Onco-Summaries: Daily Oncology Updates at a Glance

06/08/2025

Jazz Pharmaceuticals' Modeyso received accelerated approval from the FDA for Recurrent H3 K27M-mutant Diffuse Midline Glioma (Ref)

The US FDA granted accelerated approval to Jazz Pharmaceuticals' Modeyso™ (dordaviprone; oral antagonist of dopamine receptor D2 and agonist of ClpP) for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.

• Continued approval for this indication may be contingent upon validation of benefit in the Phase 3 ACTION confirmatory trial

  
  

• The approval was based on an integrated efficacy analysis from five open-label clinical trials (ONC006, ONC013, ONC014, ONC016, and ONC018) of 50 patients with recurrent H3 K27M-mutant diffuse midline glioma

  
  

  • ORR: 22% (95% CI: 12 to 36)

    
    

  • Median DOR: 10.3 months (95% CI: 7.3 to 15.2)

Galapagos' GLPG5101 CAR-T therapy received the RMAT designation from the US FDA for R/R MCL (Ref)

The US FDA granted the Regenerative Medicine Advanced Therapy designation to Galapagos' GLPG5101 (anti-CD19 CAR-T) for the treatment of relapsed/refractory mantle cell lymphoma (R/R MCL).

• Omotayo Fasan, Clinical Development Program Head, Galapagos: “This designation reflects the promising clinical activity and safety profile observed in our ongoing Phase 1/2 study and supports our commitment to delivering an effective and timely treatment option to patients in need. With RMAT status allowing for closer collaboration with the FDA, this will enable additional opportunities for accelerated development and assessment timelines.”