Noxopharm cancer drug secures Orphan Drug Designation from FDA

By Jenni Spinner contact

- Last updated on GMT

(Dr_Microbe/iStock via Getty Images Plus)
(Dr_Microbe/iStock via Getty Images Plus)

Related tags: Rare disease, Orphan drug, Fda, Food and drug administration, FDA approval, Regulation

The Australian biotechnology company has been granted ODD status from the agency for Veyonda, a therapy intended for the treatment of soft-tissue sarcoma.

Australian biotech Noxopharm reports that its lead oncology drug candidate, Veyonda, has been granted Orphan Drug Designation (ODD). The therapy is intended for use in the treatment of patients diagnosed with soft tissue sarcoma.

The FDA established the ODD program to foster the development of treatments for rare diseases in the US. Soft tissues sarcomas are rare but often fatal; up to 50% of high-grade patients die within 12 months.

Gisela Mautner, Noxopharm CEO and managing director, spoke with Outsourcing-Pharma about the drug candidate’s journey to this stage, and what the ODD designation means for patients and the company.

OSP: Could you please talk about the landmarks/bright spots in Veyonda’s journey to this point in development?

GM: Veyonda is the lead drug candidate of Noxopharm, an innovative Australian biotechnology company with a proven ability to advance new drugs from discovery through preclinical and clinical studies to develop new treatments for cancer and inflammatory diseases.

Veyonda is currently being evaluated in multiple Phase I and II trials in a range of cancer types including prostate, breast, and lung cancer, and in soft tissue sarcoma. In these trials, it is being studied as a combination therapy with standard cancer treatments of either radiotherapy, chemotherapy, or immunotherapy, with the aim of enhancing the effectiveness of these therapies. Results to date have shown that Veyonda has a good safety profile and encouraging early-stage efficacy signals.

We are privileged to work with some outstanding investigators at world-class institutions. Currently, we have active study sites at a number of hospitals, including the MD Anderson Cancer Center and the City of Hope Cancer Center, amongst others. Similarly, our network of collaborators includes prestigious institutions such as the US National Institutes of Health, Weil Cornell Medicine, and Hudson Institute.

Noxopharm has secured two investigational New Drug (IND) approvals from the US FDA and trial approval from the European Medicines Agency, resulting in trials in these important commercial territories, as well as in leading hospitals in Australia. Most recently the FDA has awarded ODD to Veyonda for the treatment of soft tissue sarcoma.

OSP: What does this ODD status mean to patients diagnosed with soft tissue sarcomas?

GM: ODD enables companies like Noxopharm to develop treatments for rare diseases, a process that would otherwise be cost-prohibitive. Drug development is a very costly process, with large clinical trials required to ensure the safety and efficacy of every medicine that comes to market. These costs can outweigh the potential future profits to be made if the patient population requiring the medication is small.

Therefore, the FDA has developed the ODD pathway to confer a number of benefits 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.

Soft tissue sarcoma (STS) is a rare and devastating disease and is often fatal. Up to 50% of high-grade sarcoma patients develop metastases and die within 12 months. Treatment includes surgery, radiation therapy, and chemotherapy. Through our CEP program, we are investigating the use of Veyonda in combination with the chemotherapy drug doxorubicin, with the aim of improving the response to chemotherapy.

The support of the ODD makes it possible for Noxopharm to continue to do this work, and we hope to improve treatment outcomes for soft tissue sarcoma patients worldwide.

OSP:  You mention the benefits that the ODD means to Noxopharm and Veyonda—would you like to expand upon that?

GM: ODD offers benefits to Veyonda both during the development process and for commercialization. For Noxopharm, where our efforts are focused on advancing new drugs from discovery through preclinical and clinical studies, we see that ODD will aid our further development of Veyonda and will set this investigational drug up with the best possible chance of success.

Beyond this, the ODD will also benefit potential future purchasers or licensees of the molecule with financial, regulatory, and competitive advantages.

Financially there is a substantial benefit on several levels. For a company that has received an ODD, the New Drug Application fees, which in 2021 amounted to approximately $2.9m USD, are waived. Additionally, there is potential for assistance with opportunities for grant funding from the Office of Orphan Products Development that may assist with the clinical development program.

The FDA also offers assistance in the drug development process with regulatory guidance for ODD drugs, assistance that will provide streamlined clinical study and product registration processes.

Finally, orphan drug exclusivity provides seven years of market exclusivity, granting Veyonda protection from generic competition in the critical launch and post-launch period. This is a valuable protection that will give Veyonda a competitive advantage in the marketplace.

OSP: Do you have anything to add?

GM: Noxopharm is pleased to have achieved ODD for Veyonda and is poised to maximize the benefits from this opportunity. Our team has broad and deep experience across pharmaceutical commercialization and development and the FDA granting of the ODD for sarcoma as well as two IND approvals highlight that our work with Veyonda has been repeatedly validated.

In addition to Veyonda we have a strong product pipeline where we are developing novel proprietary drugs to address important targets for anticancer treatment and for the treatment of inflammatory diseases. We will continue to advance proprietary and in-licensed drugs through the drug discovery and preclinical stages into clinical trials. Combined with our active intellectual property strategy Noxopharm will continue to develop assets attractive to potential purchasers and licensees to ensure that these life-saving technologies will reach the market.

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