Market reaction
Shares of Plus Therapeutics, Inc. (NASDAQ:PSTV) rose 6.7% on Wednesday after the U.S. Food and Drug Administration granted Orphan Drug Designation for REYOBIQ (rhenium Re186 obisbemeda) to treat pediatric malignant gliomas.
Scope of the designation
The FDA’s orphan designation was issued more broadly than the original request, explicitly encompassing pediatric ependymoma in addition to other pediatric malignant gliomas. Pediatric malignant gliomas are described as rare and aggressive brain tumors that have limited treatment options and poor outcomes. The article notes that current standard treatments - including surgery and radiation - frequently do not prevent recurrence.
What orphan status means
Orphan Drug Designation is available for investigational therapies targeting rare conditions that affect fewer than 200,000 people in the United States. Per the designation’s framework, potential benefits include seven years of market exclusivity if the product is approved, tax credits for eligible clinical trial expenses, and exemptions from certain regulatory fees.
Regulatory and clinical context
The orphan designation adds to recent regulatory and clinical progress for REYOBIQ. The company has completed a Type B meeting with the FDA that supported development plans in leptomeningeal metastases, reported clinical data from the ReSPECT-LM trial, and is continuing to advance Phase 1 and Phase 2 studies. Plus Therapeutics has also received FDA clearance of its Investigational New Drug application to evaluate REYOBIQ in pediatric patients with high-grade glioma and ependymoma.
Company profile
Plus Therapeutics is a clinical-stage pharmaceutical company focused on developing targeted radiotherapeutics using platform technologies for central nervous system cancers. The company’s programs and regulatory engagements are ongoing as it advances multiple clinical and development activities for REYOBIQ.
Summary takeaway
The FDA’s orphan designation for REYOBIQ, extending to pediatric ependymoma, represents a regulatory milestone that accompanies the company’s active clinical programs and recent interactions with the FDA. The market responded with a mid-single-digit uptick in Plus Therapeutics’ share price following the announcement.