Category: Case Study
Using Real Word Evidence to Enhance Drug Development
Agent-Based Model Predicts Impact of Monoclonal Antibody Treatment, Prophylaxis & Vaccines on Viral Transmission during Pandemic
Using Modeling & Simulation to Develop a Dosing Regimen for a Bispecific Antibody for Pediatric Cancer
Using PBPK Models to Predict Effects of Hepatic Impairment & DDI in Olanzapine/Samidorphan Combination Therapy
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
Providing Around-the-Clock Regulatory Support While Enhancing Team Chemistry
Advaxis Inc. is devoted to the discovery, development, and commercialization of immunotherapies based on a technology platform which uses engineered Listeria monocytogenes. The company is headquartered in Monmouth Junction, New Jersey.
Biosimulation Supports Label Claims for a Combination Oncology Treatment
Genentech’s Zelboraf® (vemurafenib) is a small molecule B-RAF inhibitor and is FDA-approved to treat patients with metastatic or unresectable melanoma whose tumors express the B-RAF V600E mutation. To reduce the likelihood of cancer cells becoming drug resistant, Genentech wanted to combine vemurafenib with cobimetinib, a small molecule MEK inhibitor that targeted a different part of … 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.
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 formation in small blood vessels throughout the body leading to kidney failure, other organ damage, and premature death. At the start of this project, there were no FDA-approved aHUS treatments. Furthermore, as only a few thousand aHUS patients are diagnosed … Continued