LUMIZYME- alglucosidase alfa injection, powder, for solution

Pays: États-Unis

Langue: anglais

Source: NLM (National Library of Medicine)

Ingrédients actifs:

ALGLUCOSIDASE ALFA (UNII: DTI67O9503) (ALGLUCOSIDASE ALFA - UNII:DTI67O9503)

Disponible depuis:

Genzyme Corporation

DCI (Dénomination commune internationale):

ALGLUCOSIDASE ALFA

Composition:

ALGLUCOSIDASE ALFA 5 mg in 1 mL

Mode d'administration:

INTRAVENOUS

Type d'ordonnance:

PRESCRIPTION DRUG

indications thérapeutiques:

LUMIZYME® is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase [GAA] deficiency). None. Risk Summary Data from postmarketing reports and published case reports with alglucosidase alfa use in pregnant women have not identified a LUMIZYME-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. The continuation of treatment for Pompe disease during pregnancy should be individualized to the pregnant woman. Untreated Pompe disease may result in worsening disease symptoms in pregnant women [see Clinical Considerations] . Reproduction studies performed in mice and rabbits at doses resulting in exposures up to 0.4 or 0.5 times the human steady-state AUC (area under the plasma concentration-time curve), respectively, during the period of organogenesis revealed no evidence of effects on embryo-fetal development. In mice there was an increase in pup mortality during lactation at maternal exposures 0.4 times the human steady-state AUC [see Data] . The 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. Pregnant women and women of reproductive potential should be encouraged to enroll in the Pompe patient registry. The registry will monitor the effect of LUMIZYME on pregnant women and their offspring. For more information, visit www.registrynxt.com or call 1-800-745-4447, extension 15500. Clinical Considerations Disease-associated Maternal and/or Embryo-fetal Risk Untreated Pompe disease has been associated with worsening respiratory and musculoskeletal symptoms in some pregnant women. Data Animal Data All reproductive studies included pretreatment with diphenhydramine to prevent or minimize hypersensitivity reactions. The effects of alglucosidase alfa were evaluated based on comparison to a control group treated with diphenhydramine alone. Daily intravenous administration of alglucosidase alfa up to 40 mg/kg in mice and rabbits (0.4 and 0.5 times the human steady-state AUC, respectively, at the recommended biweekly dose) during the period of organogenesis had no effects on embryo-fetal development. Administration of 40 mg/kg intravenously every other day in mice (0.4 times the human steady-state AUC at the recommended biweekly dose) during the period of organogenesis through lactation produced an increase in mortality of offspring during the lactation period. Risk Summary Available published literature suggests the presence of alglucosidase alfa in human milk. There are no reports of adverse effects of alglucosidase alfa on the breastfed infant. There is no information on the effects of alglucosidase alfa on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for LUMIZYME and any potential adverse effects on the breastfed child from LUMIZYME or from the underlying maternal condition. Lactating women with Pompe disease treated with LUMIZYME should be encouraged to enroll in the Pompe disease registry [see Use in Specific Populations (8.1)] . Clinical Considerations A lactating woman may consider interrupting breastfeeding, pumping and discarding breast milk during treatment and for 24 hours after LUMIZYME administration in order to minimize drug exposure to a breastfed infant. The safety and effectiveness of alglucosidase alfa have been established in pediatric patients with Pompe disease [see Adverse Reactions (6.2)] . The safety and effectiveness of alglucosidase alfa were assessed in 57 treatment-naive infantile-onset Pompe disease patients, aged 0.2 month to 3.5 years at first infusion, in three separate clinical trials [see Clinical Studies (14.1)] . The safety and effectiveness of alglucosidase alfa were assessed in pediatric patients with late (non-infantile) onset Pompe disease in a randomized, double-blind, placebo-controlled study in 90 patients, including 2 patients 16 years of age or less [see Clinical Studies (14.2)] . Anaphylaxis, hypersensitivity reactions, and acute cardiorespiratory failure have occurred in pediatric patients [see Boxed Warning, Warnings and Precautions (5.1, 5.3)] . Additionally, cardiac arrhythmia and sudden cardiac death have occurred in pediatric patients during general anesthesia for central venous catheter placement [see Warnings and Precautions (5.4)] . The randomized, double-blind, placebo-controlled study of alglucosidase alfa did not include sufficient numbers (n=4) of patients aged 65 years and over to determine whether they respond differently from younger patients [see Clinical Studies (14.1)] .

Descriptif du produit:

LUMIZYME 50 mg vials are supplied as a sterile, nonpyrogenic, preservative-free, white to off-white lyophilized cake or powder in single-dose vials. NDC 58468-0160-1 (Carton of one single-dose vial) NDC 58468-0160-2 (Carton of ten single-dose vials) Store LUMIZYME under refrigeration between 2°C and 8°C (36°F and 46°F). Do not use LUMIZYME after the expiration date on the vial.

Statut de autorisation:

Biologic Licensing Application

Résumé des caractéristiques du produit

LUMIZYME- ALGLUCOSIDASE ALFA INJECTION, POWDER, FOR SOLUTION
GENZYME CORPORATION
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HIGHLIGHTS OF PRESCRIBING INFORMATION
THESE HIGHLIGHTS DO NOT INCLUDE ALL THE INFORMATION NEEDED TO USE
LUMIZYME SAFELY AND
EFFECTIVELY. SEE FULL PRESCRIBING INFORMATION FOR LUMIZYME.
LUMIZYME (ALGLUCOSIDASE ALFA), FOR INJECTION, FOR INTRAVENOUS USE
INITIAL U.S. APPROVAL: 2010
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, IMMUNE-
MEDIATED REACTIONS, AND RISK OF ACUTE CARDIORESPIRATORY FAILURE
_SEE FULL PRESCRIBING INFORMATION FOR COMPLETE BOXED WARNING._
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
APPROPRIATE MEDICAL MONITORING AND SUPPORT MEASURES, INCLUDING
CARDIOPULMONARY
RESUSCITATION EQUIPMENT, SHOULD BE READILY AVAILABLE. IF A SEVERE
REACTION OCCURS,
DISCONTINUE LUMIZYME IMMEDIATELY AND INITIATE APPROPRIATE MEDICAL
TREATMENT. (5.1)
IMMUNE-MEDIATED REACTIONS
IMMUNE-MEDIATED REACTIONS PRESENTING AS PROTEINURIA, NEPHROTIC
SYNDROME, AND
NECROTIZING SKIN LESIONS HAVE OCCURRED IN SOME PATIENTS FOLLOWING
LUMIZYME
TREATMENT. MONITOR PATIENTS FOR THE DEVELOPMENT OF SYSTEMIC
IMMUNE-MEDIATED
REACTIONS INVOLVING SKIN AND OTHER ORGANS WHILE RECEIVING LUMIZYME.
(5.3)
RISK OF ACUTE CARDIORESPIRATORY FAILURE
INFANTILE-ONSET POMPE DISEASE PATIENTS WITH COMPROMISED CARDIAC OR
RESPIRATORY
FUNCTION MAY BE AT RISK OF SERIOUS ACUTE EXACERBATION OF THEIR CARDIAC
OR RESPIRATORY
COMPROMISE DUE TO FLUID OVERLOAD AND REQUIRE ADDITIONAL MONITORING.
(5.4)
RECENT MAJOR CHANGES
Boxed Warnings
3/2024
Dosage and Administration (2.1, 2.2)
3/2024
Warnings and Precautions (5.1, 5.2)
3/2024
INDICATIONS AND USAGE
LUMIZYME is a hydrolytic lysosomal glycogen-specific enzyme indicated
for patients with Pompe disease
(GAA deficiency). (1)
DOSAGE AND ADMINISTRATION
20 mg per kg body weight administered every 2 weeks as an intravenous
infusion. (2)
DOSAGE FORMS AND STRENGTHS
For injection: 50 mg of LUMIZYME as lyophilized powder in a
single-dose vial for reconstitution. (3)
CONTRAINDICATIONS
None. (4)
WARNINGS AND PRECAUTIONS
Infusion Associa