FDA Approves Optune Pax for Locally Advanced Pancreatic Cancer, First New Treatment in Decades

The U.S. Food and Drug Administration approved Novocure's Optune Pax device for treating adult patients with locally advanced pancreatic cancer concomitant with gemcitabine and nab-paclitaxel. The approval marks the first new treatment option for locally advanced pancreatic cancer in decades, representing the first clearance by the agency in this indication since Eli Lilly's Gemzar (gemcitabine) chemotherapy drug in 1996.

The approval came earlier than the anticipated second-quarter 2026 timeline. Novocure's shares on the Nasdaq stock exchange climbed by around 37% to a market open of $14.40 on February 12 following news of the FDA approval, representing an uptick from $10.50 previously.

The FDA approval is based on data from a successfully completed phase III study — PANOVA-3 — which evaluated Optune Pax, concomitant with gemcitabine and nab-paclitaxel (gem/nab-pac) as a first-line treatment for locally advanced pancreatic cancer, compared with gem/nab-pac alone. The randomised Phase III trial (NCT03377491) followed 571 patients for a minimum 18 months and met its primary endpoint, demonstrating a statistically significant improvement in median overall survival.

Treatment with Optune Pax concomitantly with gem/nab-pac resulted in a median overall survival of 16.2 months, compared with 14.2 months for gem/nab-pac alone. Data indicated that 285 patients in the TTFields-gem/nab-pac arm had mOS of 16.2 months, compared to 14.2 months among 286 patients treated with gem/nab-pac alone – reflecting a statistically significant two-month improvement. The one-year survival rate was 68.1% in patients receiving Optune Pax and gem/nab-pac compared to 60.2% for gem/nab-pac alone.

The median time to pain progression also improved to 15.2 months in patients treated with Optune Pax concomitant with gem/nab-pac, compared with 9.1 months for patients receiving gem/nab-pac alone.

In the trial's modified per protocol (mPP) population, defined as patients who received at least 28 days of Optune Pax therapy with gem/nab-pac or at least one complete cycle of gem/nab-pac, 198 patients treated with Optune Pax concomitantly with gem/nab-pac had a mOS of 18.3 months compared to 15.1 months in 207 patients treated with gem/nab-pac alone.

Optune Pax uses a novel biophysical approach that delivers Tumor Treating Fields (TTFields) to disrupt cancer cell division and survival. The portable device delivers tumour treating electric fields via arrays worn on the abdomen. These target the electrical properties of cancer cells to disrupt processes critical for cancer cell division and survival. The device's approach is intended to result in cell death without significantly affecting healthy cells.

The device-drug combo therapy marks a new treatment option for locally advanced pancreatic cancer, where systematic therapies have shown poor bioavailability, leading to limited effectiveness. Data from the PANOVA-3 study demonstrated that treatment with Optune Pax led to statistically significant improvement in overall survival without adding to the systemic side effects commonly associated with existing therapies.

Optune Pax was well-tolerated and did not exacerbate gem/nab-pac-related systemic toxicity. No new safety signals were observed, and serious adverse events (SAEs) were comparable between study arms, with the most common being sepsis and cholangitis.

The approval further validates Novocure's Tumor Treating Fields technology as a non-invasive device-based approach to solid tumors. Novocure has a couple of commercialized therapies using TTFields — Optune Gio and Optune Lua — to treat glioblastoma multiforme (GBM) and metastatic non-small cell lung cancer (mNSCLC), respectively. Novocure also has a TTFields device known as Optune Lua that gained approval from the FDA in October 2024 for use alongside PD-1/PD-L1 inhibitors (immunotherapy) or docetaxel in adult patients with metastatic non-small cell lung cancer (mNSCLC).

The company has submitted a premarket approval application for using TTFields therapy to treat brain metastases from NSCLC. It is also developing therapies for treating metastatic pancreatic cancer, newly diagnosed GBM.

Last month, Novocure reported preliminary results for fourth-quarter and full-year 2025. The company recorded preliminary net revenues of $174.4 million, up 8% year over year. The metric for the full year was $655 million.

Per a Towards Healthcare report, the pancreatic cancer market is estimated to grow from $3.01 billion to $9.57 billion between 2025 and 2034, at a CAGR of 13.7%. The growth is being fueled by rising cases of pancreatic cancer, growing research and development activities, and technological advancements.