The U.S. Food and Drug Administration has granted Orphan Drug status to ADXS-cHER2 (Advaxis, Inc.) to treat patients with osteosarcoma. ADXS-cHER2 is an immunotherapy that targets the HER2 receptor in bone and breast cancers.
The Orphan Drug designation—to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.—grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.
ADXS-cHER2 is being evaluated in an ongoing veterinary clinical study in client-owned (pet) dogs with osteosarcoma. (Canine osteosarcoma is considered to be the most analogous disease to human osteosarcoma.) The data have shown that dogs treated with ADXS-cHER2 immunotherapy after standard care (amputation and follow-up chemotherapy) had a statistically significant prolonged overall survival benefit compared with dogs who received standard care without ADXS-cHER2.
The company notes that it is now planning to initiate a clinical development program with ADXS-cHER2 in pediatric patients.