How to Navigate the FDA Landscape for an Orphan Drug
Rare diseases are a public health priority. FDA has launched several programs and a final guidance to address the unmet need for orphan drugs.
Tag: Rare disease
Simcyp Simulator Helps Optimize Drug Dosing in Adults & Adolescents with Orphan Disease
Congenital adrenal hyperplasia (CAH) affects about 400,000 patients worldwide. Current therapy for CAH uses a variety of generic glucocorticoid steroids (including hydrocortisone, dexamethasone, prednisolone, and prednisone in the US) with no standard treatment regimen. The cortisol deficiency and over-production of male sex hormones caused by CAH can lead to increased mortality, infertility, and sexual development … Continued
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 treat SMA in patients ≥2 months old, followed by the European Medicine Agency. Risdiplam addresses the underlying cause of SMA: a reduced amount of survival motor neuron (SMN) protein.
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
Everolimus (Afinitor®) was recently approved as adjunctive therapy for tuberous sclerosis complex (TSC)-associated partial seizures in ages 2 and older. Unlike many other therapies for treating TSC-associated seizures, everolimus addresses the underlying pathophysiology of TSC. As TSC-associated seizures can also affect children aged between 6 months and 2 years, a modeling and simulation approach that … Continued
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®)
Pharmacylics and J&J sought to bring ibrutinib, its new tyrosine kinase inhibitor therapy targeting rare B-cell malignancies to market. The company leveraged FDA’s accelerated approval program; it was one of the first to be awarded breakthrough status by the agency. Ibrutinib is susceptible to interactions with a strong inhibitor and inducer of CYP3A4 enzymes, thus … Continued
Voxelotor for Treating Sickle Cell Disease: Leveraging Dose Prediction Model for DDI Prediction Without Any Clinical Studies
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