OncoSil Medical Receives FDA Exemption Approval for Targeted Radiation Device

OncoSil Medical (ASX: OSL) has received humanitarian device exemption (HDE) approval from the US Food and Drug Administration (FDA) for its OncoSil device to treat the rare bile duct cancer distal cholangiocarcinoma (dCCA).

The approval represents a transformational step in the company’s US expansion, providing entry for the device into the world’s largest healthcare market to address patients with unresectable, non-metastatic dCCA who currently have limited treatment options.

This represents an estimated patient pool of approximately 1,000 per annum and an $80 million annual total addressable market for the device in the US.

The HDE provides a regulatory pathway for medical devices designed to treat or diagnose rare diseases or conditions affecting no more than 8,000 individuals in the US per year.

Unlike the traditional pre-market approval (PMA) pathway, an HDE does not require demonstration of reasonable assurance of effectiveness but instead relies on the FDA to determine that a device does not pose unreasonable or significant risk and that its probable benefits outweigh the risks of its use.

Post-Approval Study

OncoSil’s device is planned to be launched in the second half of 2027 and will be initially restricted to a maximum of five treatment centres with suitably trained practitioners, as part of an FDA-required post-approval study (PAS) to evaluate the safety and probable benefits in up to 30 patients over a period of 24 months.

It is expected that each patient enrolled under the PAS will receive a reimbursed treatment, representing US$1.7 million in revenue for OncoSil.

The company plans to enhance clinician education and training programs to support treatment teams during the launch and to build awareness, support patient identification, facilitate access to treatment, and drive adoption of the device across the US.

The HDE pathway is particularly relevant to OncoSil’s device given the rarity of dCCA, the poor prognosis associated with unresectable disease and the limited treatment options currently available.

‘Exciting New Chapter’

Managing director Nigel Lange said HDE approval marked the beginning of an “exciting new chapter” as the company works to make a meaningful difference to cancer patients.

“FDA approval of the OncoSil device under the HDE pathway represents a transformational moment for us, making it available for patients with dCCA in the US where there remains significant unmet clinical need,” he said.

“Our attention now turns to successfully launching OncoSil and we will work with leading cancer centres and clinical teams to establish treatment sites, advance reimbursement and market access, and build the foundations for sustainable adoption.”

“I welcome the opportunity for US specialist centres to gain experience with OncoSil and further understand its role in the multidisciplinary treatment pathway for eligible patients with dCCA,” Dr Juan Valle, chief medical officer at the Cholangiocarcinoma Foundation, added.

Rare and Aggressive Cancer

dCCA is a rare and aggressive cancer that develops in the distal common bile duct, located at the head of the pancreas.

The disease is often diagnosed at an advanced stage, as symptoms often only become apparent once the tumour has progressed to a point where surgical removal is difficult or no longer possible.

Patients with unresectable, non-metastatic dCCA have a poor prognosis with a median overall survival of approximately 6.7 months, while dCCA incidence is reported to be increasing by approximately 1.4% annually.

Treatment options remain limited and typically include biliary stenting to maintain bile duct patency together with systemic therapy and, in selected patients, radiation therapy.

The OncoSil device provides eligible US patients and clinicians with an additional treatment option through targeted radiation delivered directly within the tumour.