Givinostat is a synthetic histone deacetylase (HDAC) inhibitor belonging to the hydroxamic acid class. It is an orally active small-molecule pharmaceutical ingredient that inhibits HDAC enzymes involved in the regulation of gene expression, chromatin structure and cellular signaling.
Givinostat is used in the treatment of Duchenne muscular dystrophy (DMD) and is marketed in the United States under the brand name Duvyzat.
Givinostat is a specialized small-molecule pharmaceutical API used in the development and manufacture of oral solid dosage formulations. Its synthesis involves multiple chemical steps followed by purification and comprehensive analytical characterization.
CAS Number: 497833-03-9
Molecular Formula: C24H27N3O4
Molecular Weight: 421.49 g/mol
Development Code: ITF2357
Drug Class: Histone Deacetylase Inhibitor
Pharmacological Class: HDAC Inhibitor
Therapeutic Class: Antineoplastic / Musculoskeletal Therapy
Therapeutic Area: Neuromuscular Disorders
Givinostat inhibits histone deacetylases (HDACs), enzymes that remove acetyl groups from histone and non-histone proteins. By inhibiting HDAC activity, givinostat can alter gene transcription and cellular signaling.
In Duchenne muscular dystrophy, HDAC inhibition is associated with modulation of inflammatory and fibrotic pathways, which may contribute to preservation of muscle function.
Givinostat is indicated for the treatment of Duchenne muscular dystrophy in patients 6 years of age and older in the United States.
It is administered orally and is available as an oral suspension formulation.
Duchenne muscular dystrophy is a progressive genetic neuromuscular disorder characterized by muscle degeneration and weakness. Givinostat provides a pharmacological approach based on HDAC inhibition and has been developed to address disease-associated inflammatory and fibrotic processes.
Givinostat is a synthetic small molecule containing a hydroxamic acid functional group and a substituted aromatic system. It has the molecular formula C24H27N3O4 and a molecular weight of 421.49 g/mol.
Givinostat API quality evaluation may include:
Givinostat should be stored in a tightly closed container under controlled pharmaceutical conditions and protected from excessive heat, moisture and direct light. Exact storage conditions should follow validated stability data and the applicable product-specific specification.
Givinostat, also known as ITF2357, has been investigated as a broad-spectrum HDAC inhibitor in inflammatory, neuromuscular and other disease areas. Its clinical development has particularly focused on Duchenne muscular dystrophy.
Givinostat is an orally active histone deacetylase inhibitor with CAS Number 497833-03-9, molecular formula C24H27N3O4, and molecular weight 421.49 g/mol. It is primarily associated with the treatment of Duchenne muscular dystrophy and is marketed as Duvyzat in the United States.
| Parameter | Specification |
|---|---|
| Appearance | White to off-white crystalline powder |
| Identification | IR & HPLC compliant |
| Assay (HPLC) | 98.0% – 102.0% |
| Loss on Drying | NMT 1.0% |
| Heavy Metals | NMT 20 ppm |
| Individual Impurity | NMT 0.5% |
| Total Impurities | NMT 2.0% |
| Water Content | NMT 2.0% |
| Melting Point | Approximately 215–220°C |