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Tag: Rare/Orphan Disease

How to Expedite FDA Approvals of Orphan Drugs

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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. Developing drugs for rare diseases poses a range of clinical, regulatory and commercial challenges. The small number of patients are … Continued

Rare Disease and Orphan Drug Development

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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

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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.

PBPK Modeling in Regulatory Review, Product Labeling and Safety Monitoring

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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

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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

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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

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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

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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

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