FDA Accepts sNDAs for Casimersen and Golodirsen, Chris Johnson Diagnosed with ALS, Phase 2 Trial of NEU-411 Expands

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Welcome to the Neurology News Network, my name is Louie Pasculli and here’s a look at some of the top stories in Neurology.

Beginning with FDA news, the administration has accepted Sarepta Therapeutics’ supplemental new drug applications (sNDAs) for casimersen (Amondys 45) and golodirsen (Vyondys 53), 2 exon-skipping therapies indicated for Duchenne muscular dystrophy (DMD), and has assigned a PDUFA target action date of February 28, 2027. The submissions seek conversion of the existing accelerated approvals for both agents to traditional approvals, which will require the FDA to weigh confirmatory phase 3 data that narrowly missed its primary end point alongside an extensive body of real-world evidence.¹

Louise Rodino‑Klapac, PhD, president of research & development and technical operations at Sarepta, said in a statement.¹ “The FDA’s acceptance of these applications for review is an important step for the Duchenne community. The submissions draw on the ESSENCE study and years of published real-world evidence, which together offer a fuller understanding of how these therapies benefit patients and change the progression of disease. We look forward to working with the FDA throughout the review.”

In other, more sobering news, Chris Johnson, former Tennessee Titans and New York jets running back who set an NFL single-season rushing record in 2009, has been diagnosed with amyotrophic lateral sclerosis (ALS). Johnson, 40, revealed his diagnosis during an appearance on ABC's Good Morning America alongside Michael Strahan, becoming one of the most prominent athletes in recent memory to publicly disclose the disease at such a relatively young age.

Johnson’s diagnosis comes at an age well below the typical onset of ALS, which most commonly affects people between 55 and 75 years of age. The speed of his disease progression, he said, has been faster than anticipated. "ALS has continued to progress much faster than I imagined," Johnson said. "I want people to understand just how quickly ALS can attack your body."

Johnson said he first noticed weakness in his right hand last year, when his grip felt off. His condition deteriorated rapidly, and physicians confirmed the ALS diagnosis thorough testing. He now communicates using an eye-tracking speech-generating device. "ALS has changed what my body can do, but it hasn't changed who I am," Johnson said in the interview. "If sharing my story helps even one person get diagnosed sooner, inspires more research, or gives another family hope, it is worth it."

Changing gears, In a new company update, Neuron23 announced that it has received a $2.5 million grant from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to support expansion of its phase 2 NEULARK trial (NCT06680830) of NEU-411 into Israel, activating 4 clinical sites across Tel Aviv, Jerusalem, and Haifa. The company noted that the first Israeli patient has been screened, advancing what is among the first Parkinson disease (PD) trials to prospectively select participants based on genetically identified LRRK2-driven disease.²

“We were appreciative to receive support from [MJFF]. The grant represented not only important financial support for expanding NEULARK to Israel, but also validation of our scientific approach and commitment to advancing precision medicine in [PD],” Arash Rassoulpour, PhD, chief operating officer of Neuron23, told NeurologyLive®. “Expanding into Israel provides access to a highly engaged clinical and research community, enabling us to accelerate patient enrollment, deepen our understanding of genetically defined [PD] populations, and advance the development of targeted therapies such as NEU-411.”

To read the full piece and to get more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to remain informed on the latest in neurology. I’m Louie Pasculli, thanks for watching Neurology News Network.

REFERENCES
1. Sarepta Announces FDA Acceptance of sNDAs for AMONDYS 45® and VYONDYS 53®. News release. Sarepta Therapeutics. June 30, 2026. Accessed June 30, 2026. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-fda-acceptance-sndas-amondys-45r-and-vyondys
2. Neuron23 to Accelerate Expansion of NEULARK Phase 2 Trial with Support from The Michael J. Fox Foundation. News release. Neuron23. June 17, 2026. Accessed June 25, 2026. https://neuron23.com/neuron23-to-accelerate-expansion-of-neulark-phase-2-trial-with-support-from-the-michael-j-fox-foundation/