In late September 2022, biotech company Oncoheroes Biosciences, Inc. (“Oncoheroes”) shared via news release that its product dovitinib, which the company has an exclusive pediatric licensing agreement for from Allarity Therapeutics, had received Rare Pediatric Disease designation. Dovitinib received this designation for the treatment of osteosarcoma, a rare form of bone cancer that often manifests in children.

So what is dovitinib? Dovitinib is part of a class of therapies called tyrosine kinase inhibitors (TKIs). This pan-TKI targets receptor tyrosine kinases, fibroblast growth factor receptor (FGFR), and vascular endothelial growth factor receptor (VEGFR). Allarity explains that dovitinib:

binds to and inhibits the phosphorylation of type III-V RTKs, such as VEGFR and platelet-derived growth factor receptor (PDGFR) that promote tumor cell proliferation and survival in certain cancer cells. In addition, this agent also inhibits other members of the RTK superfamily…[which] may further lead to a reduction of cellular proliferation and angiogenesis.

Rare Pediatric Disease designation is granted to drugs or biologics intended to treat, prevent, or diagnose a rare condition in patients aged 18 or younger. These are conditions affecting fewer than 200,000 pediatric patients across the country. Under this program, drug developers whose product received this designation are eligible for a Priority Review Voucher at the time of marketing/approval.

Outside of osteosarcoma, researchers are also evaluating dovitinib for other cancers.

What is Osteosarcoma?

Also referred to as osteogenic sarcoma, osteosarcoma is a form of bone cancer which usually appears in areas of quick bone growth (such as the femur near the knee or the shinbone). Although this is a rare cancer, it is also considered the most common form of bone cancer, especially in children. In many cases, osteosarcoma appears during growth spurts in teens. For this reason, it may be misdiagnosed due to the fact that symptoms relate to what individuals may experience physically during growth spurts. Boys are more likely to develop osteosarcoma than girls.

Symptoms of osteosarcoma vary based on where the specific tumor is located. Some common symptoms can (but do not always) include:

• Limping
• Limited joint motion
• Bone pain
• Swelling or redness around the tumor location
• Bone fractures with no known cause

The cancer may be easily overlooked because of other more common causes of joint and bone pain.

Treatments for osteosarcoma include chemotherapy and surgical resection. In rare cases, surgical amputation of an affected limb may be necessary.