Medpacto said that the U.S. Food and Drug Administration has granted an orphan drug designation (ODD) to the combination therapy of Vactosertib and PD-L1 inhibitors for treating osteosarcoma.

Vactosertib received the FDA designation for the second time this year alone.  Medpacto received orphan drug designation for Vactocertib in pancreatic cancer in July, rapidly expanding the indications from common carcinomas to rare cancers.

Patients with osteosarcoma generally receive surgery, chemotherapy, and radiation therapy. About 45.5 percent of them undergo operation combined with chemotherapy, and only 15.9 percent solely depend on medicines. As chemotherapy relies on relatively old methods, however, the demand for new therapeutic options steadily increases.

In addition, Medpacto and researchers of the Case Western Reserve University Angie Fowler Adolescent and Young Adult (AYA) Cancer Institute in the U.S. tested the effect of inhibiting the growth and metastasis of osteosarcoma cells in a joint study of the combo therapy of Vactosertib and immune-oncology in an animal model. Vactosertib was also effective when administered alone.

“We have confirmed that cancer cell growth was inhibited according to the dose and concentration of Vactosertib in tests on several osteosarcoma cells,” a Medpacto official said. “We hope that this combination therapy with an immuno-oncology drug in osteosarcoma will be a new treatment now that the FDA has designated it as an orphan drug.”

Osteosarcoma is a malignant tumor that occurs in bone or osteoid tissue, such as cartilage around the bone, and it is rare cancer that accounts for 5.6 percent of sarcoma. It is one of the most common childhood cancers.

With the recent ODD status, Medpacto can apply for priority review with exempted review fee. In addition, the company gets seven-year exclusive marketing rights when the drug wins FDA approval. Medfacto plans to receive ODD for Vactosertib in treating other rare diseases.