ELFABRIO- pegunigalsidase alfa injection, solution, concentrate

Country: United States

Language: English

Source: NLM (National Library of Medicine)

Active ingredient:

PEGUNIGALSIDASE ALFA (UNII: 8M7V7Q6537) (PEGUNIGALSIDASE ALFA - UNII:8M7V7Q6537)

Available from:

Chiesi USA, Inc.

Administration route:

INTRAVENOUS

Prescription type:

PRESCRIPTION DRUG

Therapeutic indications:

ELFABRIO is indicated for the treatment of adults with confirmed Fabry disease. None. 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 recommended dose of 1 mg/kg every two weeks. There was no increase in fetal external, skeletal, or visceral malformations. Risk Summary There are no data on the presence of pegunigalsidase alfa-iwxj in either human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for ELFABRIO and any potential adverse effects on the breastfed infant from pegunigalsidase alfa-iwxj or from the underlying maternal condition. The safety and effectiveness of ELFABRIO have not been established in pediatric patients. Clinical trials of ELFABRIO did not include patients 65 years of age and older to determine if they respond differently from younger adult patients. Patients that received prior ERT are more likely to have pre-existing anti-drug antibodies (ADA) to pegunigalsidase alfa-iwxj which could be due to the ADA cross-reactivity to pegunigalsidase alfa-iwxj by prior ERT. When switching from other ERT to ELFABRIO: - Pre-existing ADA may reduce the plasma pegunigalsidase alfa-iwxj concentrations, which may reduce ELFABRIO efficacy [see Clinical Pharmacology ( 12.2 , 12.6 ) ]. - The risk of ELFABRIO-related hypersensitivity and infusion-associated reactions may be increased in certain patients with pre-existing ADA from prior ERT [ see Warnings and Precautions ( 5.1 , 5.2 ) and Adverse Reactions ( 6.1 )] . Consider monitoring clinical or pharmacodynamic responses (e.g., plasma lyso-Gb3 levels) when switching from agalsidase beta to ELFABRIO, in patients with pre-existing ADA. 

Product summary:

How Supplied ELFABRIO (pegunigalsidase alfa-iwxj) injection is a sterile, preservative-free, clear and colorless solution supplied in a single-dose vial. Each vial contains 20 mg/10 mL or 5 mg/2.5 mL (2 mg/mL) of pegunigalsidase alfa-iwxj. ELFABRIO is available as: Storage and Handling Store refrigerated at 2°C to 8°C (36°F to 46°F). Do not freeze. Do not shake.

Authorization status:

Biologic Licensing Application

Summary of Product characteristics

ELFABRIO- PEGUNIGALSIDASE ALFA INJECTION, SOLUTION, CONCENTRATE
CHIESI USA, INC.
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HIGHLIGHTS OF PRESCRIBING INFORMATION
THESE HIGHLIGHTS DO NOT INCLUDE ALL THE INFORMATION NEEDED TO USE
ELFABRIO SAFELY AND
EFFECTIVELY. SEE FULL PRESCRIBING INFORMATION FOR ELFABRIO.
ELFABRIO (PEGUNIGALSIDASE ALFA-IWXJ) INJECTION, FOR INTRAVENOUS USE
INITIAL U.S. APPROVAL: 2023
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)
INDICATIONS AND USAGE
ELFABRIO is a hydrolytic lysosomal neutral glycosphingolipid-specific
enzyme indicated for the treatment of
adults with confirmed Fabry disease. (1)
DOSAGE AND ADMINISTRATION
For pretreatment recommendations, see Full Prescribing Information.
(2.1)
Recommended dosage is 1 mg/kg every 2 weeks administered as an
intravenous infusion. (2.2)
For dosage and administration modifications due to hypersensitivity
reactions or infusion-associated
reactions (IARs), see Full Prescribing Information. (2.3)
For instructions on preparation (including dilution), storage, and
administration (including rates for the
initial 4-6 infusions for ERT-experienced and ERT-naïve patients),
see Full Prescribing Information. (2.4,
2.5, 2.6)
DOSAGE FORMS AND STRENGTHS
Injection: 20 mg/10 mL or 5 mg/2.5 mL (2 mg/mL) solution in a
single-dose vial. (3)
CONTRAINDICATIONS
None. (4)
WARNINGS AND PRECAUTIONS
_Infusion-Associated Reactions:_ If severe IARs occur, discontinue
ELFABRIO and initiate appropriate
medical treatment. (5.2)
_Membranoproliferative Glomerulonephritis:_ Monitor serum creatinine
and urinary protein to creatinine
ratio. Discontinue ELFABRIO if glomerulonephritis is suspected, until
a diagnostic evaluation can be
conducted. (5.3)
ADVERSE REACTIONS
Most common adverse reactions (�