In the United States, Orphan Drug designation is granted by the FDA to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. “Rare” is defined as any condition affecting under 200,000 Americans. Additionally, Orphan Drug designation comes with benefits for the drug developer. These include fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity upon approval. According to a late May 2022 news release from biotechnology company Noxopharm, its leading candidate Veyonda, developed for patients with soft tissue sarcoma (STS), recently earned this designation.
About Veyonda
Noxopharm describes Veyonda as:
A novel proprietary formulation of idronoxil, a first-in-class, dual-acting oncotoxic and immuno-oncology molecule. In cancer, Veyonda appears to have two actions, both directly killing cancer cells and working with the body’s immune system to destroy tumors.
Currently, Noxopharm is evaluating Veyonda for STS in a Phase 1 clinical trial. The drug will be evaluated in conjunction with doxorubicin, a chemotherapy drug.
What is Soft Tissue Sarcoma (STS)?
Soft tissue sarcoma (STS) is a rare form of cancer which manifests in soft tissues – such as muscles, nerves, tendons, or blood vessels – throughout the body. There are multiple different forms of STS, including (but not limited to) synovial sarcoma, liposarcoma, and angiosarcoma. In fact, there are over 50 different forms!
Unfortunately, STS can sometimes be hard to detect and diagnose. This is because many people remain asymptomatic until the cancer has progressed to later stages. As the tumor progresses, symptoms may begin to appear. These differ based on which form of STS one has. However, some potential common symptoms include:
- Pain (if the tumor is pressing on nerves or muscles)
- Indigestion or constipation (if the tumor is in the abdomen)
- Black, tarry stools
- Blood in your stool or vomit
- A noticeable lump or mass or a mass which was removed and has returned
