Leveraging Clinical Data from One Rare Disease to Support Drug Approval for Another Case Study Leveraging Clinical Data from One Rare Disease to Support Drug Approval for Another Atypical hemolytic uremic syndrome (aHUS) is an ultra-rare genetic disease that causes abnormal blood clot…CertaraJune 1, 2022
A Bird’s Eye View on Rare Diseases: Key Drug Development Considerations for Thyroid Eye Disease Blog A Bird’s Eye View on Rare Diseases: Key Drug Development Considerations for Thyroid Eye Disease New therapeutics discovery and development for ocular diseases have been traditionally associated with a low…CertaraDecember 15, 2021
Dose Optimization Using Population PK for an Orphan Drug Case Study Dose Optimization Using Population PK for an Orphan Drug RAPALIMUS® Tablets (sirolimus) is Nobelpharma’s orphan drug developed from an oral medication that is sold…CertaraNovember 17, 2021
The Scary Future of Rare Disease Management On-Demand Webinar The Scary Future of Rare Disease Management In a world of rising healthcare costs, the greatest nightmare for payers is reimbursing drug…CertaraSeptember 23, 2021
Using a PBPK-PD Receptor Occupancy Model to Determine the Optimal Dose of Radiprodil for Neonates Case Study Using a PBPK-PD Receptor Occupancy Model to Determine the Optimal Dose of Radiprodil for Neonates Radiprodil is a selective allosteric modulator of the NR2B N-methyl-D-aspartate receptor GluN2B-NMDA that was being…CertaraAugust 26, 2021
Simcyp’s Age-Defining Feature Facilitates Everolimus Dosing in Neonates Case Study Simcyp’s Age-Defining Feature Facilitates Everolimus Dosing in Neonates Everolimus (Afinitor®) was recently approved as adjunctive therapy for tuberous sclerosis complex (TSC)-associated partial seizures…CertaraAugust 26, 2021
Eliglustat for Gaucher Disease: Quantifying the Impact of Pharmacogenetic Status on DDIs Case Study Eliglustat for Gaucher Disease: Quantifying the Impact of Pharmacogenetic Status on DDIs In 2014, Eliglustat (Cerdelga®) was approved by the FDA as the first long-term treatment for…CertaraAugust 26, 2021
Using PBPK for Label Recommendations in Rare Disease: Deflazacort for Duchenne Muscular Dystrophy Case Study Using PBPK for Label Recommendations in Rare Disease: Deflazacort for Duchenne Muscular Dystrophy CertaraAugust 20, 2021
DDI Regulatory Poster Child using the Simcyp Simulator: Ibrutinib (Imbruvica®) Case Study DDI Regulatory Poster Child using the Simcyp Simulator: Ibrutinib (Imbruvica®) Pharmacylics and J&J sought to bring ibrutinib, its new tyrosine kinase inhibitor therapy targeting rare…CertaraJuly 29, 2021
Voxelotor for Treating Sickle Cell Disease: Leveraging Dose Prediction Model for DDI Prediction Without Any Clinical Studies Case Study Voxelotor for Treating Sickle Cell Disease: Leveraging Dose Prediction Model for DDI Prediction Without Any Clinical Studies In November 2019, the US FDA granted accelerated approval for Oxbryta™ (voxelotor) tablets for the…CertaraJuly 14, 2021