The BioPharm Brief: New Approval, Pediatric Progress, and Durable Remission

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. We're looking at a new FDA approval for rectal cancer, encouraging results in children with ulcerative colitis, and longer-term data for a difficult complication of Crohn disease.

First, GSK's Jemperli has received FDA accelerated approval for certain adults with locally advanced rectal cancer. The treatment is for patients whose tumors have specific genetic features known as M-S-I-high or mismatch repair deficient. In the phase 2 AZUR-1 trial, 68% of evaluable patients maintained a complete clinical response at 12 months. For eligible patients, the treatment could offer an alternative to the traditional combination of chemotherapy, radiation, and surgery. The accelerated approval means further research is needed to confirm the treatment's long-term benefits.

Next, Eli Lilly's Omvoh has delivered one-year remission results in children and adolescents with ulcerative colitis. In the phase 3 SHINE-2 trial, half of the patients who initially responded to treatment were in clinical remission at week 52. Half also achieved remission without needing corticosteroids, a class of anti-inflammatory drugs. More than half of the patients enrolled had previously not responded adequately to a biologic treatment. Lilly plans to submit the findings to regulators to support a potential pediatric label expansion. Omvoh is already approved for adults with ulcerative colitis and Crohn disease.

And Johnson & Johnson's Tremfya has shown continued improvement in fistula remission in adults with Crohn disease. In the phase 3 FUZION study, 28% of patients receiving one maintenance dosing regimen had remission of their fistulas at week 24. By week 48, that figure had risen to 35%. Fistulas are abnormal connections that can form between the bowel and nearby areas of the body, and they can be particularly difficult to treat in people with Crohn disease. The findings offer a longer-term look at Tremfya's potential to help patients manage this complication.

That’s today’s BioPharm Brief. For analysis and expert insights, please visit BioPharmInternational.com.

Key takeaways

• FDA approval: Dostarlimab offers a new treatment option for eligible patients with biomarker-defined locally advanced rectal cancer.
• Pediatric IBD: Half of the pediatric patients who initially responded to Omvoh achieved clinical remission at one year.
• Longer-term results: Tremfya maintained or improved fistula remission through 48 weeks in the phase 3 FUZION study.