EVRYSDI Oral solution (2020)
Βιβλιογραφική αναφορά
Συγγραφείς
Genentech Inc.
Λέξεις κλειδιά
50242-175
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1. Indications and Usage
EVRYSDI is indicated for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older.
2. Dosage and Administration
2.1 Important Administration Instructions Dose Preparation It is recommended that a healthcare provider discuss with the patient or caregiver how to prepare the prescribed daily dose prior to administration ...
3. Dosage Forms and Strengths
EVRYSDI for oral solution: 60 mg as a light yellow, yellow, greyish yellow, greenish yellow, or light green powder for constitution. Following constitution, the volume of the greenish yellow to yellow ...
4. Contraindications
None.
6.1. Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in clinical trials of another ...
7. Drug Interactions
7.1 Effect of EVRYSDI on Substrates of Multidrug and Toxin Extrusion (MATE) Protein Transporters Based on in vitro data, EVRYSDI may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K ...
8.1. Pregnancy
Risk Summary There are no adequate data on the developmental risk associated with the use of EVRYSDI in pregnant women. In animal studies, administration of risdiplam during pregnancy or throughout pregnancy ...
8.2. Lactation
Risk Summary There are no data on the presence of risdiplam in human milk, the effects on the breastfed infant, or the effects on milk production. Risdiplam was excreted in the milk of lactating rats orally ...
8.3. Females and Males of Reproductive Potential
Studies of risdiplam in juvenile and adult rats and in monkeys demonstrated adverse effects on the reproductive organs, including germ cells, in males at clinically-relevant plasma exposures <em>[see Use ...
8.4. Pediatric Use
The safety and effectiveness of EVRYSDI in pediatric patients 2 months of age and older have been established <em>[see Clinical Studies (14)]</em>. Safety and effectiveness in pediatric patients below ...
8.5. Geriatric Use
Clinical studies of EVRYSDI did not include patients aged 65 years and over to determine whether they respond differently from younger patients.
8.7. Hepatic Impairment
The safety and efficacy of EVRYSDI in patients with hepatic impairment have not been studied. Because risdiplam is predominantly metabolized in the liver, hepatic impairment may potentially increase the ...
11. Description
EVRYSDI for oral solution contains risdiplam, which is a survival of motor neuron 2 (SMN2)-directed RNA splicing modifier. The chemical name of risdiplam is 7-(4,7-diazaspiro[2.5]octan-7-yl)2(2,8 dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido-4H-[1,2-a]pyrimidin-4-one. ...
12.1. Mechanism of Action
Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat patients with spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. ...
12.2. Pharmacodynamics
In clinical trials, EVRYSDI led to an increase in SMN protein with a greater than 2-fold median change from baseline within 4 weeks of treatment initiation. The increase was sustained throughout the treatment ...
12.3. Pharmacokinetics
Pharmacokinetics of EVRYSDI have been characterized in healthy adult subjects and in patients with SMA. After administration of EVRYSDI as an oral solution, pharmacokinetics of risdiplam were approximately ...
13.1. Carcinogenesis, Mutagenesis, Impairment of Fertility
Carcinogenesis The carcinogenic potential of risdiplam has not been fully evaluated. Risdiplam was not carcinogenic in Tg.rasH2 mice when administered at oral doses of up to 9 mg/kg/day for 26 weeks. ...
13.2. Animal Toxicology and/or Pharmacology
Retinal toxicity Risdiplam-induced functional and structural retinal abnormalities were seen in animal studies. In a 39-week toxicity study in monkeys, oral administration of risdiplam (0, 1.5, 3, or 7.5/5 ...
14. Clinical Studies
The efficacy of EVRYSDI for the treatment of patients with infantile-onset and later-onset SMA was evaluated in two clinical studies, Study 1 (NCT02913482) and Study 2 (NCT02908685). The overall findings ...
16.1. How Supplied
Each amber glass bottle of EVRYSDI is packaged with a bottle adapter, two 6 mL reusable oral syringes, and two 12 mL reusable oral syringes. EVRYSDI for oral solution is a light yellow, yellow, greyish ...
16.2. Storage and Handling
Store the dry powder at 20°C to 25°C (68°F to 77°F), excursions permitted between 15°C to 30°C (59°F to 86°F) [see USP controlled room temperature]. Keep in the original carton. Keep the constituted oral ...
17. Patient Counseling Information
Advise the patient to read the FDA-approved patient labeling (Patient Information and Instructions for Use). Pregnancy and Fetal Risk Inform pregnant women and women of reproductive potential that, based ...