PBPK Modeling in Adults Enables DDI Risk Assessment in Children with SMA
In 2020, the US FDA approved Risdiplam (Evrysdi®) as the first orally administered drug to…
Certara2022 年 6 月 2 日
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…
Certara2022 年 6 月 1 日
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…
Certara2021 年 12 月 15 日
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…
Certara2021 年 11 月 17 日
The Scary Future of Rare Disease Management
In a world of rising healthcare costs, the greatest nightmare for payers is reimbursing drug…
Certara2021 年 9 月 23 日
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…
Certara2021 年 8 月 26 日
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…
Certara2021 年 8 月 26 日
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…
Certara2021 年 8 月 26 日
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…
Certara2021 年 7 月 29 日
