Florham Park, N.J. — Shionogi & Co. announced a continued partnership with the ALS/MND Natural History Consortium to collect real-world data on amyotrophic lateral sclerosis and strengthen understanding of the disease and its treatments.
The collaboration will provide Shionogi with access to data on ALS progression and approved therapies, including RADICAVA ORS, or edaravone. The company said the partnership is intended to generate insights into how ALS develops across different patient populations and how treatments perform in routine clinical settings.
“A major scientific challenge in ALS is determining how the disease presents and evolves across diverse patient populations in real-world settings. Access to real-world data is critical to addressing this complexity,” said Gustavo A. Suarez Zambrano, M.D., Vice President, Medical Affairs, Rare Disease, Shionogi Inc. “This partnership will advance our knowledge of ALS and help improve care for people living with the disease, reinforcing Shionogi’s deep commitment to research in the ALS community.”
The partnership centers on NeuroBANK, a patient-focused observational platform supported by the Neurological Clinical Research Institute at Massachusetts General Hospital. The platform collects standardized clinical data during routine visits while placing minimal additional burden on patients.
More than 4,500 people with ALS have enrolled in the study through 18 multidisciplinary clinics in the United States, Europe and Israel.
“ALS remains a devastating disease with profound unmet need, underscoring the urgency to further our understanding of disease progression,” said Alexander Sherman, Director of Center for Innovation and Bioinformatics at NCRI and the Consortium’s Principal Investigator. “By collaborating with Shionogi, we aim to deepen our perspective of ALS in real-world settings to generate insights that can support more informed care for people living with the disease.”
The U.S. Food and Drug Administration approved RADICAVA ORS in May 2022 for the treatment of ALS. The oral suspension provides an alternative to intravenous administration and received FDA orphan drug exclusivity in 2024 based on its contribution to patient care.
RADICAVA ORS is administered in treatment cycles. Patients take the therapy for 14 consecutive days followed by a 14-day drug-free period during the initial cycle. In subsequent cycles, it is taken for 10 days within a 14-day period, followed by another 14-day drug-free period.
Edaravone was discovered and developed for ALS by Tanabe Pharma Corporation. The therapy was first approved as RADICUT in Japan and South Korea in 2015 and later received regulatory authorizations in several other markets.
Shionogi acquired the global RADICAVA business and now holds worldwide rights to RADICAVA ORS and intravenous RADICAVA. The company said the two formulations have been used to treat more than 22,800 people with ALS in the United States.
