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FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy
Published on MedED: 14 April 2024
Originally published: 12 March 2024
Source: FDA Press Release
Type of article: Novel Drug & Emergent Therapy News
MedED Catalogue Reference: MNDN006
Category: Paediatrics & Neonatology
Crossreference: Rare Diseases
Category tags: Duchenne Muscular Dystrophy, DMS, givinostat, Duvyzat, genetics
Originally published: 12 March 2024
Source: FDA Press Release
Type of article: Novel Drug & Emergent Therapy News
MedED Catalogue Reference: MNDN006
Category: Paediatrics & Neonatology
Crossreference: Rare Diseases
Category tags: Duchenne Muscular Dystrophy, DMS, givinostat, Duvyzat, genetics
| Product Category | Product Name | Company | Status |
|---|---|---|---|
| Completed |
March 21, 2024/ FDA Press Release
The U.S. Food and Drug Administration (FDA) has approved Duvyzat (givinostat) as an oral medication for treating Duchenne Muscular Dystrophy (DMD) in patients aged six years and older.
DMD is a rare neurological disorder predominantly affecting males, characterized by progressive muscle weakness due to insufficient dystrophin protein. Duvyzat, the first nonsteroidal drug sanctioned for all genetic variants of DMD, functions as a histone deacetylase (HDAC) inhibitor, targeting pathological mechanisms to mitigate inflammation and muscle degeneration.
In a phase 3 study, Duvyzat's effectiveness in treating Duchenne Muscular Dystrophy (DMD) was assessed through a randomized, double-blind, placebo-controlled trial spanning 18 months.
The primary outcome measure was the change in muscle function, evaluated by the time taken to climb four stairs from baseline to month 18.
- Participants continued a standard steroid regimen during the study, and those treated with Duvyzat exhibited a statistically significant reduction in the time taken to climb stairs compared to the placebo group, with a mean change of 1.25 seconds versus 3.03 seconds respectively.
- Additionally, as a secondary endpoint, the North Star Ambulatory Assessment (NSAA) was used to assess physical function, showing less deterioration in NSAA scores over the 18-month period in patients treated with Duvyzat compared to those on placebo.
Despite its efficacy, Duvyzat is associated with common side effects like diarrhoea, abdominal pain, and decreased platelets. There are also warnings regarding platelet counts, triglyceride levels, and QTc prolongation.
Duvyzat's approval was granted priority review and fast-track designation, and the drug received orphan drug and rare pediatric disease designations.
Clinical Trials & Supporting Information
| Study Phase | ClinicalTrial.gov Registration | Clinical Trial Study Name |
|---|---|---|
| Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy |
Access Original Press Release
21 March 2024.FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy
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This article is compiled from a variety of resources. Every effort has been made to correctly attribute quotes and content. Where possible all information has been independently verified. The Medical Education Network bears no responsibility for any inaccuracies which may occur from the use of third-party sources. If you have any queries regarding this article contact us
Fact-checking Policy
The Medical Education Network makes every effort to review and fact-check the articles used as source material in our summaries and original material. We have strict guidelines in relation to the publications we use as our source data, favouring peer-reviewed research wherever possible. Every effort is made to ensure that the information contained here is an accurate reflection of the original material. Should you find inaccuracies, out of date content or have any additional issues with our articles, please make use of the contact us form to notify us.
