Rare and Neglected Diseases Archives

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…

Becca BucciJune 1, 2022

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

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…

Jim GallagherNovember 17, 2021

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…

Jim GallagherSeptember 23, 2021

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…

Jim GallagherAugust 26, 2021

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…

Jim GallagherAugust 26, 2021

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…

Jim GallagherAugust 26, 2021

Case Study

Using PBPK for Label Recommendations in Rare Disease: Deflazacort for Duchenne Muscular Dystrophy

Jim GallagherAugust 20, 2021

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…

Jim GallagherJuly 29, 2021

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…

Jim GallagherJuly 14, 2021

Rare and Neglected Diseases

Leveraging Clinical Data from One Rare Disease to Support Drug Approval for Another

A Bird’s Eye View on Rare Diseases: Key Drug Development Considerations for Thyroid Eye Disease

Dose Optimization Using Population PK for an Orphan Drug

The Scary Future of Rare Disease Management

Using a PBPK-PD Receptor Occupancy Model to Determine the Optimal Dose of Radiprodil for Neonates

Simcyp’s Age-Defining Feature Facilitates Everolimus Dosing in Neonates

Eliglustat for Gaucher Disease: Quantifying the Impact of Pharmacogenetic Status on DDIs

Using PBPK for Label Recommendations in Rare Disease: Deflazacort for Duchenne Muscular Dystrophy

DDI Regulatory Poster Child using the Simcyp Simulator: Ibrutinib (Imbruvica®)

Voxelotor for Treating Sickle Cell Disease: Leveraging Dose Prediction Model for DDI Prediction Without Any Clinical Studies

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