The U.S. Food and Drug Administration has approved Vyondys 53 (golodirsen) to treat patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping.

Golodirsen is an antisense oligonucleotide that can ‘skip’ over aspects of the RNA to make person’s with certain dystrophin gene mutations have a dystrophin protein that is more functional than their current mutated version.

The approval was based on a statistically significant increase in dystrophin production in skeletal muscle observed in patients treated with golodirsen. According to the developers of the drug, Sarepta Therapeutics, this accelerated approval may be contingent on a placebo-controlled, post-marketing confirmatory trial – titled ESSENCE – that is currently enrolling and expected to end by 2024.

This approval comes as somewhat of a surprise given that the FDA issued a a complete response letter (CRL) in August of this year. In a news release, Sarepta noted that “With the support of the Review Division, the matters raised in the CRL were rapidly evaluated and resolved by Dr. Peter Stein, Director of the Office of New Drugs (OND). OND granted the Company’s appeal and Sarepta re-submitted its NDA to the Review Division, which worked expeditiously to review and approve VYONDYS 53.”

Golodirsen may treat up to 8% of the DMD community. It, along with Sarepta’s other exon-skipping drug, eteplirsen means the company has medicines available for about 20% of the DMD community.

Common adverse events observed in patients taken golodirsen were headache (41%), pyrexia (41%), fall (29%), abdominal pain (27%), nasopharyngitis (27%), cough (27%), vomiting (27%), and nausea (20%).

DMD is a progressive form of muscular dystrophy that occurs primarily in males. DMD causes progressive weakness and loss (atrophy) of skeletal and heart muscles. Early signs of DMD may include delayed ability to sit, stand, or walk and difficulties learning to speak. Most children with DMD use a wheelchair full time by the age of 13 years. Heart and respiratory muscle problems begin in the teen years and lead to serious, life threatening complications. DMD is caused by changes (mutations) in the DMD gene that  codes for the protein dystrophin.

For more information about DMD, visit our learning center at checkrare.com/duchenne-muscular-dystrophy/

 

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