PBPK, a Regulatory Necessity, Delivers ROI through Reduced R&D Cost and Time
In the heavily regulated pharmaceutical industry, changes to the manufacturing process can be quite complex and costly.
Pharmacylics and J&J sought to bring ibrutinib, its new tyrosine kinase inhibitor therapy targeting rare B-cell malignancies to market.
Many cystic fibrosis (CF) patients take several medications at one time to manage symptoms and the disease.
In November 2019, the US FDA granted accelerated approval for Oxbryta™ (voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older.
Guanfacine (Intuniv® XR) extended release (GXR) is an orally administered, selective alpha2A-adrenergic receptor agonist, non-stimulant treatment for children and adolescents with attention deficit/hyperactivity disorder (ADHD).
Radiprodil is a selective allosteric modulator of the NR2B N-methyl-D-aspartate receptor GluN2B-NMDA that was being investigated to treat infantile spasm syndrome.
Quetiapine is an atypical antipsychotic for treating schizophrenia, bipolar depression, bipolar mania that helps to restore the balance of certain natural substances (neurotransmitters) in the brain.
Everolimus (Afinitor®) was recently approved as adjunctive therapy for tuberous sclerosis complex (TSC)-associated partial seizures in ages 2 and older.
In 2014, Eliglustat (Cerdelga®) was approved by the FDA as the first long-term treatment for adults with type 1 Gaucher disease.
Deflazacort (Emflaza®) was fast-tracked, given orphan status, and approved by the US FDA in February 2017 for patients 5 years and older with Duchenne Muscular Dystrophy (DMD).
Aripiprazole lauroxil (Aristada®) was approved by the FDA for treating schizophrenia in adults in October 2015 at monthly and 6-week dosing options.
Cobimetinib (Cotellic®), approved by the US FDA in 2015, is a kinase inhibitor for treating advanced melanoma.