FDA OKs First 'Targeted Treatment' For Rare DMD Mutation
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FDA has approved the antisense oligonucleotide casimersen injection for the treatment of patients with Duchenne muscular dystrophy (DMD) plus a rare DMD mutation. This particular mutation of the DMD gene is amenable to exon 45 skipping. It added that this is its first approval of a targeted treatment for patients with the mutation.

Approved — With Cautions

The FDA approved viltolarsen for the treatment of DMD in patients with a confirmed mutation amenable to exon 53 skipping, following approval of golodirsen injection for the same indication in December 2019. The DMD gene mutation that is amenable to exon 45 skipping is present in about 8% of patients with DMD.

The trial that carried weight with the FDA included 43 male participants with DMD aged 7-20 years and. All were confirmed to have the exon 45-skipping gene mutation and all were randomly assigned 2:1 to received IV casimersen 30 mg/kg or matching placebo. Results showed that, between baseline and 48 weeks posttreatment, the casimersen group showed a significantly higher increase in levels of dystrophin protein than the placebo group.

Upper respiratory tract infections, fever, joint and throat pain, headache, and cough were the most common adverse events experienced by the active-treatment group. Although the clinical studies assessing casimersen did not show any reports of kidney toxicity, the adverse event was observed in some nonclinical studies.

Overall, the FDA has concluded that the data submitted by the applicant demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in this patient population. However, it noted that definitive clinical benefits such as improved motor function were not established.

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