Plus Therapeutics Inc. (NASDAQ:PSTV) said the U.S. Food and Drug Administration has granted Orphan Drug Designation to REYOBIQ (rhenium Re186 obisbemeda) for the treatment of pediatric malignant gliomas.
The FDA awarded the designation for malignant glioma more broadly than the company had initially sought, extending it to include pediatric ependymoma. Pediatric malignant gliomas are rare and aggressive brain cancers, affecting roughly 3.3 people per 100,000, and current treatment options remain limited with generally poor outcomes.
Orphan Drug Designation is reserved for therapies targeting rare diseases that affect fewer than 200,000 people in the United States. The status provides several incentives, including seven years of market exclusivity if the therapy is approved, tax credits for eligible clinical trial expenses and waivers of certain regulatory fees.
“Receiving orphan drug designation for REYOBIQ in pediatric malignant gliomas, including the broader scope for progressive pediatric ependymoma, is an important milestone and further validates our approach to delivering targeted radiotherapy directly to CNS tumors,” said Marc Hedrick, president and chief executive officer of the company.
REYOBIQ is an injectable radiotherapy designed to deliver concentrated radiation doses directly to tumors in the central nervous system. According to the company, standard treatments such as surgical tumor removal and external beam radiation therapy often fail to prevent recurrence, with five-year survival rates for high-grade gliomas as low as 22%.
Plus Therapeutics has also received FDA clearance for its Investigational New Drug application to evaluate REYOBIQ in pediatric patients with high-grade glioma and ependymoma. The company is currently conducting several clinical studies, including the ReSPECT-GBM, ReSPECT-LM and ReSPECT-PBC trials.
The Houston-based clinical-stage pharmaceutical company focuses on developing targeted radiotherapeutics aimed at treating cancers of the central nervous system. The information reported is based on a company press release.