Orphan Drug Designation Granted By US FDA For Anal Cancer - Fast Paced Pathway
- Noel Ong
- Aug 29
- 6 min read
Announcement
Invion Limited (ASX: IVX) has achieved a major regulatory milestone with the United States Food and Drug Administration (FDA) granting Orphan Drug Designation (ODD) to INV043 for the treatment of anal cancer. This recognition is more than just a regulatory tick—it represents a strategic shift in the company’s development pathway. By securing orphan status, Invion gains access to a range of benefits, including seven years of US market exclusivity, financial incentives such as tax credits and fee waivers, and the potential for accelerated approvals through smaller and shorter clinical trials.
The FDA designation not only validates the scientific promise of INV043 but also positions Invion favourably as it advances towards clinical trials in partnership with the Peter MacCallum Cancer Centre. At a time when traditional treatments for anogenital cancers, such as surgery, often come with severe side effects, INV043 offers a novel therapeutic approach that combines safety, efficacy, and innovation. For investors, this milestone underscores the potential for INV043 to become a cornerstone in Invion’s pipeline while enhancing the company’s strategic value in the broader oncology landscape.
Highlights - A Rapid Path for Anal Cancer Tests.
FDA Orphan Drug Designation Granted: INV043, Invion’s lead cancer drug candidate, now holds orphan status for anal cancer.
Commercial and Regulatory Incentives: Benefits include seven years of exclusive US marketing rights, potential tax credits, fee waivers, and a faster route to market.
Strong Preclinical Efficacy: INV043 achieved ~80% tumour control in mouse models when combined with immune checkpoint inhibitors (ICI), compared to ~12% with ICI alone.
Next Steps in Clinical Development: Invion and the Peter MacCallum Cancer Centre (Peter Mac) are preparing an anogenital cancer clinical trial, targeting anal, vulvar, and penile cancers—diseases where current surgical treatments carry severe side effects.
Clinical Impact
INV043 has shown efficacy across six squamous cell carcinoma (SCC) cell lines, covering the full spectrum of anal cancers. Importantly, no negative side effects were reported in preclinical models. This positions INV043 as a potentially safer and more effective option compared to mainstream interventions.
Invion is also advancing trials in other cancer indications, further broadening the scope of its Photosoft™ technology. In non-melanoma skin cancer, early Phase I/II clinical data have shown that topical application of INV043 is well tolerated, with patients demonstrating encouraging signs of efficacy. This highlights the potential of INV043 as a safe and non-invasive treatment option.
In addition, a Phase II prostate cancer trial has reported 40–44% positive response rates with no significant adverse events. These results reinforce the versatility of INV043 across multiple cancer types and underline the platform’s potential to deliver meaningful clinical outcomes beyond its primary anal cancer program.
Invion’s Executive Chair and CEO, Prof Thian Chew, commented:
“This regulatory milestone enhances our ability to bring INV043 forward more quickly and cost-efficiently, with meaningful benefits for patients suffering from this challenging disease.”
“Over the next five years, the pharmaceutical industry is facing its biggest patent cliff since 2010 as several blockbuster drugs, including ICIs, are expected to lose their patent protection. The Orphan Drug Designation may increase our appeal to strategic partners because of the potentially accelerated pathway to leverage intellectual property that combines their ICIs with our complementary technology.”
About Invion and Photosoft™ Technology
Invion is a life sciences company leading the global research and development of Photosoft™ photodynamic therapy (PDT). PDT uses photosensitisers and light to selectively kill cancer cells and stimulate anti-cancer immune responses. The therapy is less invasive than surgery, carries minimal side effects, and has potential applications in oncology, atherosclerosis, and infectious diseases, including treatment-resistant “superbugs”.
What is IN043?
Potential for INV043 to assist surgeons in more accurately removing cancers
Immunity – Selective Targeting (Figure 1)
Figure 1: Selective Targeting
INV043 is selectively retained in malignant tissue while sparing healthy organs, reducing toxicity and collateral damage. It combines fluorescence (405nm) for diagnostics with ablation (660nm) for therapy, offering dual theragnostic potential across multiple cancers, including pancreatic, triple-negative breast, and T-cell lymphoma.
Samso Concluding Comments
The FDA’s decision to grant Orphan Drug Designation to INV043 is a pivotal moment for Invion. It not only validates the science behind the company’s Photosoft™ platform but also creates a regulatory framework that accelerates clinical progress while reducing development costs.
The strength of the preclinical data is demonstrating meaningful tumour control with no adverse effects, and this is paired with the strategic support of the Peter MacCallum Cancer Centre. These elements provide a foundation for moving swiftly into human trials where the medical need is clear, and existing treatments often carry severe burdens.
Equally important is the timing within the pharmaceutical landscape. As several high-value drugs face patent expiry, INV043 offers potential as a complementary therapy, making Invion a more attractive partner for global pharma companies. This opens the door for strategic collaborations that could significantly alter the company’s trajectory.
For investors, the story here is not limited to anal cancer. Invion’s broader pipeline—in skin, prostate, and other cancers—underscores the versatility of the Photosoft™ technology. With regulatory momentum now in hand, Invion has an opportunity to translate clinical promise into shareholder value, marking this announcement as more than just a milestone, but rather a step into a new phase of growth.
Figure 2: Invion Limited share price chart as of 25th August 2025 (source: commsec).
From the investor's point of view, IVX is very early in its growth stage with a market capitalisation of just over AUD $10M. One may say that the risk is very high, but the invitation to receive orphan designation is a good step in the right direction. Although it is not an endorsement of the success of the business, it is a leg up. Looking at the share price journey (Figure 2), there seems to be a rise in interest positively from the recent news. One could say that a floor for the share price is developing, which would be a good stage to get some good old research happening.
This is exactly the type of development investors should be watching closely. Samso News is where investors can find grounded insights to guide their DYOR and put announcements like these into the bigger picture.
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