ROCTAVIAN- valoctocogene roxaparvovec-rvox injection, solution

Země: Spojené státy

Jazyk: angličtina

Zdroj: NLM (National Library of Medicine)

Aktivní složka:

VALOCTOCOGENE ROXAPARVOVEC (UNII: 681K1JDI8M) (VALOCTOCOGENE ROXAPARVOVEC - UNII:681K1JDI8M)

Dostupné s:

BioMarin Pharmaceutical Inc.

Podání:

INTRAVENOUS

Druh předpisu:

PRESCRIPTION DRUG

Terapeutické indikace:

ROCTAVIAN is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. Administration of ROCTAVIAN is contraindicated in: - patients with active infections, either acute (such as acute respiratory infections or acute hepatitis) or uncontrolled chronic (such as chronic active hepatitis B). - patients with known significant hepatic fibrosis (stage 3 or 4 on the Batts-Ludwig scale or equivalent), or cirrhosis [see Dosage and Administration (2)]. - patients with known hypersensitivity to mannitol. Risk Summary ROCTAVIAN is not intended for administration in women. There are no data on the use of ROCTAVIAN in pregnant women to inform a drug-associated risk of adverse developmental outcome. Animal reproduction and developmental toxicity studies have not been conducted with ROCTAVIAN. It is not known whether ROCTAVIAN can cause fetal harm when administered to a pregnant woman or can affect reproduction capacity. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. In the US general population, the estimated background risk of major birth defects occurs in 2 to 4% of the general population and miscarriage occurs in 15 to 20% of clinically recognized pregnancies. Risk Summary ROCTAVIAN is not intended for administration in women. There is no information regarding the presence of ROCTAVIAN in human milk, the effects on the breastfed infant, or the effects on milk production. ROCTAVIAN is not intended for administration in women. Contraception After Administration to Males In clinical studies, after administration of ROCTAVIAN, transgene DNA was detectable in semen [see Clinical Pharmacology (12.3)] . In nonclinical studies in healthy mice, the vector DNA was detected in the testes for at least 182 days post-administration of ROCTAVIAN at a dose level of 2.1 × 1014 vg/kg [see Clinical Pharmacology (12.3)]. In a mating study in immune-deficient mice, ROCTAVIAN was not detected in liver tissues of offspring of naïve females mated with dosed males [see Nonclinical Toxicology (13.1)]. For 6 months after administration of ROCTAVIAN - men of reproductive potential and their female partners must prevent or postpone pregnancy using an effective form of contraception, and - men must not donate semen. The safety and effectiveness of ROCTAVIAN in pediatric patients have not been established. A single patient ≥ 65 years of age was treated with ROCATVIAN in clinical studies. Clinical studies of ROCTAVIAN did not include sufficient numbers of patients aged 65 and over to determine whether the efficacy or safety differs compared to younger patients. In clinical studies, 3 HIV infected patients have been treated with ROCTAVIAN. Clinical studies of ROCTAVIAN did not include sufficient numbers of patients with HIV to determine whether the efficacy and safety differs compared to patients without HIV infection. A single HIV infected patient treated with ROCTAVIAN developed hepatocellular injury that subsequently resolved and was attributed to concomitant administration with antiretroviral drug efavirenz [see Drug Interactions (7.2)] . The safety and effectiveness of ROCTAVIAN in patients with prior or active factor VIII inhibitors have not been established [see Clinical Pharmacology (12.6)] . Patients with active factor VIII inhibitors should not take ROCTAVIAN. After administration of ROCTAVIAN, patients should be monitored for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests [see Warnings and Precautions (5.4)] . The safety and effectiveness of ROCTAVIAN in patients with hepatic impairment has not been established. Clinical studies excluded patients with known hepatic cirrhosis, significant fibrosis (stage 3 or 4 on the Batts-Ludwig scale or equivalent), current hepatitis B or C, or history of hepatic malignancy. No dose adjustments can be recommended for patients with hepatic impairment. The safety and effectiveness of ROCTAVIAN in patients with renal impairment has not been established. No dose adjustments can be recommended for patients with renal impairment.

Přehled produktů:

ROCTAVIAN (valoctocogene roxaparvovec-rvox) injection is supplied as a sterile, preservative-free, clear and colorless to pale yellow suspension for intravenous infusion. ROCTAVIAN contains 2 × 1013 vector genomes (vg) per mL. Each carton of ROCTAVIAN (NDC 68135-927-48) contains one single-dose vial (NDC 68135-927-01) with an extractable volume of not less than 8 mL, containing 16 × 1013 vector genomes (vg). Product as Packaged for Sale Transport frozen at ≤ -60°C (-76°F) Store upright at ≤ -60°C (-76°F). Store ROCTAVIAN vial in carton until ready to use. Protect ROCTAVIAN from light. During Preparation and Administration Thaw at room temperature, up to 25°C (77°F). After thawing, ROCTAVIAN can be held at room temperature for a maximum of 10 hours, including preparation and infusion times [see Dosage and Administration (2.2)] . If necessary, an intact vial (stopper not yet punctured) that has been thawed can be stored refrigerated (2 to 8 °C) for up to 3 days, upright and protected from light (e.g., in the original carton). Do not expose ROCTAVIAN to the light of an ultraviolet radiation disinfection lamp. Once thawed, DO NOT REFREEZE. Treat spills of ROCTAVIAN with a virucidal agent with proven activity against non-enveloped viruses and blot using absorbent materials. Dispose of unused product and disposable materials that may have come in contact with ROCTAVIAN in accordance with local guidance for pharmaceutical waste.

Stav Autorizace:

Biologic Licensing Application

Charakteristika produktu

ROCTAVIAN- VALOCTOCOGENE ROXAPARVOVEC-RVOX INJECTION, SOLUTION
BIOMARIN PHARMACEUTICAL INC.
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HIGHLIGHTS OF PRESCRIBING INFORMATION
THESE HIGHLIGHTS DO NOT INCLUDE ALL THE INFORMATION NEEDED TO USE
ROCTAVIAN SAFELY AND
EFFECTIVELY. SEE FULL PRESCRIBING INFORMATION FOR ROCTAVIAN.
ROCTAVIAN (VALOCTOCOGENE ROXAPARVOVEC-RVOX) SUSPENSION FOR INTRAVENOUS
INFUSION
INITIAL U.S. APPROVAL: 2023
INDICATIONS AND USAGE
ROCTAVIAN is an adeno-associated virus vector-based gene therapy
indicated for the treatment of adults
with severe hemophilia A (congenital factor VIII deficiency with
factor VIII activity < 1 IU/dL) without pre-
existing antibodies to adeno-associated virus serotype 5 detected by
an FDA-approved test. (1)
DOSAGE AND ADMINISTRATION
FOR ONE-TIME SINGLE-DOSE INTRAVENOUS USE ONLY. (2)
Perform baseline testing to select patients, including testing for
pre-existing antibodies to adeno-
associated virus serotype 5 (AAV5), factor VIII inhibitor presence,
and liver health assessments. (2)
The recommended dose of ROCTAVIAN is 6 × 10
vector genomes (vg) per kg of body weight. (2.1)
Start the infusion at 1 mL/min. If tolerated, the rate may be
increased every 30 minutes by 1 mL/min
up to a maximum rate of 4 mL/min. (2.1)
DOSAGE FORMS AND STRENGTHS
ROCTAVIAN is a suspension for intravenous infusion. (3)
ROCTAVIAN has a nominal concentration of 2 × 10
vg valoctocogene roxaparvovec-rvox per mL, each
vial contains an extractable volume of not less than 8 mL (16 × 10
vg). (3)
CONTRAINDICATIONS
Active infections, either acute or uncontrolled chronic. (4)
Known significant hepatic fibrosis (stage 3 or 4), or cirrhosis. (4)
Known hypersensitivity to mannitol. (4)
WARNINGS AND PRECAUTIONS
Infusion-related reactions: Infusion reactions, including
hypersensitivity reactions and anaphylaxis, have
occurred. Monitor during and for at least 3 hours after ROCTAVIAN
administration. If symptoms occur,
slow or interrupt administration and give appropriate treatment.
Restart infusion at slower rate once
symptoms resolve. Disconti