Home Medizin FDA genehmigt Givinostat für Duchenne-Muskeldystrophie

FDA genehmigt Givinostat für Duchenne-Muskeldystrophie

von NFI Redaktion

The US Food and Drug Administration (FDA) has approved Givinostat (Duvyzat) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 6 years and older.

Givinostat, a histone deacetylase inhibitor, is the first non-steroidal medication approved for treating patients with all genetic variants of DMD. It works by targeting pathogenic processes to reduce inflammation and muscle wasting, as explained in an FDA press release announcing the approval of the drug.

Givinostat is administered twice daily with a meal. The recommended dosage is based on the patient’s body weight.

„DMD denies affected children the chance of a healthy life. This approval provides another treatment option to reduce the burden of this progressive, devastating disease for individuals affected by DMD, regardless of genetic mutation,“ stated Dr. Emily Freilich in a press release from the Office of Neuroscience at the Center for Drug Evaluation and Research of the FDA.

The approval was based on the results of the randomized, double-blind, placebo-controlled Phase 3 study EPIDYS. Givinostat demonstrated statistically and clinically significant benefits compared to placebo in ambulatory children with DMD, including reduced muscle function and strength decline when added to standard corticosteroid therapy.

Although the results of the four-step rise assessment from baseline to week 72, the study’s primary endpoint, deteriorated in both the treatment and placebo groups, the decline in Givinostat was significantly lower, as reported by Medical News from Medscape.

Potential for Combination Therapy

„There is an immense unmet need for novel DMD therapies that can provide significant benefits for a wide range of patients,“ said study researcher Craig M. McDonald, MD, from UC Davis Health, in a company press release. „Duvyzat’s unique mechanism of action has shown a positive risk-benefit profile and the ability to delay disease progression, highlighting its potential to become a key component of standard care for people with DMD.“

The approval of Givinostat „provides another important treatment option“ for people with DMD, said Sharon Hesterlee, PhD, Chief Research Officer of the Muscular Dystrophy Association (MDA), in a separate statement.

Givinostat’s different mode of action from traditional DMD treatments suggests that „combining this new drug with other therapies could further improve the lives of our DMD patients,“ added Dr. Barry Byrne, Chief Medical Officer of MDA.

The most common side effects of Givinostat include diarrhea, abdominal pain, decreased platelet count potentially leading to increased bleeding, nausea/vomiting, elevation of triglycerides, and fever.

Prior to prescribing Givinostat, doctors are advised to assess a patient’s platelet count and triglycerides. Patients with a platelet count < 50 × 10^9 cells/L should not take the medication. Platelet count and triglyceride levels should be monitored as recommended during treatment to determine if dosage adjustments are necessary. Dosage adjustments may also be required for moderate to severe diarrhea.

Givinostat can cause QTc prolongation. Patients taking certain medications that also cause QTc prolongation and patients with certain types of heart disease should avoid taking Givinostat.

The FDA accepted the company’s New Drug Application and granted Givinostat priority review in June 2023. Italfarmaco announced in November that the Prescription Drug User Fee Act originally scheduled for December 21 was extended to March 21 to give the agency more time to review data submitted by the company.

Complete prescribing information and a medication guide are available online.

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