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Pombiliti is a brand name for Cipaglucosidase Alfa. The medicine, its uses, side effects and dosage are the same regardless of brand.
Used for: 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).
Verbatim from this product's EMA label. Tap a section to expand.
Treatment should be supervised by a physician experienced in the management of patients with Pompe disease or other inherited metabolic or neuromuscular diseases. Cipaglucosidase alfa must be used in combination with miglustat 65 mg hard capsules.
Because of this, the summary of product characteristics (SmPC) for miglustat 65 mg hard capsules should be consulted before taking cipaglucosidase alfa concerning number of capsules (based on body weight), dose time, and fasting. 3 Posology The recommended dose of cipaglucosidase alfa is 20 mg/kg of body weight every other week.
The Pombiliti infusion should start 1 hour after taking miglustat capsules. In the event of infusion delay, the start of infusion should not exceed 3 hours from taking miglustat. Figure 1. Dose timeline * The cipaglucosidase alfa infusion should start 1 hour after taking miglustat capsules.
In the event of infusion delay, the start of infusion should not exceed 3 hours from taking miglustat. Patient response to treatment should be routinely evaluated based on a comprehensive evaluation of all clinical manifestations of the disease.
In case of an insufficient response or intolerable safety risks, discontinuation of cipaglucosidase alfa in combination with miglustat treatment should be considered, see section
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.
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.
8). Contraindication to miglustat.
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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.
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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.
The rate of infusion should be guided by the patient’s previous experience during infusion. The infusion rate may be slowed or temporarily stopped in the event of mild to moderate IARs. 4). Home infusion Infusion of cipaglucosidase alfa at home may be considered for patients who are tolerating their infusions well and have no history of moderate or severe IARs for a few months.
The decision to have a patient move to home infusion should be made after evaluation and upon recommendation by the treating physician. A patient’s underlying co-morbidities and ability to adhere to the home infusion requirements need to be taken into account when evaluating the patient for eligibility to receive home infusion.
The following criteria should be considered: • The patient must have no ongoing concurrent condition that, in the opinion of the physician, may affect patient’s ability to tolerate the infusion. • The patient is considered medically stable.
A comprehensive evaluation must be completed before the initiation of home infusion. • The patient must have received cipaglucosidase alfa infusions supervised by a physician with expertise in management of Pompe patients for a few months that could be in a hospital or in another appropriate setting of outpatient care.
Documentation of a pattern of well-tolerated infusions is a prerequisite for the initiation of home infusion. • The patient must be willing and able to comply with home infusion procedures. • Home infusion infrastructure, resources, and procedures, including training, must be established and available to the healthcare professional.
The healthcare professional should be always available during the home infusion and for a specified time after infusion, depending on patient’s tolerance prior to starting home infusion. 4). Subsequent infusions may need to occur in a hospital or in an appropriate setting of outpatient care until no such adverse reaction is present.
Dose and infusion rate must not be changed without consulting the responsible physician. 5 The reconstituted product prior to dilution appears as a clear to opalescent colourless to slightly yellow solution. 6. 8). Contraindication to miglustat.
4 Special warnings and precautions for use Traceability In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded. Anaphylaxis and infusion-associated reactions Serious anaphylaxis and IARs have occurred in some patients during infusion and following […]