records · Last regulator revision: February 27, 2026🚩 Report this page

Cipaglucosidase Alfa

Enzymes

Sold as POMBILITI

CA Health CanadaEU EMAGB MHRA

Drug class: Enzymes
Availability: See label
Routes: Intravenous
Markets covered: 3
Products on record: 3

Overview

Cipaglucosidase Alfa is an active pharmaceutical ingredient in the Enzymes group (A16AB). The information below is compiled per regulator from the product labels on record, with direct links to the original documents.

Regulatory status by market

Market	Regulator	Products	Last revision
CA Canada	Health Canada	1	December 16, 2025
EU European Union	EMA	1	November 22, 2024
GB United Kingdom	MHRA	1	February 27, 2026

CACanada· Health Canada

1 product

Uses

CAOfficial regulatory label· revised December 16, 2025[1]

POMBILITI® (cipaglucosidase alfa for injection) is indicated: • in combination with the enzyme stabiliser OPFOLDA (65 mg miglustat capsule) for the treatment of adult patients with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency) weighing ≥ 40 kg.
POMBILITI must be used in combination with 65 mg miglustat capsules. Consult the Product Monograph of OPFOLDA (65 mg miglustat capsule) for detailed information. 1 Pediatrics Pediatrics (< 18 years of age): No data are available to Health Canada; therefore, Health Canada has not authorized an indication for pediatric use.
3 Pharmacokinetics).

How to take

EUEuropean Union· EMA

1 product

Uses

EUOfficial regulatory label· revised November 22, 2024[2]

Pombiliti (cipaglucosidase alfa) is a 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).

How to take

EU

GBUnited Kingdom· MHRA

1 product

1 product on record with this regulator. Detailed label text (uses, dosage, side effects) is being ingested — the original document is linked under Sources [3].

Sources & citations

[1]Health Canada (DPD) · 02556898 · revised December 16, 2025
[2]European Medicines Agency · EMEA/H/C/005703 · revised November 22, 2024
[3]MHRA (UK) · PLGB258230003 · revised February 27, 2026

Information on this page is compiled from public regulatory records. Drugvu is not affiliated with any regulator or pharmaceutical manufacturer. This is not medical advice. Always consult a qualified healthcare professional.

CAOfficial regulatory label· revised December 16, 2025[1]

1 Dosing Considerations • POMBILITI must be used in combination with 65 mg miglustat capsules. Refer to the Product Monograph of OPFOLDA (65 mg miglustat capsule) prior to reconstituting POMBILITI for the number of capsules (based on body weight), dose time, fasting, and dose adjustments for patients with renal impairment.
1 Pregnancy). • Premedication and/or treatment during infusion with corticosteroids, oral antihistamines, and antipyretics may be administered to assist with signs and symptoms related to IARs (see 2 CONTRAINDICATIONS and 7 WARNINGS AND PRECAUTIONS).
• Patient response to treatment should be routinely evaluated based on a comprehensive evaluation of all clinical manifestations of the disease. In the case of an insufficient response or intolerable safety risks, discontinuation of POMBILITI in combination with 65 mg miglustat capsules treatment should be considered (see 7 WARNINGS AND PRECAUTIONS).
Both medicinal products should either be continued or discontinued together. • Vials are single-use only. Any unused product should be discarded. 2 Recommended Dose and Dosage Adjustment POMBILITI must be used in combination with 65 mg miglustat capsule.
Refer to the Product Monograph of OPFOLDA (65 mg miglustat capsule) for detailed information on dosage and administration recommendations for 65 miglustat capsules. 4 Administration). The POMBILITI infusion should start 1 hour after taking 65 mg miglustat capsules (see Figure 1 for the dosing timeline).
In the event of infusion delay, the start of infusion should not exceed 3 hours from taking miglustat. POMBILITI® (cipaglucosidase alfa for injection) Page 6 of 35 Figure 1 - Dose Timeline * In the event of POMBILITI infusion delay, the start of infusion should not exceed 3 hours from taking 65 miglustat capsules.
, approximately 2 weeks after the last ERT administration). Patients who have switched from another ERT to the POMBILITI-miglustat therapy should be advised to continue with any premedications used with the previous ERT therapy to minimise IARs.
Depending on tolerability, premedication may be modified. Patients with renal and hepatic impairment The safety and efficacy of POMBILITI in combination with 65 mg miglustat capsules therapy have not been evaluated in patients with renal and/or hepatic impairment and no specific dose adjustments for POMBILITI can be recommended for these patients.
Refer to the Product Monograph of OPFOLDA (65 mg miglustat capsule) for the effects of renal impairment on miglustat exposures and miglustat dosage reduction recommendations in patients with moderate or severe renal impairment. 3 Special Populations and Conditions -Geriatrics).
Pediatric use (<18 years) Health Canada has not authorized an indication for pediatric use (see 1 INDICATIONS). 3 Reconstitution Preparation before the infusion • Use aseptic technique. • Each vial of POMBILITI is for single-use only.
Calculating the dose Determine the number of POMBILITI vials to be reconstituted based on patient’s body weight. 1. Patient’s body weight (kg) x dose (mg/kg) = Patient dose (mg) 2. Patient’s dose (in mg) divided by 105 (mg per vial) = Number of vials to reconstitute • If the number of vials includes a fraction, round up to the next whole number.
0 mL. 38 vials and round up to 13 vials. • Remove 7 mL from each of the first 12 vials. 7 mL from the 13th vial. , approximately 30 minutes at 20°C to 25°C). • Do not use if the vial is chipped, cracked, has fluid, lyophilized powder is discolored, closure is damaged, or button of overseal is removed.
Reconstituting the lyophilized powder 1. 2 mL sterile water for injection dropwise down the inside of the vial rather than directly onto the lyophilized powder. Avoid forceful impact of sterile water for injection on the lyophilized powder and avoid foaming.
2. Tilt and roll each vial gently to dissolve the powder. Do not invert, swirl, or shake. Reconstitution of the lyophilized powder typically takes 2 minutes. 3. Perform an inspection of the reconstituted vials for particulate matter and discoloration.
The reconstituted volume appears as a colorless to slightly yellow solution, clear to opalescent, and appears practically free of particles in the form of white to translucent visible particles in a vial. If upon immediate inspection, foreign matter is observed […]

This is not medical advice. Consult a qualified healthcare professional.

Side effects & warnings

CAOfficial regulatory label· Adverse reactions· revised December 16, 2025[1]

1 Adverse Reaction Overview The most common adverse reactions (≥ 5%) reported in POMBILITI in combination with miglustat treated subjects in all 3 studies were headache, diarrhoea, fatigue, nausea, abdominal pain, pyrexia, and chills.
7%). 2 Clinical Trial Adverse Reactions Clinical trials are conducted under very specific conditions. Therefore, the frequencies of adverse reactions observed in the clinical trial may not reflect frequencies observed in clinical practice and should not be compared to frequencies reported in the clinical trials of another drug.
The pooled safety analysis from 3 clinical trials included 151 adult patients with LOPD treated with POMBILITI in combination with miglustat including: • 85 patients in the randomized, double-blind, active-controlled trial in adults, • 37 patients in the open-label extension trial where patients switched from an approved alglucosidase alfa product to POMBILITI in combination with miglustat, • 29 patients in an open-label trial.
The assessment of adverse reactions was informed by subjects treated with POMBILITI in combination with miglustat across 3 clinical trials. 0 months. Phase 3 clinical trial (PROPEL) included 123 adult patients with LOPD who were randomized to receive treatment with POMBILITI in combination with miglustat or alglucosidase alfa in combination with placebo.
Adverse reactions from the Phase 3 clinical trial (PROPEL) are listed by MedDRA system organ class in Table 3. 4%)* 0 * Related to POMBILITI/alglucosidase alfa only ** Related to miglustat/Placebo only ‡ Adverse reactions that are medically related were grouped to a single preferred term.
1 Abdominal pain, abdominal pain upper, and abdominal pain lower are grouped under abdominal pain. POMBILITI® (cipaglucosidase alfa for injection) Page 16 of 35 2 Rash, rash erythematous and rash macular are grouped under rash. 3 Urticaria, mechanical urticaria, and urticaria rash are grouped under urticaria.
4 Tachycardia and sinus tachycardia are grouped under tachycardia. 5 Headache, migraine, and migraine with aura are grouped under headache. Infusion-Associated reactions (IARs) The following IARs were reported in at least 2 subjects in the Phase 3 clinical trial (PROPEL) during the POMBILITI infusion or within 2 hours after completion of the infusion: abdominal distension, chills, pyrexia, dizziness, dysgeusia, dyspnea, pruritus, rash, and flushing.
7% of patients experienced a serious adverse reaction of anaphylaxis (characterized by generalised pruritus, dyspnea, and hypotension) during the PROPEL study receiving POMBILITI and miglustat. 3% of patients receiving POMBILITI and miglustat discontinued treatment due to IARs (anaphylaxis and chills).
Most IARs were mild or moderate in severity, transient in nature and none were assessed as life--threatening or fatal. Most subjects who experienced IARs were able to continue treatment with POMBILITI in combination with miglustat. 3 Less Common Clinical Trial Adverse Reactions Adverse reactions from the clinical trials reported in less than 2% of patients from pooled analysis of 3 clinical trials were: Gastrointestinal disorders: abdominal discomfort†, constipation†, dyspepsia*, oesophageal pain*, oesophageal spasm, oral discomfort*, oral pain, swollen tongue* General disorders and administration site conditions: asthenia, chest discomfort*, facial pain, feeling jittery†, infusion site pain*, infusion site swelling*, malaise*, non-cardiac chest pain, pain*, peripheral swelling, swelling face* Immune system disorders: anaphylactic reaction‡2, hypersensitivity Injury, poisoning and procedural complications: skin abrasion* Investigations: body temperature fluctuation*, lymphocyte count decreased, platelet count decreased† Musculoskeletal and connective tissue disorders: arthralgia, flank pain, muscle fatigue, muscular weakness, musculoskeletal stiffness Nervous system disorders: balance disorder, burning sensation*, dysgesusia, migraine‡1, paraesthesia, presyncope* Respiratory, thoracic and mediastinal disorders: asthma, cough*, oropharyngeal discomfort*, pharyngeal oedema*, wheezing* Skin and subcutaneous tissue disorder: skin discolouration, skin oedema* Vascular disorders: hypotension, pallor * Related to cipaglucosidase alfa only; † Related to miglustat only; ‡ Adverse reactions that are medically related were grouped to a single preferred term; 1 Migraine and migraine with aura are grouped under migraine; 2 Anaphylactic reaction, Anaphylaxis are grouped under Anaphylactic reaction.
Anaphylactoid reaction is manually coded to Anaphylaxis. 4 Abnormal Laboratory Findings: Hematologic, Clinical Chemistry and Other Quantitative Data There were no clinically significant […]

CAOfficial regulatory label· Warnings and precautions· revised December 16, 2025[1]

, Immune, Anaphylaxis and Infusion Associated Reactions). For a complete listing, see 6 DOSAGE FORMS, STRENGTHS, COMPOSITION AND PACKAGING. • Patients with a contraindication to 65 mg miglustat capsule including: o Patients who are hypersensitive to miglustat or to any ingredient in the formulation, including any non-medicinal ingredient, or component of the container.
For a complete listing, refer to the Product Monograph of OPFOLDA (65 mg miglustat capsule) o Women who are or may become pregnant. If POMBILITI in combination with 65 mg miglustat capsules are administered to women of reproductive potential, they should be informed of the potential hazard to the fetus.
1 Pregnancy). 3 SERIOUS WARNINGS AND PRECAUTIONS BOX Serious Warnings and Precautions Hypersensitivity Reactions Including Anaphylaxis • Patients treated with POMBILITI have experienced life-threatening hypersensitivity reactions, including anaphylaxis.
Appropriate medical support measures, including cardiopulmonary POMBILITI® (cipaglucosidase alfa for injection) Page 5 of 35 resuscitation equipment, should be readily available during POMBILITI administration. , anaphylaxis) occurs, infusion should be immediately paused, appropriate medical treatment initiated, and the benefits and risks of re-administration of POMBILITI should be considered (see 7 WARNINGS AND PRECAUTIONS).
Infusion-Associated Reactions (IARs) • Patients treated with POMBILITI have experienced severe IARs. If severe IARs occur, infusion should be immediately paused, appropriate medical treatment initiated, and the benefits and risks of re-administration of POMBILITI should be considered following severe IARs (see 7 WARNINGS AND PRECAUTIONS).
Risk of Acute Cardiorespiratory Failure in Susceptible Patients • 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 due to fluid volume overload during POMBILITI infusion.
Appropriate medical support and monitoring measures should be readily available during POMBILITI infusion (see 7 WARNINGS AND PRECAUTIONS). 1 Dosing Considerations • POMBILITI must be used in combination with 65 mg miglustat capsules.

This is not medical advice. Consult a qualified healthcare professional.

Side effects & warnings

EUOfficial regulatory label· Adverse reactions· revised November 22, 2024[2]

8. Premedication with oral antihistamine, antipyretics, and/or corticosteroids may be administered to assist with signs and symptoms related to IARs experienced with prior ERT treatment. Reduction of the infusion rate, temporary interruption of the infusion, symptomatic treatment with oral antihistamine, or antipyretics, and appropriate resuscitation measures should be considered to manage serious IARs.
Mild to moderate and transient IARs may be adequately managed by slowing the infusion rate or interrupting the infusion; medical treatment or discontinuation of cipaglucosidase alfa may not be required. 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. The risks and benefits of re-administering cipaglucosidase alfa following anaphylaxis or severe allergic reaction should be carefully considered, and appropriate resuscitation measures made available if the decision is made to readminister the medicinal product.
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. Risk of acute cardiorespiratory failure in susceptible patients 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 cipaglucosidase alfa infusion. Immune complex-related reactions Immune complex-related reactions have been reported with other ERTs in patients who had high IgG antibody titres, including severe cutaneous reactions and nephrotic syndrome.
A potential class effect cannot be excluded. Patients should be monitored for clinical signs and symptoms of systemic immune complex-related reactions while receiving cipaglucosidase alfa with miglustat. If immune complex-related reactions occur, discontinuation of the administration of cipaglucosidase alfa should be considered and appropriate medical treatment should be initiated.
The risks and benefits of re- 6 administering cipaglucosidase alfa following an immune complex-related reaction should be reconsidered for each individual patient. 5 mg sodium per vial. 52% of the WHO recommended maximum daily intake of 2 g sodium for an adult.
5 Interaction with other medicinal products and other forms of interaction No interaction studies have been performed related to the use of cipaglucosidase alfa or with cipaglucosidase alfa in combination with miglustat. As cipaglucosidase alfa is a recombinant human protein, it is an unlikely candidate for cytochrome P450 or P-gP mediated interactions with other medicinal products.
3). The medicinal product is not recommended in women of childbearing potential not using reliable contraception. Pregnancy There are no clinical data from the use of cipaglucosidase alfa in combination with miglustat in pregnant women.
3). Cipaglucosidase alfa in combination with miglustat therapy is not recommended during pregnancy. Breast-feeding It is not known if cipaglucosidase alfa and miglustat are secreted in human breast milk. 3). A risk to newborns/infants cannot be excluded.
A decision must be made whether to discontinue breast-feeding or to discontinue/abstain from cipaglucosidase alfa in combination with miglustat therapy taking into account the benefit of breast-feeding for the child and the benefit of therapy for the woman.
Fertility There are no clinical data on the effects of cipaglucosidase alfa alone or in combination with miglustat on fertility. 3). 3). 7 Effects on ability to drive and use machines Cipaglucosidase alfa has minor influence on the ability to drive and to use machines since dizziness, hypotension, and somnolence have been reported as adverse reactions.
Caution is required when driving or using any tools or machines after receiving cipaglucosidase alfa. 3%). 7%). Tabulated list of adverse reactions The assessment of adverse reactions was informed by subjects treated with cipaglucosidase alfa in combination with miglustat therapy from the pooled safety analysis of the 3 clinical trials.
0 […]

EUOfficial regulatory label· Warnings and precautions· revised November 22, 2024[2]

4. Both medicinal products should either be continued or discontinued. e. approximately 2 weeks after the last ERT administration). Patients who have switched from another ERT to cipaglucosidase alfa in combination with miglustat therapy should be advised to continue with any premedications used with the previous ERT therapy to minimise infusion-associated reactions (IARs).
4). Missed dose If the cipaglucosidase alfa infusion cannot be started within 3 hours of oral administration of miglustat, reschedule treatment of cipaglucosidase alfa and miglustat at least 24 hours after taking miglustat. If cipaglucosidase alfa and miglustat are both missed, treatment should occur as soon as possible.
Special populations Elderly There is limited experience with the use of cipaglucosidase alfa in combination with miglustat therapy in patients above the age of 65 years old. 2). 4 Renal and hepatic impairment The safety and efficacy of cipaglucosidase alfa in combination with miglustat therapy have not been evaluated in patients with renal and/or hepatic impairment.
When administering every other week, increased plasma miglustat exposure as a result of moderate or severe renal or hepatic impairment is not expected to appreciably impact cipaglucosidase alfa exposures and is not anticipated to affect efficacy and safety of cipaglucosidase alfa in a clinically meaningful manner.
No dose adjustment is required in patients with renal impairment. The safety and efficacy of cipaglucosidase alfa in patients with hepatic impairment have not been evaluated and no specific dose regimen can be recommended for these patients.
Paediatric population The safety and efficacy of cipaglucosidase alfa in combination with miglustat therapy in paediatric patients less than 18 years old have not yet been established. No data are available. Method of administration Cipaglucosidase alfa is to be administered by intravenous infusion.
Infusion of the 20 mg/kg dose is normally administered over the course of 4 hours if tolerated. Infusion should be administered in a stepwise manner. An initial cipaglucosidase alfa infusion rate of 1 mg/kg/hr is recommended. This infusion rate may be gradually increased by 2 mg/kg/hr approximately every 30 minutes if there are no signs of IARs until a maximum infusion rate of 7 mg/kg/hr is reached.

This is not medical advice. Consult a qualified healthcare professional.