Drug intelligence / Profile preview

all-trans retinoic acid + danazol

Development stage
Phase 2
Lead developer
Peking University People's Hospital
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules
Administration
Oral
01

Overview

All-trans retinoic acid + danazol is a combination therapy of two small molecule drugs used primarily as a second-line treatment for adults with corticosteroid-resistant or relapsed primary immune thrombocytopenia (ITP). All-trans retinoic acid (ATRA, also known as tretinoin) is a derivative of vitamin A that acts as an immunomodulator and promotes hematopoietic cell differentiation. Danazol is a synthetic steroid with gonadotropin-inhibiting properties that modulates the immune response and increases platelet counts by suppressing pituitary gonadotropins and affecting steroid hormone receptors. Clinical studies have shown that this combination leads to more rapid and sustained platelet responses compared to danazol monotherapy in ITP patients who are non-splenectomized and resistant or relapsed after corticosteroids. The regimen has demonstrated safety and efficacy in phase 2 clinical trials[1][2][4][7].

Other names
ATRA + danazoltretinoin + danazol
02

Targets

AR (Adrenergic receptors)GR (Glucocorticoid receptor)FSH (Follicle-stimulating hormone)PR (Progesterone receptor)RARA (Retinoic acid receptor alpha)RARB (Retinoic acid receptor beta)RARG (Retinoic acid receptor gamma)

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