On September 8, 2025 NeuroNOS, a biopharmaceutical company focused on developing treatments for neurological disorders and neuro-oncology, and a subsidiary of Beyond Air (NASDAQ: XAIR), reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead investigational therapy, BA-101, for the treatment of Glioblastoma (GBM) (Press release, Beyond Air, SEP 8, 2025, View Source [SID1234655836]).
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GBM is an aggressive primary brain tumor with limited treatment options and poor prognosis under current standard-of-care approaches. While surgery, radiation, and temozolomide are standard of care and can extend survival, they are not considered curative in glioblastoma. Median survival is less than 12 months with two and five-year survival being less than 20% and 10%, respectively.
"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. "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 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."
The FDA grants ODD to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. ODD provides certain incentives, such as tax credits toward the cost of clinical trials upon approval and prescription drug user fee waivers. If a product receives Orphan Drug Status from the FDA, that product is entitled to seven years of market exclusivity for the disease in which it has ODD, which is independent from intellectual property protection.
"Glioblastoma represents a profound unmet need," said Prof. Haitham Amal, CSO of NeuroNOS. "Our published papers and unpublished data showed a strong link between NO and GBM". Prof. Amal continues, "We are committed to working closely with regulators, investigators, patient groups, and foundations to accelerate development of BA-101 toward first-in-human studies."
About Glioblastoma
Glioblastoma (GBM) is the most common and aggressive malignant primary brain tumor in adults. Despite surgery, radiation, and chemotherapy (typically temozolomide), median survival is measured in months. Dysregulated nitric oxide (NO) signaling and aberrant nitric oxide synthase (NOS) activity-including neuronal NOS (nNOS)-have been implicated in GBM biology, supporting tumor proliferation, invasiveness, angiogenesis, and therapy resistance. Preclinical studies report that NOS inhibition, including nNOS-targeting compounds, can reduce GBM cell proliferation and tumor growth and may enhance responses to temozolomide in model systems.