FDA Grants Orphan Drug Designation to Novel Glioblastoma Therapy

The FDA has granted orphan drug designation to BA-101, an investigational agent for the treatment of glioblastoma (GBM), marking an important step forward in identifying novel therapies in this hard-to-treat disease.¹

“We are pleased to receive orphan drug designation from the FDA for the treatment of glioblastoma. This is our second orphan drug designation and further highlights our mission to bring targeted therapies to individuals and families affected by rare neurological conditions, while also marking our entrance into oncology,” said Amir Avniel, CEO of NeuroNOS, in a press release. “Glioblastoma is one of the most common and deadliest brain cancers in adults; however, patients have seen little improvement in treatment options over the past several decades. Emerging industry research shows that [nitric oxide (NO)] is an important modulator of biological therapy response in glioblastoma. We believe this data and the urgent unmet medical need have highlighted the opportunity for our groundbreaking science to develop small molecule therapies that balance nitric oxide levels in the brain. We believe an NO inhibition strategy has the potential to transform outcomes for patients.”

Orphan drug designation is granted to drugs and biologics that are intended for the treatment of conditions that affect less than 200,000 people per year. The designation offers incentives including tax credits toward clinical trials, fee waivers, and the potential for 7 years of market exclusivity upon approval.²

GBM is the most common primary brain malignancy in adults; however, treatment options remain limited in their efficacy. Dysregulated NO signaling and aberrant NO synthase activity have been observed in GBM biology, offering a potential target for novel therapies like BA-101. Preclinical data show that NO synthase inhibition can reduce GBM cell proliferation and tumor growth. It has also shown the potential to enhance responsiveness to temozolomide, a standard of care in this disease.

REFERENCES:

1. NeuroNOS Granted FDA Orphan Drug Designation for Glioblastoma, the Most Common and Deadliest Primary Malignant Brain Cancer in Adults. News release. NeuroNOS. September 8, 2025. Accessed September 9, 2025. https://tinyurl.com/5az8tw52

2. Designating an Orphan Product: Drugs and Biological Products. US FDA. Updated August 12, 2024. Accessed September 9, 2025. https://tinyurl.com/5ckfaxtv