Saturday, April 20, 2024

Breakthrough in Duchenne Muscular Dystrophy Treatment: FDA Greenlights Duvyzat, First of Its Kind

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The Food and Drug Administration (FDA) has authorized Duvyzat, the first nonsteroidal medication to treat patients with all genetic variants of Duchenne Muscular Dystrophy (DMD), a rare neurological disorder. Duvyzat, a histone deacetylase (HDAC) inhibitor, works by targeting pathogenic processes to reduce inflammation and muscle loss.

This approval is based on the results of the EPIDYS clinical trial, a multicentre, randomized, double-blind, and placebo-controlled Phase III trial involving 179 ambulant boys. The trial investigated Duvyzat, which was administered twice daily, in addition to glucocorticosteroid treatment in both study arms.

The trial successfully met the primary endpoint. Subjects in the Duvyzat arm had a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment, compared to those given a placebo. The mean change was 1.25 seconds for Duvyzat patients versus 3.03 seconds for placebo. A secondary endpoint, the North Star Ambulatory Assessment (NSAA), also recorded less deterioration in physical function in Duvyzat-treated patients after 18 months.

Duchenne Muscular Dystrophy

A Milestone for Duchenne Muscular Dystrophy Treatment and Patient Care

Common side effects associated with Duvyzat include diarrhea, abdominal pain, reduced platelets leading to increased bleeding, and nausea/vomiting. The FDA granted Duvyzat priority review and fast-track designation. It also received orphan drugs and rare pediatric disease designations.

Paolo Bettica, chief medical officer at Italfarmaco Group, stated that the FDA approval of Duvyzat, supported by a robust and successful clinical development program, exemplifies Italfarmaco’s commitment to providing safe and proven-effective therapy for people living with Duchenne Muscular Dystrophy. The focus now is to make Duvyzat available for Duchenne Muscular Dystrophy management in the US as quickly as possible.

The authorization of Duvyzat signifies a significant advance in the treatment of Duchenne Muscular Dystrophy. This medication, which effectively slows the progression of physical symptoms, offers a new therapeutic option for patients suffering from all genetic variants of this rare and debilitating neurological disorder.


Resource: Pharmaceutical Business Review, March 22, 2024

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