FDA Grants Orphan Drug Designation to BA-101 for Glioblastoma Treatment.

NeuroNOS, a biopharmaceutical company, has received FDA Orphan Drug Designation for BA-101, its lead investigational therapy, for the treatment of Glioblastoma (GBM). GBM is an aggressive primary brain tumor with limited treatment options and poor prognosis. The designation highlights NeuroNOS' mission to bring targeted therapies to individuals with rare diseases.

NeuroNOS, a biopharmaceutical company specializing in neurological disorders and neuro-oncology, has received a significant milestone in its quest to develop innovative treatments. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to NeuroNOS' lead investigational therapy, BA-101, for the treatment of Glioblastoma (GBM) [1].

Glioblastoma is an aggressive primary brain tumor with limited treatment options and a poor prognosis. Current standard-of-care approaches, including surgery, radiation, and temozolomide, can extend survival but are not curative. Median survival is less than 12 months, with two and five-year survival rates below 20% and 10%, respectively [1].

The FDA grants ODD to drugs and biologics intended for rare diseases or disorders affecting fewer than 200,000 people in the U.S. This designation provides certain incentives, such as tax credits for clinical trials and prescription drug user fee waivers. If a product receives ODD, it is eligible for seven years of market exclusivity for the disease, independent of intellectual property protection [1].

NeuroNOS' CEO, Amir Avniel, expressed satisfaction with the designation, stating, "We are pleased to receive orphan drug designation from the FDA for the treatment of glioblastoma. This further highlights our mission to bring targeted therapies to individuals and families affected by rare neurological conditions, while also marking our entrance into oncology" [1].

The company's Chief Scientific Officer, Prof. Haitham Amal, emphasized the unmet medical need for Glioblastoma, noting that "Glioblastoma represents a profound unmet need. Our published papers and unpublished data showed a strong link between NO and GBM" [1].

NeuroNOS' research focuses on managing nitric oxide (NO) levels in the brain, a key modulator of biological therapy response in Glioblastoma. The company aims to develop small molecule therapies that balance NO levels, potentially transforming outcomes for patients [1].

This designation is a significant step forward for NeuroNOS, which has demonstrated promising preclinical results in managing NO levels in brain-related diseases such as Alzheimer's and brain cancers. The company's collaboration with leading research institutions and experts in the field underscores its commitment to advancing medical innovation and delivering life-changing treatments [1].

References:
[1] https://www.stocktitan.net/news/XAIR/neuro-nos-granted-fda-orphan-drug-designation-for-glioblastoma-the-56vtkdrvf7l5.html