This week, Neurology News Network covered the designation of Jazz Pharmaceuticals' solriamfetol as a Schedule IV medication for excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea, ABO-202, a potential therapy for infantile Batten disease, being granted fast track designation, and new study findings that suggest that neurofilament light in the cerebrospinal fluid has multi-disease use as a biomarker for neuroaxonal damage (transcript below). 

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

Jazz Pharmaceuticals’ solriamfetol, marketed as Sunosi, an FDA-approved therapy indicated to improve wakefulness in adults with excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea, has been designated as a Schedule IV medication by the DEA.

Solriamfetol is the first and only FDA-approved dual-acting dopamine and norepinephrine reuptake inhibitor, and will be commercially available in the United States in once-daily 75 mg and 150 mg tablets in July 2019, according to Jazz.

The FDA has granted fast track designation to Abeona Therapeutics’ ABO-202, a novel one-time AAV9 gene therapy for CLN1 disease, also known as infantile Batten disease. Through its vector, the agent is designed to deliver a functional copy of the PPT1 gene to the CNS and peripheral organs using a combination of intravenous and intrathecal administration.

In May, Abeona was cleared to begin a phase 1/2 clinical trial, evaluating its novel therapy for treatment of Batten disease, following acceptance by the FDA of its investigational new drug application. The company is expected to provide guidance as to the timing of the clinical trial later this year.

New study findings suggest that neurofilament light in the CSF has multi-disease use as a biomarker for neuroaxonal damage, with the potential to help differentiate between frontotemporal dementia and Alzheimer disease, and Parkinson disease from atypical parkinsonian syndromes.

The investigators noted that there may be a need for age- and sex-specific, as well as possibly disease-specific, reference values when using cNfL as a biomarker.

As an ultrasensitive assay has already been developed for serum NfL’s use in MS, serum NfL may ultimately replace CSF NfL, though the investigators wrote that these findings can be readily translated.

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