FABRAZYME- agalsidase beta injection, powder, lyophilized, for solution

국가: 미국

언어: 영어

출처: NLM (National Library of Medicine)

유효 성분:

AGALSIDASE BETA (UNII: RZD65TSM9U) (AGALSIDASE BETA - UNII:RZD65TSM9U)

제공처:

Genzyme Corporation

INN (International Name):

AGALSIDASE BETA

구성:

AGALSIDASE BETA 5 mg in 1 mL

관리 경로:

INTRAVENOUS

처방전 유형:

PRESCRIPTION DRUG

치료 징후:

FABRAZYME® is indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease. None. Risk Summary Available data from a pregnancy sub-study within the Fabry Disease registry, post-marketing case reports, and case series with FABRAZYME use during pregnancy have not identified a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes (see Data). Reproduction studies performed in rats at doses up to 68 times the human dose have revealed no evidence of effects on embryo-fetal development (see Data) . The background risk of major birth defects and miscarriage for 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. Data Human Data Available data from a pregnancy sub-study within the Fabry Disease registry, post-marketing case reports, and case series with FABRAZYME use during pregnancy have not identified a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes. In the Fabry Disease registry pregnancy sub-study, 33 pregnancies exposed to FABRAZYME prior to or during pregnancy had a known outcome; 5 were reported as exposed in the first trimester. Animal Data The effects of agalsidase beta on embryo-fetal development in rats were evaluated at doses of 3, 10, and 30 mg/kg/day (up to 68 times the human dose of 1 mg/kg every 2 weeks on a body surface area basis) during gestation days 7 to 17. Hepatocellular necrosis consistent with accumulation of test article was evident in maternal livers in the 10 and 30 mg/kg/day groups (23 and 68 times the human dose on a body surface area basis). There were no adverse effects of agalsidase beta on embryo-fetal development in rats. Risk Summary The available human data detected small amounts of agalsidase beta in human milk. Available data from the clinical study, global pharmacovigilance database, and published scientific literature are insufficient to determine the effects of FABRAZYME on the breastfed infant or on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for FABRAZYME and any potential adverse effects on the breastfed child from FABRAZYME or from the underlying maternal condition. The safety and effectiveness of FABRAZYME have been established in pediatric patients based on adequate and well-controlled studies in adults, a single-arm, open-label study in 16 pediatric patients with Fabry disease aged 8 to 16 years, and additional data in 24 patients with Fabry disease aged 2 to 7 years [see Clinical Pharmacology (12.2) and Clinical Studies (14)] . The overall safety profile of FABRAZYME was similar between the pediatric and the adult population [see Adverse Reactions (6.1) and Clinical Studies (14)] . Clinical studies of FABRAZYME did not include sufficient numbers of subjects aged 65 years and older to determine whether they respond differently from younger subjects.

제품 요약:

FABRAZYME (agalsidase beta) for injection is supplied as a sterile, nonpyrogenic, white to off-white lyophilized cake or powder in single-dose vials. 35 mg vial: NDC 58468-0040-1 5 mg vial: NDC 58468-0041-1 Refrigerate vials of FABRAZYME at 2°C to 8°C (36°F to 46°F). Do not use FABRAZYME after the expiration date on the vial. This product contains no preservatives. Reconstituted and diluted solutions of FABRAZYME should be used immediately. If immediate use is not possible, the reconstituted and diluted solution may be stored for up to 24 hours at 2°C to 8°C (36°F to 46°F) [see Dosage and Administration (2.2)] .

승인 상태:

Biologic Licensing Application

제품 특성 요약

FABRAZYME- AGALSIDASE BETA INJECTION, POWDER, LYOPHILIZED, FOR
SOLUTION
GENZYME CORPORATION
----------
HIGHLIGHTS OF PRESCRIBING INFORMATION
THESE HIGHLIGHTS DO NOT INCLUDE ALL THE INFORMATION NEEDED TO USE
FABRAZYME SAFELY AND EFFECTIVELY. SEE FULL
PRESCRIBING INFORMATION FOR FABRAZYME .
FABRAZYME (AGALSIDASE BETA) FOR INJECTION, FOR INTRAVENOUS USE
INITIAL U.S. APPROVAL: 2003
RECENT MAJOR CHANGES
Warning and Precautions (5.1)
2/2024
INDICATIONS AND USAGE
FABRAZYME is a hydrolytic lysosomal neutral glycosphingolipid-specific
enzyme indicated for the treatment of adult and pediatric
patients 2 years of age and older with confirmed Fabry disease. (1)
DOSAGE AND ADMINISTRATION
Consider administering antihistamines, antipyretics, and/or
corticosteroids prior to FABRAZYME administration. (2.1)
The recommended dosage is 1 mg/kg body weight given every two weeks as
an intravenous infusion. (2.2)
See the full prescribing information for rechallenge, preparation,
storage, and administration instructions. (2.3, 2.4, 2.5, 2.6)
DOSAGE FORMS AND STRENGTHS
For injection: 5 mg or 35 mg of agalsidase beta as a lyophilized cake
or powder in a single-dose vial for reconstitution. (3)
CONTRAINDICATIONS
None. (4)
WARNINGS AND PRECAUTIONS
_Hypersensitivity Reactions Including Anaphylaxis_: Life-threatening
hypersensitivity reactions, including anaphylaxis, have occurred
during FABRAZYME infusions. If a severe hypersensitivity (e.g.,
anaphylaxis) reaction occurs, discontinue FABRAZYME immediately
and initiate appropriate medical treatment. Rechallenge of these
patients should only occur under the direct supervision of qualified
personnel with appropriate medical monitoring and support measures
readily available. (5.1)
_Infusion-Associated Reactions_: If a severe infusion-associated
reaction occurs, discontinue FABRAZYME immediately and initiate
appropriate medical treatment. (5.2)
ADVERSE REACTIONS
Most common adverse reactions (≥20%) are: upper respiratory tract
infection, chills, pyrexia, headache, cough, paresthesia, fatigue,
pe