ORFADIN- nitisinone suspension

País: Estats Units

Idioma: anglès

Font: NLM (National Library of Medicine)

ingredients actius:

NITISINONE (UNII: K5BN214699) (NITISINONE - UNII:K5BN214699)

Disponible des:

Swedish Orphan Biovitrum AB (publ)

Designació comuna internacional (DCI):

nitisinone

Composición:

nitisinone 4 mg in 1 mL

Vía de administración:

ORAL

tipo de receta:

PRESCRIPTION DRUG

indicaciones terapéuticas:

ORFADIN ® is indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. None. Risk Summary Limited available data with nitisinone use in pregnant women are not sufficient to determine a drug-associated risk of adverse developmental outcomes. Animal reproduction studies have been conducted for nitisinone. In these studies, nitisinone was administered to mice and rabbits during organogenesis with oral doses of nitisinone up to 20 and 8 times respectively, the recommended initial dose of 1 mg/kg/day. In mice, nitisinone caused incomplete skeletal ossification of fetal bones and decreased pup survival at doses 0.4 times the recommended initial dose, and increased gestational length at doses 4 times the recommended initial dose. In rabbits, nitisinone caused maternal toxicity and incomplete skeletal ossification of fetal bones at doses 1.6 times the recommended initial dose

Resumen del producto:

Capsules: White capsules marked in black with "NTBC" and identified as 2 mg, 5 mg, 10 mg or 20 mg strengths of nitisinone. The capsules are packed in a high density (HD) polyethylene container with a tamper-resistant low density (LD) polyethylene snap-on cap. Each bottle contains 60 capsules. Oral suspension: White, slightly viscous opaque suspension. 1 mL contains 4 mg of nitisinone. The suspension is provided in a 100 mL brown bottle (type III glass) with a white child resistant HDPE screw cap with sealing and tamper evidence. Each bottle contains 90 mL oral suspension.

Estat d'Autorització:

New Drug Application

Fitxa tècnica

ORFADIN- NITISINONE SUSPENSION
SWEDISH ORPHAN BIOVITRUM AB (PUBL)
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HIGHLIGHTS OF PRESCRIBING INFORMATION
THESE HIGHLIGHTS DO NOT INCLUDE ALL THE INFORMATION NEEDED TO USE
ORFADIN SAFELY AND
EFFECTIVELY. SEE FULL PRESCRIBING INFORMATION FOR ORFADIN.
ORFADIN (NITISINONE) CAPSULES, FOR ORAL USE
ORFADIN (NITISINONE) ORAL SUSPENSION
INITIAL U.S. APPROVAL: 2002
RECENT MAJOR CHANGES
Warnings and Precautions ( 5.1)
05/2019
INDICATIONS AND USAGE
ORFADIN is a hydroxy-phenylpyruvate dioxygenase inhibitor indicated
for the treatment of adult and
pediatric patients with hereditary tyrosinemia type 1 (HT-1) in
combination with dietary restriction of
tyrosine and phenylalanine. ( 1)
DOSAGE AND ADMINISTRATION
Recommended Dosage ( 2.1) :
The recommended starting dosage is 0.5 mg/kg orally twice daily.
In patients 5 years of age and older who have undetectable serum and
urine succinylacetone
concentrations after a minimum of 4 weeks on a stable dosage of
nitisinone, the total daily dose may
be given once daily.
Titrate the dosage based on biochemical and/or clinical response, as
described in the full prescribing
information.
The maximum total daily dosage is 2 mg/kg orally.
Preparation and Administration Instructions ( 2.2) :
For instructions on preparing, measuring and administering the oral
suspension, see the full prescribing
information.
Maintain dietary restriction of tyrosine and phenylalanine
Take ORFADIN capsules at least one hour before, or two hours after a
meal
For patients who have difficulties swallowing capsules and who are
intolerant to the oral suspension, the
capsules may be opened and the contents suspended in a small amount of
water, formula or apple
sauce immediately before use.
Take ORFADIN oral suspension without regard to meals.
DOSAGE FORMS AND STRENGTHS
Capsules: 2 mg, 5 mg, 10 mg, 20 mg. ( 3)
Oral suspension: 4 mg/mL ( 3)
CONTRAINDICATIONS
None ( 4)
WARNINGS AND PRECAUTIONS
Elevated Plasma Tyrosine Levels, Ocular Symptoms, Developmental Delay
and Hyperkeratotic Plaques:
Inadequate restriction of