Kintara’s VAL-083 Gets Orphan Drug Status for Rare Childhood Cancer

The FDA has granted Orphan Drug status to Kintara Therapeutics’ VAL-083 candidate for the treatment of diffuse intrinsic pontine glioma, a rare, very aggressive and hard to treat childhood brainstem tumor.

VAL-083 is a chemotherapeutic that has shown potential effects against several cancers, including in the central nervous system, ovaries, lung, bladder, head and neck.

The drug is being advanced into a phase 2/3 clinical trial supported by the company’s internal research and National Cancer Institute-sponsored clinical studies.

Orphan Drug status is given to investigational drugs or biological products to prevent, diagnose or treat a rare disease or condition. Sponsors benefit from tax credits for qualified clinical trials, exemption from user fees and the potential for seven years of market exclusivity.