Orphan drugs affect 350,000 people worldwide, including 10% of the US population and 1 in 25 Europeans. Model-informed drug development (MIDD) approaches, such as PBPK and PopPK have been embraced by sponsors and regulators, and play a key role in modernizing and accelerating orphan drug development.
Tag: Rare/Orphan Disease
PRINCETON, NJ – Dec. 20, 2017 – Certara today congratulated Medicines Development for Global Health (MDGH), a not-for-profit global health company, on the submission of its new drug application (NDA) for moxidectin as an oral treatment for river blindness (onchocerciasis).
Certara’s Phoenix Technology Services team helped Arvinas integrate D360 Express with McNeil’s Assay Capture and Analysis System to facilitate structure-activity relationship analysis and save time.
Watch this webinar with Jeffrey Edwards to learn how he used physiologic pharmacokinetic modeling to understand the relationship between systemic and hepatic exposure of OCA in patients with and without hepatic impairment. By watching this webinar, you will learn how pharmacokinetic modeling can support optimal dosing for patients with organ impairment and facilitate regulatory approval.
A selection of short essays from our blog containing insights from our thought leaders on how modeling and simulation has impacted and is continuing to reshape our approach to drug development.
A selection of short essays from our blog, written to empower our customers with modeling and simulation (M&S) and regulatory writing solutions in order to help them solve the toughest drug development problems. Certara staff contributions range in topic from pharmacometrics to systems biology to the growing importance of regulatory writing and sharing clinical trial results.
Certara scientists deliver an integrated solution that helps Intercept Pharmaceuticals achieve FDA approval for Ocaliva.
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.