Noxopharm Ltd (ASX:NOX)’s lead oncology drug candidate has been granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA).
This means Noxopharm’s Veyonda® formulation gains a number of commercial advantages as it’s developed to treat soft tissue sarcoma, a broad term for cancers that start in crucial tissues like muscles and tendons.
The US FDA’s ODD program is designed to encourage companies to develop treatments for less common disorders. Essentially, it grants orphan status to drugs that show promise to be a safe and effective treatment for diseases affecting fewer than 200,000 people per year in the US.
Noxopharm is currently undertaking a CEP-2 soft tissue sarcoma trial in the states.
Commercially valuable milestone
Speaking on the grant, Noxopharm CEO Dr Gisela Mautner said: “It is pleasing that the Noxopharm application for Orphan Drug Designation was approved so quickly.
“Considering that out of approximately 360 approved ODDs last year, only four went to Australian companies, demonstrates the high bar that is being set by the FDA.
“The seven-year period of market exclusivity is commercially extremely valuable, as it means that the FDA will not approve a subsequent drug for the same use within this timeframe.
“The ODD will significantly increase the value proposition of Veyonda to potential purchasers or licensees by both lowering current development costs and by providing future competitive and financial advantages as Veyonda progresses through the clinical trial stages towards registration and approval for sale in the US.
“With the FDA orphan drug designation now secured for Veyonda, the Noxopharm team is excited to move our preclinical assets along the drug development process, while continuing to deliver on our clinical program plan.”
Orphan Drug Designation confers a number of commercial advantages, including:
- Seven years of market exclusivity;
- Waiver of New Drug Application fees (valued at roughly $2.9 million in 2021);
- Opportunities for grant funding from the Office of Orphan Products Development; and
- Regulatory guidance and assistance from the FDA with the drug development process.
About the CEP-2 trial
CEP-2 is a phase one, open-label, dose-escalation and dose-expansion study of Veyonda® administered to cohorts of patients being treated with doxorubicin for the treatment of metastatic soft tissue sarcoma.
Roughly 30 patients in the United States with a range of soft tissue sarcomas are being enrolled to be treated with the Veyonda/doxorubicin combination as a first-line treatment.
A number of major sites are participating in CEP-2. The first, the City of Hope Cancer Center in Los Angeles, has commenced treatment, with other sites to be activated shortly.
Soft tissue sarcomas are often fatal cancers and up to 50% of high-grade sarcoma patients develop metastases and die within 12 months.
They are defined as rare cancers, with fewer than 20,000 new cases diagnosed in the US in 2021.
The CEP-Program is based on pre-clinical and clinical findings of Veyonda enhancing the anti-cancer effect of a number of standard chemotherapeutic agents.
Findings from the CEP-1 clinical trial were published in the Current Therapeutic Research journal in April 2021.