Pegunigalsidase alfa
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From MedlinePlus, U.S. National Library of Medicine
Boxed warning
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with ELFABRIO have experienced hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during ELFABRIO administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue ELFABRIO immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reaction, a desensitization procedure to ELFABRIO may be considered [see Warnings and Precautions ( 5.1 )] .
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, discontinue ELFABRIO immediately and initiate appropriate medical treatment. ( 5.1 )
Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme
Dosing in kidney impairment
Patients may be rechallenged using slower infusion rates. Once a patient tolerates the infusion, the infusion rate may be increased to reach the recommended infusion rate. If a mild or moderate IAR occurs, consider temporarily holding the infusion or slowing the infusion rate [see Dosage and Administration] . 5.3 Membranoproliferative Glomerulonephritis A case of membranoproliferative glomerulonephritis with immune depositions in the kidney was reported during clinical trials [see Adverse Reactions ] .
This event led to a decline in renal function that slowly improved upon discontinuation of ELFABRIO but did not return to baseline by the end of the trial. Monitor serum creatinine and urinary protein to creatinine ratio. If glomerulonephritis is suspected, discontinue ELFABRIO until a diagnostic evaluation can be conducted.
Preparation and administration
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Storage of the Diluted Solution If the diluted ELFABRIO solution is not used immediately: ○ Refrigerate the diluted solution at 2°C to 8°C (36°F to 46°F) for up to 24 hours. The solution must be infused within 8 hours after removal from the refrigerator, inclusive of the total infusion time, or discarded. ○ Store the diluted solution at room temperature at 20°C to 25°C (68°F to 77°F) for up to 8 hours.
The solution must be used within 8 hours, inclusive of infusion time, or discarded. Do not freeze or shake. 2. 6 Administration Instructions Administer ELFABRIO as follows: Use an in-line low protein-binding, 0.2 micron, in-line filter during administration. For the initial 4-6 infusions, infuse ELFABRIO using the infusion rates described in Table 1 for ERT-experienced patients and Table 2 for ERT-naïve patients.
If a patient tolerates the initial 4-6 ELFABRIO infusions, the duration of every third infusion may be decreased in decrements of 30 minutes as tolerated. The minimum recommended infusion duration is 1.5 hours. At the end of the infusion, flush the line with 0.9% Sodium Chloride Injection using the same infusion rate as the one used for the last part of the ELFABRIO infusion.
Do not infuse ELFABRIO in the same intravenous line with other products. Table 1 presents the recommended infusion rates for the initial 4-6 ELFABRIO infusions based on actual body weight for ERT-experienced patients. [See table below.] 1 Infusion rate may be increased if the patient tolerates the initial 4-6 infusions (see above).
Infusion rate may be slowed in case of a hypersensitivity reaction or an IAR [see Dosage and Administration]. Table 2 presents the recommended infusion rates for the initial 4-6 ELFABRIO infusions based on actual body weight for ERT-naïve patients. [See table below.] 1 Infusion rate may be increased if the patient tolerates the initial 4-6 infusions (see above).
Infusion rate may be slowed in case of a hypersensitivity reaction or an IAR [see Dosage and Administration]. Administration of ELFABRIO to Patients Previously Treated with ERT w ith an Infusion Duration Over 3 Hours In patients previously treated with an ERT with an infusion duration over 3 hours: Use the same infusion rate for the ELFABRIO infusion.
May decrease the duration of every third infusion after the initial 4-6 ELFABRIO infusions in decrements of 30 minutes as tolerated. The recommended minimum infusion duration of the maintenance infusion is 1.5 hours. Home Infusion Home administration under the supervision of a healthcare provider may be considered for patients who have reached an infusion duration that is tolerated well [see Dosage and Administration ( 2.1 , 2.3 )] .
The decision to have a patient move to home infusion should be made after evaluation and recommendation by a healthcare provider. The infusion duration should remain constant for home administration and the duration should only be decreased in a healthcare facility. In case of a missed dose or delayed infusion, a healthcare provider should be contacted.
| Actual Body Weight | Total Infusion Volume | Infusion Rate1 |
| ˂ 70 kg | 150 mL | 0.83 mL/min (50 mL/h) |
| 70 -100 kg | 250 mL | 1.39 mL/min (83 mL/h) |
| > 100 kg | 500 mL | 2.78 mL/min (167 mL/h) |
| Actual Body Weight | Total Infusion Volume | Infusion Rate1 |
| ˂ 70 kg | 150 mL | 0.63 mL/min (37.5 mL/h) |
| 70 -100 kg | 250 mL | 1 mL/min (60 mL/h) |
| > 100 kg | 500 mL | 1.38 mL/min (83 mL/h) |
Contraindications
None. None. ( 4 )
Pregnancy and lactation
Pregnancy
8.1 Pregnancy Risk Summary There are no available data on ELFABRIO use in pregnant females to evaluate a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes; however, as an enzyme replacement, ELFABRIO is not expected to cause adverse outcomes . Animal reproduction studies have been conducted with pegunigalsidase alfa-iwxj in pregnant rats and rabbits. No adverse effects on embryofetal development were observed in pregnant rats intravenously administered pegunigalsidase alfa-iwxj twice per week at exposures up to 3.6 times that of the maximum recommended human dose (MRHD) (based on area under the concentration-time curve (AUC)).
Maternal toxicity was observed in pregnant rabbits intravenously administered pegunigalsidase alfa-iwxj twice per week at doses that were ≥ 3.2 times the MRHD (based on human equivalent dose) [ see Data ] . The estimated background risk of major birth defects and miscarriage in the indicated population is unknown. All pregnancies have a background risk of birth defect, loss or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. There is a pregnancy safety study for ELFABRIO.
If a patient becomes pregnant while receiving ELFABRIO, healthcare providers should report ELFABRIO exposure by calling 1-888-661-9260 or visiting https://chiesirarediseases.com/contact-us/medical-information-form. Data Animal Data In an embryofetal development study in the rat, pegunigalsidase alfa-iwxj was administered during the period of organogenesis on gestation day 6, 9, 12, and 15. No maternal or fetal adverse effects were noted at exposures that were up to 3.6-fold greater than the recommended dose of 1 mg/kg every two weeks.
In an embryofetal development study in the rabbit, administration of pegunigalsidase alfa-iwxj during the period of organogenesis on gestation day 6, 9, 12, 15, and 18, resulted in maternal toxicity, including maternal mortality, decreased body weight, and decreased feed consumption. These effects were observed at exposures that were ≥ 3.2-fold greater than the recommended dose of 1 mg/kg every two weeks. Adverse embryofetal effects included abortion, increased late resorptions, number of does with resorptions, and increased post-implantation loss at exposures that were 6.5 fold greater than the recommended dose of 1 mg/kg every two weeks.
Decreased fetal body weight was observed at exposures that were ≥ 3.2 times greater than the…
For breastfeeding, also see LactMed (National Library of Medicine), which summarizes milk levels and infant effects.
Supply and cost
Drug shortage status
Recalls (last 24 months)
Therapeutic equivalence (Orange Book)
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