Takeda and Recordati secure PBAC nods in oncology and rare diseases

The Pharmaceutical Benefits Advisory Committee (PBAC) has recommended the listing of two drugs: one for metastatic bowel cancer and the other for Cushing syndrome, a rare disease.

Takeda Pharmaceuticals has received a positive recommendation for its VEGF inhibitor Fruzaqla (fruquintinib) in the treatment of metastatic bowel (colorectal) cancer. Takeda acquired the rights to the drug outside of China in January 2023 from Hutchmed for an upfront payment of $400 million, with potential milestone payments totalling $730 million.

The PBAC highlighted the significant clinical need for alternative treatments within this patient group, stating that Fruzaqla would offer an additional option with a modest benefit in both the third- and fourth-line settings.

Their recommendation was based, in part, on an assessment that the cost-effectiveness of Fruzaqla would be acceptable if it were cost-minimised to Servier’s Lonsurf (trifluridine/tipiracil) based on equi-effective doses.

Notably, data from a global trial involving nearly 700 heavily pre-treated patients demonstrated a 34% reduction in the risk of death for those with refractory metastatic bowel cancer who received fruquintinib compared to those given a placebo.

In related news, Recordati has finally secured a positive recommendation for Isturisa (osilodrostat), a cortisol synthesis inhibitor. Although the therapy was approved by the Therapeutic Goods Administration (TGA) in 2022, the PBAC had previously rejected its submission twice due to cost concerns, despite acknowledging its efficacy and the high clinical need.

Novartis, which previously owned Isturisa, transferred the asset to Recordati in 2019 as part of a $390 million divestment of its endocrinology portfolio. Phase 3 data indicated that 86% of patients treated with Isturisa achieved normal cortisol levels in their urine after eight weeks, compared to just 29% of patients receiving a placebo.

The PBAC recognised the evidence presented in the early resolution resubmission, which underscored the value of reducing cortisol levels in the short, medium, and long term. They concurred that Isturisa could have a positive impact on multiple patient-relevant outcomes over time, distinguishing it from other chronic therapies that were recommended based on incremental cost per responder analyses.

However, the PBAC noted that the expected duration of use remains uncertain and indicated that a risk-sharing arrangement would be necessary to manage the estimated cost to the government.

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