OPFOLDA- miglustat capsule

Država: Sjedinjene Američke Države

Jezik: engleski

Izvor: NLM (National Library of Medicine)

Aktivni sastojci:

MIGLUSTAT (UNII: ADN3S497AZ) (MIGLUSTAT - UNII:ADN3S497AZ)

Dostupno od:

AMICUS THERAPEUTICS US, LLC

Administracija rute:

ORAL

Tip recepta:

PRESCRIPTION DRUG

Terapijske indikacije:

OPFOLDA is indicated, in combination with Pombiliti, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). OPFOLDA in combination with Pombiliti is contraindicated in Pregnancy [see Use in Specific Populations (8.1)] Risk Summary Based on findings from animal reproduction studies, OPFOLDA in combination with Pombiliti may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. In a rabbit embryo-fetal development study, great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with miglustat in combination with cipaglucosidase alfa-atga at 3-fold and 16-fold, respectively, the MRHD of OPFOLDA and Pombiliti based on plasma AUC exposure. A No Observed Adverse Effect Level (NOAEL) was not identified for the combination. In a pre- and post-natal development study in rats, inc

Proizvod sažetak:

How Supplied OPFOLDA (miglustat) capsules are supplied as 65 mg, white opaque hard gelatin capsules with a grey opaque cap with “AT2221” printed in black on the body, and are available in bottles with child resistant caps. See Table 5 for the available OPFOLDA packages. Table 5. OPFOLDA Packages Storage and Handling Store at 20°C to 25°C (68°F to 77°F). Excursions are permitted between 15°C to 30°C (59°F to 86°F), [see USP Controlled Room Temperature]. Store in the original container to protect from light.

Status autorizacije:

New Drug Application

Svojstava lijeka

OPFOLDA- MIGLUSTAT CAPSULE
AMICUS THERAPEUTICS US, LLC
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HIGHLIGHTS OF PRESCRIBING INFORMATION
THESE HIGHLIGHTS DO NOT INCLUDE ALL THE INFORMATION NEEDED TO USE
OPFOLDA™ SAFELY AND
EFFECTIVELY. SEE FULL PRESCRIBING INFORMATION FOR OPFOLDA.
OPFOLDA (MIGLUSTAT) CAPSULES, FOR ORAL USE
INITIAL U.S. APPROVAL: 2003
INDICATIONS AND USAGE
OPFOLDA is an enzyme stabilizer indicated, in combination with
Pombiliti, a hydrolytic lysosomal glycogen-
specific enzyme, for the treatment of adult patients with late-onset
Pompe disease (lysosomal acid alpha-
glucosidase [GAA] deficiency) weighing ≥40 kg and who are not
improving on their current enzyme
replacement therapy (ERT). (1)
DOSAGE AND ADMINISTRATION
Verify pregnancy status in females of reproductive potential prior to
initiating treatment. (2.1)
Administer OPFOLDA in combination with Pombiliti. (2.2)
Recommended OPFOLDA dosage (based on actual body weight), administered
orally every other week,
is: (2.2)
260 mg for patients weighing ≥50 kg.
195 mg for patients weighing ≥40 kg to <50 kg.
Start OPFOLDA in combination with Pombiliti 2 weeks after the last ERT
dose. (2.2)
Take OPFOLDA with an unsweetened beverage approximately 1 hour before
the start of Pombiliti
infusion; do not consume other beverages or food for at least 2 hours
prior to and 2 hours after taking
OPFOLDA. (2.2)
Missed dose: If the OPFOLDA dosage is missed, Pombiliti should not be
administered and treatment
should be rescheduled at least 24 hours after OPFOLDA was last taken.
If OPFOLDA in combination with
Pombiliti are both missed, re-start treatment as soon as possible.
(2.2)
See full prescribing information for recommended OPFOLDA dosage in
patients with renal impairment.
(2.3)
DOSAGE FORMS AND STRENGTHS
Capsules: 65 mg (3)
CONTRAINDICATIONS
Pregnancy. (4, 5.1, 8.1)
WARNINGS AND PRECAUTIONS
_Embryo-Fetal Toxicity:_ May cause embryo-fetal harm. Advise females
of reproductive potential of the
potential risk to a fetus and to use effective contraception during
treatment and for at least 60 da