Australian biotech Noxopharm has received an Orphan Drug Designation (ODD) from the Food and Drug Administration (FDA) for its lead oncology candidate in the treatment of soft tissue sarcoma.
Noxopharm’s Veyonda is a formulation of a first-in-class, dual-acting oncotoxic and immuno-oncology molecule known as idronoxil. Idronoxil is currently under investigation for improving the effectiveness of immunotherapy, chemotherapy and radiation therapy treatment for a range of cancers. As an immuno-oncology drug, Veyonda works by killing cancer cells and working with the body’s immune system to destroy tumours.
Veyonda is currently being investigated in a Phase 1 CEP-2 trial at City of Hope in Los Angeles in combination with the chemotherapy drug doxorubicin, for first-line treatment of soft tissue sarcoma.
The FDA developed the ODD programme to encourage the development of treatments for rare diseases and disorders that affect fewer than 200,000 people in the US every year. Soft tissue sarcomas are a rare type of cancer that are often fatal for many who get diagnosed with the disease.
The ODD programme has been designed to provide a number of benefits for companies including a seven-year period of market exclusivity. It also includes a waiver of new drug application fees, valued at around $2.9 million, opportunities for grant funding, and regulatory guidance from the FDA.
“Only four of approximately 360 approved ODDs last year went to Australian companies, which demonstrates the high bar that is being set by the FDA,” said Noxopharm CEO and Managing Director Gisela Mautner. “It is pleasing that the Noxopharm application for Orphan Drug Designation was approved so quickly.”
“This designation will allow us to lower current development costs and provide a future competitive and financial advantage of Veyonda,” Dr Mautner said. “With the ODD now secured, my team will be able to focus on moving our preclinical assets along the drug development process, while continuing to deliver on our clinical programme plan.”