BA-101 aims to address urgent unmet needs in one of the deadliest brain cancers
NeuroNOS, a subsidiary of Beyond Air (XAIR), has announced 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). The designation provides significant incentives for development and represents an important step forward in addressing one of the most aggressive brain tumors in adults.
Glioblastoma remains one of the most difficult cancers to treat. Despite surgery, radiation, and chemotherapy with temozolomide being standard-of-care, these options are not curative. The median survival rate for patients is less than 12 months, with two- and five-year survival rates below 20% and 10%, respectively. This grim prognosis underscores the urgent need for novel treatment strategies.
“We are pleased to receive orphan drug designation from the FDA for the treatment of glioblastoma,” said Amir Avniel, CEO of NeuroNOS. “This marks our second orphan designation and highlights our mission to deliver targeted therapies for rare neurological conditions while advancing into oncology. We believe nitric oxide inhibition has the potential to transform patient outcomes.”
Research has increasingly shown that nitric oxide (NO) plays a role in modulating therapeutic responses in glioblastoma. By developing small molecule therapies that balance NO levels in the brain, NeuroNOS aims to pioneer a novel approach.
The FDA’s orphan designation is granted to drugs intended to treat rare diseases affecting fewer than 200,000 people in the U.S. Benefits include tax credits for clinical trials, waiver of certain fees, and seven years of market exclusivity if approved.
“Glioblastoma represents a profound unmet need,” added Prof. Haitham Amal, CSO of NeuroNOS. “We are committed to working with regulators, investigators, and patient groups to bring BA-101 into first-in-human studies as quickly as possible.”
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