South Korea’s Ministry of Food and Drug Safety (MFDS) updates the stem cell therapy’s approval in May 2026 — as the developer works to resume supply and build a U.S. research base
SEOUL, South Korea, July 20, 2026 /PRNewswire/ — Patients with amyotrophic lateral sclerosis (ALS) in South Korea will keep their access to Neuronata-R® (lenzumestrocel) — a stem cell therapy for ALS — after the Ministry of Food and Drug Safety (MFDS) approved its continued use in May 2026, updating the label to reflect data from the completed Phase 3 clinical trial.
The MFDS approved a product license modification for the therapy, which was first conditionally approved in the country in 2014. The update keeps Neuronata-R® on the market.
CorestemChemon said it is now working to resume manufacturing so patients can keep receiving the therapy, while also building a research footprint in the United States that could support broader access over time.
What the updated approval means for patients
In the ALSUMMIT (NCT04745299) Phase 3 trial, Neuronata-R® did not reach statistical significance across the full study population. In the subgroup of slow progressors, however, the therapy met its primary endpoint, the Combined Assessment of Function and Survival (CAFS), and a secondary endpoint, the ALS Functional Rating Scale-Revised (ALSFRS-R), alongside consistent improvement in slow vital capacity (SVC).
In the same subgroup, treatment lowered neurofilament light chain (NfL), a marker of axonal damage released when neurons are injured. NfL is gaining acceptance as a key biomarker in ALS and is increasingly recognized as a basis for regulatory decisions on disease-modifying therapies.
Building a U.S. research base
CorestemChemon has also been establishing a presence in the United States through the regenerative medicine research community. In late 2025, it became the first South Korean company to join an innovation engine supported by the U.S. National Science Foundation (NSF) — the Piedmont Triad Regenerative Medicine Engine, anchored by the Wake Forest Institute for Regenerative Medicine (WFIRM).
Joining the engine gives the company access to WFIRM’s research infrastructure and network, and it has consolidated its U.S. operations in North Carolina to anchor that work.
On the regulatory front, CorestemChemon said it is pursuing a Type C meeting with the FDA, with the goal of filing a Biologics License Application (BLA) for U.S. approval next year.
Full-scale production of Neuronata-R® is expected to be up and running early next year, when treatment is set to resume. From that point, patients from outside South Korea will also be able to travel to the country to receive the therapy.
CorestemChemon has framed the MFDS decision as a step toward keeping a cell-based option available for the patients most likely to benefit, and toward extending that option to patients beyond South Korea over time.
How Neuronata-R works
Neuronata-R® uses autologous bone marrow-derived mesenchymal stem cells (MSCs), grown in the lab from the patient’s own bone marrow and delivered by intrathecal injection.
Rather than replacing motor neurons, the MSCs are thought to act through the factors they secrete. These include anti-inflammatory factors — TGF-β1, IL-4 and IL-10, which help dampen the neuroinflammation that contributes to motor neuron loss — and neuroprotective factors such as VEGF, BDNF and IGF, which support neuronal survival. Together, these factors are proposed to slow the death of motor neurons.
To date, the therapy has been given to more than 400 patients in South Korea, building a body of real-world experience and safety data. It holds Orphan Drug Designation from the U.S. Food and Drug Administration (FDA), granted in 2018, and from the European Medicines Agency (EMA), granted in 2019.
Sumber artikel: http://www.prnasia.com/story/archive/5006054_CN06054_0
