Source: European Medicines Agency (EU) Revision Year: 2023 Publisher: Chiesi Farmaceutici S.p.A., Via Palermo 26/A, 43122 Parma, Italy
Elfabrio is indicated for long-term enzyme replacement therapy in adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase).
Elfabrio 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 Elfabrio is administered to patients who have not had treatment before, or who have experienced severe hypersensitivity reactions to Elfabrio in the past.
Pre-treatment with antihistamines and/or corticosteroids may be advisable for patients who had previously experienced hypersensitivity reactions to Elfabrio or to another enzyme replacement therapies (ERT) treatment (see section 4.4).
The recommended dose of pegunigalsidase alfa is 1 mg/kg of body weight administered once every two weeks.
For instructions on reconstitution, see section 6.6.
For the initial 3 months (6 infusions) of treatment with Elfabrio, pre-treatment regimen should be preserved with stepwise discontinuation of pre-treatment based on appropriate tolerability of the patients.
No dose adjustment is needed in patients with renal or hepatic impairment.
Safety and efficacy of Elfabrio in patients older than 65 years have not been evaluated and no alternative dose regimens can be recommended for these patients. Elderly patients may be treated with the same dose as other adult patients, see section 5.1.
The safety and efficacy of Elfabrio in children and adolescents aged 0-17 years have not yet been established. No data are available.
For intravenous infusion use only.
Elfabrio must not be infused in the same intravenous line with other products.
For instructions on dilution of the medicinal product before administration, see section 6.6.
After preparation, the dilution should be administered via intravenous infusion and filtered through an in-line low protein-binding 0.2 μm filter.
The patient should be observed for infusion-related reactions (IRRs) for two hours after the infusion; see section 4.4.
Further details on how to handle Elfabrio before administration, see section 6.6.
Infusion of Elfabrio at home may be considered if the patient is tolerating his infusions well and have no history of moderate or severe IRRs for a few months.
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.
Table 1. Recommended dose and infusion time for intravenous administration of Elfabrio:
| Initial infusion 1 mg/kg of body weight every 2 weeks | |||
| Body weight (Kg) | Total volume (mL) | Infusion time | Infusion rate* |
| up to 70 | 150 mL | not less than 3 hours | 0.83 mL/min (50 mL/hr) |
| 70-100 | 250 mL | not less than 3 hours | 1.39 mL/min (83.33 mL/hr) |
| >100 | 500 mL | not less than 3 hours | 2.78 mL/min (166.67 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. | |||
| 1 mg/kg of body weight every 2 weeks | |||
| Body weight (Kg) | Total volume (mL) | Infusion time | Infusion rate* |
| up to 70 | 150 mL | not less than 1.5 hours | 1.68 mL/min (100 mL/hr) |
| 70-100 | 250 mL | not less than 1.5 hours | 2.78 mL/min (166.67 mL/hr) |
| >100 | 500 mL | not less than 1.5 hours | 5.56 mL/min (333.33 mL/hr) |
* infusion rate may be adjusted in case of infusion reaction (see section 4.4)
If patients experience infusion-related reactions, including hypersensitivity reactions or anaphylactic reactions during the infusion, the infusion must be immediately stopped and appropriate medical treatment should be initiated (see section 4.4).
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.
There are no reports of overdose of Elfabrio during clinical studies. The maximum dose of Elfabrio studied was 2 mg/kg body weight every two weeks and no specific signs and symptoms were identified following the higher doses. The most common adverse reactions reported were infusion related reaction and pain in extremity. If overdose is suspected, seek emergency medical attention.
4 years.
Diluted solution for infusion:
Chemical and physical in use stability has been demonstrated for 72 hours both at 2°C-8°C and below 25°C.
From a microbiological point of view, the product should be used immediately. If not used immediately, in-use storage times and conditions prior to use are the responsibility of the user and would normally not be longer than 24 hours in the refrigerator (2°C-8°C) or 8 hours if stored below 25°C, unless dilution has taken place in controlled and validated aseptic conditions.
Store in a refrigerator (2°C-8°C).
For storage conditions after dilution of the medicinal product, see section 6.3.
10 mL vial (15R clear glass) closed with coated rubber stopper and sealed with aluminium flip off cap.
Pack sizes of 1, 5 or 10 vials.
Not all pack sizes may be marketed.
Elfabrio is for intravenous infusion only. Aseptic technique to be used. Vials are for single use only.
If contamination is suspected, the vial has not to be used. Shaking or agitating this medicinal product must be avoided.
Filter needles do not have to be used during the preparation of the infusion. The number of vials to be diluted should be determined based on the individual patient’s weight and the required vials should be removed from the refrigerator in order to allow them to reach room temperature (in approximately 30 minutes).
Dilution:
1) Determine the total number of vials required for the infusion. The number of vials required is based on the total dose required for each individual patient and requires calculation for weight-based dosing.
An example calculation for total dose in an 80 kg patient prescribed 1 mg/kg is as follows:
2) Allow the required number of vials to reach room temperature prior to dilution (approximately 30 minutes).
Visually inspect the vials. Do not use if cap is missing or broken. Do not use if there is particulate matter or if it is discoloured.
Avoid shaking or agitating the vials.
3) Remove and discard the same volume as calculated in step 1 of sodium chloride 9 mg/mL (0.9%) solution for infusion from the infusion bag.
4) Withdraw the required volume of Elfabrio solution from the vials, and dilute with sodium chloride 9 mg/mL (0.9%) solution for infusion, to a total volume based on patient weight specified in Table 4 below.
Table 4. Minimum total infusion volume for patients by body weight:
| Patient weight | Minimum total infusion volume |
|---|---|
| <70 kg | 150 mL |
| 70–100 kg | 250 mL |
| >100 kg | 500 mL |
Inject the Elfabrio solution directly into the infusion bag.
Do NOT inject in the airspace within the infusion bag.
Gently invert the infusion bag to mix the solution, avoiding vigorous shaking and agitation.
The diluted solution should be administered using an inline low protein binding 0.2 μm filter.
Disposal:
Any unused medicinal product or waste material must be disposed of in accordance with local requirements.
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