Givinostat: First Approval

<div><strong>Declarations</strong></div> <div><strong>Funding</strong> The preparation of this review was not supported by any external funding.</div> <div><strong>Authorship and Conflict of interest</strong> During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Yvette N. Lamb is a contracted employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.</div> <div><strong>Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability</strong> not applicable</div> <p>Additional information about this Adis Drug Review can be found<a href="https://www.springer.com/gp/adis/products-services/adis-journals-newsletters/adis-drug-reviews" target="_blank"> here.</a></p> <div><strong>Abstract</strong> </div> <p>Givinostat (DUVYZAT™), an orally available histone deacetylase inhibitor, is being developed by Italfarmaco for the treatment of muscular dystrophy and polycythemia vera. Givinostat received its first approval on 21 March 2024, in the USA, for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. Approval was based on the results of the multinational phase III EPIDYS trial, in which givinostat recipients showed less decline than placebo recipients in the time taken to perform a functional task. Givinostat represents the first nonsteroidal treatment for DMD to be approved for use in patients irrespective of the specific genetic variant underlying their disease. Givinostat is available as an oral suspension to be administered twice daily with food. The recommended dosage is based on the body weight of the patient. In the EU, regulatory review of givinostat in DMD is currently underway. This article summarizes the milestones in the development of givinostat leading to this first approval for DMD.</p> <p>© Springer Nature Switzerland AG 2024</p>