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FDA Authorizes First Test To Aid In Newborn Screening For Duchenne Muscular Dystrophy
Clinical Trials and Research

FDA Authorizes First Test to aid in Newborn Screening for Duchenne Muscular Dystrophy

Today, the U.S. Food and Drug Administration authorized marketing of the first test to aid in newborn screening for Duchenne Muscular Dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness.

“Diagnostics that can safely and effectively screen newborns can help health care professionals identify and discuss potential treatment options with parents and caregivers before symptoms or effects on a baby’s health may be noticeable,” said Tim Stenzel, M.D., Ph.D., director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health. “This authorization reflects our commitment to fostering innovation in devices to help inform and provide options to patients and their caregivers. Early screening can help identify individuals who need additional follow up or treatment.”

Dec 12, 2019
Sarepta Therapeutics Announces FDA Approval Of VYONDYS 53™ (golodirsen) Injection For The Treatment Of Duchenne Muscular Dystrophy (DMD) In Patients Amenable To Skipping Exon 53
Clinical Trials and Research

Sarepta Therapeutics Announces FDA Approval of VYONDYS 53™ (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved VYONDYS 53™ (golodirsen). VYONDYS 53 is an antisense oligonucleotide from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping.

This indication is based on a statistically significant increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53, which is reasonably likely to predict clinical benefit for those patients who are exon 53 amenable. Consistent with the accelerated approval pathway, the continued approval of VYONDYS 53 may be contingent on confirmation of a clinical benefit in this post-marketing confirmatory trial.

Sarepta’s placebo-controlled, post-marketing confirmatory trial to support the VYONDYS 53 accelerated approval – titled ESSENCE – is currently enrolling and expected to conclude by 2024.

Dec 12, 2019