This week, Neurology News Network covered the FDA acceptance of Sarepta Therapeutics' NDA for SRP-4053 for treatment of patients who have Duchenne muscular dystrophy with mutations subject to exon 53 skipping and the Magnetic Resonance Parkinsonism Index 2.0 that has accurately differentiated Parkinson disease from a progressive supranuclear palsy-parkinsonism phenotype. Additionally, the network discussed the statement issued by the FDA regarding young donor plasma infusions being offered as treatment for conditions for which benefits are unproven. (Transcript below)

Jenna:
Welcome to Neurology News Network. I’m Jenna Payesko. Let’s get into the news from this week.

The FDA has accepted Sarepta Therapeutics’ NDA seeking the accelerated approval of SRP-4053, also known as golodirsen, for the treatment of patients who have Duchenne muscular dystrophy with genetic mutations subject to exon 53 skipping.

The NDA is backed by data from a phase 1/2 study that assessed the safety, tolerability, pharmacokinetics, and dystrophin expression of SRP-4053. Sarepta completed the NDA at the end of 2018 as part of a rolling submission and requested priority review, which the FDA granted. The therapy currently has a PDUFA action date of Aug. 19, 2019.

A precise measurement done by MRI, called the Magnetic Resonance Parkinsonism Index 2.0, has been shown to accurately predict the clinical evolution toward a progressive supranuclear palsy-parkinsonism phenotype, differentiating it from Parkinson disease.

Ultimately, after the final follow-up, 100 patients out of the total cohort of 110 patients with Parkinson maintained their original Parkinson diagnosis, while 10 developed vertical gaze abnormalities. The MRPI 2.0 classified all 10 of these patients as probable PSP-parkinsonism with 100% sensitivity, specificity, positive and negative predictive values, and accuracy, with a cutoff value of ≥2.88.

The FDA has issued a joint statement from its commissioner, Dr. Scott Gottlieb, and Dr. Peter Marks, the director of the agency’s Center for Biologics Evaluation and Research, regarding young donor plasma infusions being offered at a variety of establishments as a treatment for conditions for which its benefits are unproven.

The conditions in question range from standard aging and memory loss issues, to conditions such as dementia, Parkinson disease, multiple sclerosis, Alzheimer disease, heart disease, and post-traumatic stress disorder.

“We have significant public health concerns about the promotion and use of plasma for these purposes,” the pair said. “There is no proven clinical benefit of infusion of plasma from young donors to cure, mitigate, treat, or prevent these conditions, and there are risks associated with the use of any plasma product.”

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