Tag: 希少疾患
FDAによる希少疾病用医薬品の承認を迅速化するには
by Thomas Peyret 350 million patients worldwide suffer from 7,000 rare diseases, yet only 300 of these diseases have approved treatments. This gap, impacting 95% of rare disease patients, represents a huge unmet medical need. 希少疾患治療薬（オーファンドラッグ）の開発には、臨床・規制・商業面において多様な課題を伴います。The small number of patients are … Continued
希少疾病と希少疾病用医薬品の開発
There are 7,000 rare diseases impacting 350 million patients worldwide, yet only 300 of these diseases have approved treatments. This gap, impacting 95% of those suffering from rare diseases, represents a huge unmet medical need. Modeling and simulation approaches are not only ideal for the development of drugs for rare diseases, but are encouraged by regulators.
Informing Pediatric Drug Development: A Selection from Certara’s Best of Blogs
A selection of short essays by Certara’s pediatric drug development experts. Learn about our technologies and strategies for pediatric drug development to inform dose selection, including PK/PD simulations using sparse data analysis and our Simcyp Pediatric Simulator. Certara’s regulatory writing consultancy, Synchrogenix, also offer regulatory strategy for pediatrics.
Physiologically-based Pharmacokinetic Modeling in Pediatric Oncology Drug Development
PBPK Modeling in Regulatory Review, Product Labeling and Safety Monitoring
Physiologically-based pharmacokinetic (PBPK) modeling can address various questions raised in drug development and regulatory review, and is used most extensively to predict and quantify the extent of drug-drug interactions (DDIs) from both in vitro and clinical data. This assists with dose selection and the design of clinical studies as well as informing decisions relating to … Continued
Understanding Complex PK
To solve a mystery in clinical development for a promising new formulation to combat a rare disease, Certara performed physiologically relevant modeling.
Population Pharmacokinetic and Pharmacodynamic Analyses from a 4-Month Intra-dose Escalation and Its Subsequent 12-month Dose Titration Studies for a Human Monoclonal Anti-FGF23 Antibody (KRN23) in Adults with X-linked Hypophosphatemia
X-linked hypophosphatemia (XLH) is an inherited metabolic bone disease with abnormally elevated serum FGF23 resulting in low renal maximum threshold for phosphate reabsorption, low serum phosphate (Pi) and 1,25-dihydroxyvitamin D levels with subsequent development of short stature and skeletal deformities. KRN23 is a novel human anti-FGF23 antibody for the treatment of XLH. The pharmacokinetics (PK) … Continued
Precision Dosing Using PBPK Modeling
Precision dosing― the right dose, for the right patient, at the right time― is crucial to providing patients with the most efficacious medications with minimal probability of adverse events. One key step towards achieving the delivery of individualized dosing is testing potential dosing regimens in a patient’s ‘virtual twin.’ The other key step is to … Continued
Understanding the Relationship Between Systemic and Hepatic Exposure of Obeticholic Acid for the Treatment of Liver Disease in Patients with Cirrhosis
Obeticholic acid (OCA) is a selective and potent farnesoid X receptor (FXR) agonist in development for several chronic liver diseases. OCA is a semi-synthetic analogue of chenodeoxycholic acid (CDCA) with similar pharmacokinetic (PK) properties. There was a significant increase in systemic exposure of OCA in patients with hepatic impairment. A proportionally similar increase in systemic … Continued
