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Elfabrio is a brand name for Pegunigalsidase Alfa. The medicine, its uses, side effects and dosage are the same regardless of brand.
Used for: Elfabrio is indicated for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase).
Verbatim from this product's EMA label. Tap a section to expand.
Pegunigalsidase alfa treatment must be managed by a physician experienced in the treatment of patients with Fabry disease. Appropriate medical support measures should be readily available when pegunigalsidase alfa is administered to patients who have not had treatment before, or who have experienced severe hypersensitivity reactions to pegunigalsidase alfa in the past.
4). Posology The recommended dose of pegunigalsidase alfa is 1 mg/kg of body weight administered once every two weeks. The treatment can be also administered at the dose of 2 mg/kg of body weight once every four weeks in patients stable with an ERT treatment (see section
2% of patients. 5%) experienced a serious reaction that was considered related to pegunigalsidase alfa. Four of these reactions were confirmed IgE-mediated hypersensitivity (bronchospasm, hypersensitivity) that occurred at the first infusion of pegunigalsidase alfa and resolved within the day after occurrence.
Tabulated summary of adverse reactions The data described below reflects data from 141 patients with Fabry disease who received pegunigalsidase alfa in 8 clinical studies, following the posology of 1 mg/kg every two weeks or 2 mg/kg every four weeks for a minimum of 1 infusion up to 7 years.
Adverse reactions are listed in Table 3. Information is presented by system organ class. Frequencies are defined as: very common (≥ 1/10); common (≥ 1/100 to < 1/10); uncommon (≥ 1/1 000 to < 1/100); rare (≥ 1/10 000 to < 1/1 000); very rare (< 1/10 000); frequency not known (cannot be estimated from available data).
Table 3:
Adverse reactions reported during treatment with pegunigalsidase alfa System organ class Frequency Common Uncommon Immune system disorders hypersensitivity* type I hypersensitivity* Psychiatric disorders agitation* insomnia Nervous system disorders paraesthesia* dizziness* headache* restless legs syndrome peripheral neuropathy neuralgia burning sensation tremor* syncope* Ear and labyrinth disorders vertigo Vascular disorders flushing hypotension* hypertension* lymphoedema 8 System organ class Frequency Common Uncommon Respiratory, thoracic and mediastinal disorders bronchospasm* sneezing* nasal congestion* dyspnoea* throat irritation* throat tightness Gastrointestinal disorders nausea* abdominal pain* diarrhoea vomiting* gastrooesophageal reflux disease gastritis dyspepsia flatulence Skin and subcutaneous issue disorders rash* erythema* pruritus* hypohidrosis Musculoskeletal and connective tissue disorders arthralgia musculoskeletal pain* Renal and urinary disorders membranoproliferative glomerulonephritis chronic kidney disease proteinuria Reproductive system and breast disorders nipple pain General disorders and administration site conditions asthenia* chills* chest pain* pain* influenza-like illness infusion site extravasation infusion site pain oedema Investigations body temperature increased* urine protein/creatinine ratio increased white blood cells urine positive blood uric acid increased weight increased Injury, poisoning and procedural complications infusion related reaction* Cardiac disorders supraventricular extrasystoles The following preferred terms have been grouped in Table 3: • hypersensitivity includes: drug hypersensitivity • agitation includes: nervousness • abdominal pain includes: abdominal discomfort • rash includes: rash maculo-papular and rash pruritic • musculoskeletal stiffness recorded as musculoskeletal pain includes: myalgia • asthenia includes: malaise and fatigue • chest pain includes: chest discomfort and non-cardiac chest pain • pain includes: pain in extremity • oedema peripheral recorded as oedema * Preferred terms considered as IRR as described in the section below.
4 Treatment monitoring). 6. Patients switching treatment from agalsidase alfa or beta For the initial 3 months of treatment with pegunigalsidase alfa, pre-treatment regimen should be preserved with stepwise discontinuation of pre-treatment based on appropriate tolerability of the patients.
Special populations Hepatic impairment No dose adjustment is needed in patients with hepatic impairment. Renal impairment No dose adjustment is needed in patients with renal impairment. 4). Elderly (≥ 65 years old) Safety and efficacy of pegunigalsidase alfa in patients older than 65 years have not been evaluated and no alternative dose regimens can be recommended for these patients.
1. Paediatric population The safety and efficacy of pegunigalsidase alfa in children and adolescents aged 0-17 years have not yet been established. No data are available. Method of administration For intravenous infusion use only. Pegunigalsidase alfa must not be infused in the same intravenous line with other products.
6. 2 μm filter. 4. 6. Home administration Infusion of pegunigalsidase alfa at home may be considered if the patient is tolerating infusions well and has no history of moderate or severe IRRs for a few months. 4 The decision to move to home infusion should be made after evaluation and recommendation by the treating physician.
The patient should be medically stable. Home infusion infrastructure, resources, and procedures, including training, must be established and available to the healthcare professional in charge of home infusion. The healthcare professional should be available at all times during the home infusion and for a specified time after infusion.
Appropriate training should be given by the treating physician and/or nurse to the patient and/or caregiver prior to initiation of home infusion. The dose and infusion rate used in the home setting should remain the same as was used in the hospital setting; they should be changed only under the supervision of the treating physician.
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Description of selected adverse reactions Infusion related reactions (adverse reactions within 2 hours of infusion) IRRs were reported in a total of 36 patients (25%): 26 patients (23%) treated with 1 mg/kg every two weeks and 10 patients (34%) treated with 2 mg/kg every four weeks.
The most commonly reported symptoms associated with IRRs reported for 1 mg/kg dosage were: hypersensitivity, chills, dizziness, rash and itching. For the 2 mg/kg dose the most commonly reported symptoms were pain, headache and nausea.
IRRs were mostly mild or moderate in intensity and resolved with continuous treatment; however, 5 patients (all male, 1 mg/kg dose) experienced 5 severe IRRs. These 5 IRRs were also 9 serious. Four of these events were confirmed type I hypersensitivity reactions and 3 led to the discontinuation from the study.
Another patient was later withdrawn from the study, after the occurrence of another moderate IRR. All 5 patients however recovered within the day after of occurrence with appropriate treatment. IRRs predominantly occurred within the first year of treatment with pegunigalsidase alfa and no serious IRR was observed during the second year and beyond.
4%) treated with 2 mg/kg pegunigalsidase alfa every four weeks developed treatment-induced anti-drug antibodies (ADAs). Membranoproliferative glomerulonephritis During the clinical development of pegunigalsidase alfa, one patient out of 141 reported a severe event of membranoproliferative glomerulonephritis after receiving treatment for more than 2 years.
The patient was ADA positive at the start of the infusions. The event led to a transitory reduction in the eGFR and an increase on the level of proteinuria, with no additional signs or symptoms. A biopsy revealed the immune-complex mediated nature of this event.
Upon discontinuation of the treatment, the eGFR values stabilised and the glomerulonephritis was reported as resolving. Reporting of suspected adverse reactions Reporting suspected adverse reactions after authorisation of the medicinal product is important.
It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system listed in Appendix V.
67 ml/hr) Maintenance infusion The target infusion duration can be achieved pending patient’s tolerability. The increase in the infusion rate should be achieved gradually starting from the rate given at the first infusion. 33 ml/hr) Maintenance infusion The target infusion duration can be achieved pending patient’s tolerability.
The increase in the infusion rate should be achieved gradually starting from the rate given at the first infusion. 4). 5 Any patients experiencing adverse events during the home infusion need to immediately stop the infusion process and seek the attention of a healthcare professional.
Subsequent infusions may need to occur in a clinical setting. 1. 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.
: after 3, 6, 12, 18 and 24 months) should be performed. Monitoring should include at least the evaluation of lyso-Gb3, renal (eGFR, proteinuria), cardiac (LVMi, NT-proBNP, troponin or ECG), and biochemical parameters. A change in any individual parameter should be interpreted in the context of the patient's overall clinical status, and clinically relevant deterioration should prompt re- evaluation of the treatment regimen.
Infusion related reactions […]