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POMBILITI is a brand name for Cipaglucosidase Alfa, supplied as a powder for solution. The medicine, its uses, side effects and dosage are the same regardless of brand.
Used for: 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…
Verbatim from this product's HC label. Tap a section to expand.
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.
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.
, 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.
, anaphylaxis and severe cutaneous reactions) to cipaglucosidase alfa, or to any ingredient in the formulation, including any non-medicinal ingredient, or component of the container when rechallenge was unsuccessful (see 7 WARNINGS AND PRECAUTIONS, 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).
Not medical advice. Always read the patient information leaflet and follow your prescriber or pharmacist.
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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 […]
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 […]
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.
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 […]