Aptorum Group Receives FDA Orphan Drug Designation for SACT-1 to Treat Neuroblastoma

Jan 20, 2022 By MarketDepth

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FDA Approval

Aptorum Group Limited (NASDAQ: APM) has announced that the United States Food and Drug Administration Office has granted Orphan Drug Designation to SACT-1, small molecule compound to treat patients with Neuroblastoma.  Aptorum Group will file an Investigational New Drug Application to proceed to phase 1b/2a clinical trial for SACT-1 to test the drug in neuroblastoma patients in 2022.

“The granting of orphan drug designation for SACT-1 for the treatment of neuroblastoma is another important step forward in the development of our drug candidate and reflects both the FDA’s and Aptorum’s commitment to addressing the unmet clinical needs of patients with neuroblastoma.” Further to our recently announced completion of Phase 1 clinical trial and patent grant for SACT-1, we are currently focusing on our IND preparation for entering into the exciting Phase Ib/2a clinical trials for SACT-1 in the United States.”

Mr. Darren Lui, President and Executive Director of Aptorum Group

SACT-1 is an orally administered repurposed small molecule drug to target neuroblastoma.  Neuroblastoma is one of the most prevailing solid tumor cancers in children, representing 8% – 10% of all childhood tumors, accounting for c. 15% of all cancer related deaths in the pediatric population.