PBPK, a Regulatory Necessity, Delivers ROI through Reduced R&D Cost and Time
全世界约有 400,000 名先天性肾上腺增生症（CAH）患者。目前治疗 CAH 的方法是使用各种普通糖皮质激素类固醇（在美国包括 hydrocortisone, dexamethasone, prednisolone 和 prednisone），没有标准的治疗方案。The cortisol deficiency and over-production of male sex hormones caused by CAH can lead to increased mortality, infertility, and sexual development issues. Sufferers, even if treated, remain at risk of death from an adrenal crisis.
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.
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.
Genentech was developing polatuzumab vedotin (Polivy), an anti-CD79b-vc-monomethyl auristatin E (MMAE) antibody-drug conjugate (ADC) to treat patients with transplantation-ineligible relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL). MMAE is a potent anti-cancer drug that is a CYP3A substrate and a weak competitive CYP3A inhibitor.
In October 2019, Galderma announced that the U.S. FDA approved AKLIEF® (trifarotene) Cream, 0.005% for the topical treatment of acne. It is the only topical retinoid that selectively targets retinoic acid receptor gamma and is also the first new retinoid molecule to receive FDA approval for the treatment of acne in more than 20 years.
