SYDNEY– Australian biotech company Noxopharm (ASX:NOX) has announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to Veyonda®, its lead oncology drug candidate, for use in the treatment of soft tissue sarcoma.
The ODD program was established by the FDA to encourage development of safe and effective treatments of rare diseases and disorders that affect fewer than 200,000 people in the U.S. annually. Soft tissue sarcomas are rare but often fatal cancers — up to 50% of high-grade sarcoma patients develop metastases and die within 12 months.
“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, M.D.-Ph.D., MPH. “It is pleasing that the Noxopharm application for Orphan Drug Designation was approved so quickly.”
Noxopharm recently began its Phase 1 CEP-2 trial at City of Hope in Los Angeles for Veyonda, in combination with the chemotherapy drug doxorubicin, for first-line treatment of soft tissue sarcoma.
The ODD program provides a number of significant benefits, including:
- A seven-year period of market exclusivity — the FDA will not approve a subsequent drug for the same use within this timeframe
- Waiver of new drug application fees, valued at approximately $2.9 million in 2021
- Opportunities for grant funding from the Office of Orphan Products Development
- Regulatory guidance and assistance from the FDA for the drug development process
“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 program plan.”
