FDA Grants Orphan Drug Designation to BA-101 in Glioblastoma

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the investigational therapy BA-101 for the treatment of patients with glioblastoma.

The advancement was announced in a news release from NeuroNOS, a biopharmaceutical company that is a subsidiary of Beyond Air and manufactures BA-101.

The FDA, as the agency explains on its website, grants orphan drug designation to a drug or biological product used to prevent, diagnose or treat a rare disease or condition, and this designation qualifies sponsors for incentives such as tax credits for clinical trials, exemption from user fees and as many as seven years of market exclusivity after approval.

“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] is an important modulator of biological therapy response in glioblastoma,” said Amir Avniel, CEO of NeuroNOS, in a statement included in the news release. “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 [a nitric oxide] inhibition strategy has the potential to transform outcomes for patients.”

Glioblastoma, as defined by the National Cancer Institute, is a fast-growing type of central nervous system tumor that forms in the supportive glial tissue of the brain and spinal cord and has cells that look very different than typical cells. Glioblastoma usually occurs in adults and affects the brain more often than the spinal cord.

“Glioblastoma represents a profound unmet need," said Prof. Haitham Amal, chief science officer of NeuroNOS, in a statement of the news release. “Our published papers and unpublished data showed a strong link between [nitric oxide] and [glioblastoma]”.

Amal continued, “We are committed to working closely with regulators, investigators, patient groups, and foundations to accelerate development of BA-101 toward first-in-human studies.”

Surgery, radiation, and temozolomide chemotherapy are standard of care for patients with glioblastoma and can potentially extend survival, but they do not cure glioblastoma, according to the news release from NeuroNOS. Median survival of the disease is less than 12 months with two- and five-year survival rates lower than 20% and 10%, respectively, according to the news release.

According to the American Cancer Society, in 2025 approximately 24,820 malignant tumors of the brain or spinal cord, including 14,040 in males and 10,780 in females, will be diagnosed. Likewise, about 18,330 people, 10,170 males and 8,160 females, will die from brain and spinal cord tumors.

Study findings in mice published earlier this year in Genomic Press: Brain Medicine stated that “combining BA-101 with the antitumor drug temozolomide (TMZ) synergistically reduced tumor size and significantly increased survivability in mice bearing TMZ-sensitive cells.”

References:

1. “NeuroNOS Granted FDA Orphan Drug Designation for Glioblastoma, the Most Common and Deadliest Primary Malignant Brain Cancer in Adults,” news release, Sept. 8, 2025; https://www.globenewswire.com/news-release/2025/09/08/3146057/0/en/NeuroNOS-Granted-FDA-Orphan-Drug-Designation-for-Glioblastoma-the-Most-Common-and-Deadliest-Primary-Malignant-Brain-Cancer-in-Adults.html
2. “Designating an Orphan Product: Drugs and Biological Products,” FDA; https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products
3. “Glioblastoma,” National Cancer Institute; https://www.cancer.gov/publications/dictionaries/cancer-terms/def/glioblastoma
4. “Key Statistics for Brain and Spinal Cord Tumors,” American Cancer Society; https://www.cancer.org/cancer/types/brain-spinal-cord-tumors-adults/about/key-statistics.html

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