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Aakash Desai: A summary of What Biopharma Should Learn

Aakash Desai/LinkedIn

May 22, 2025, 06:38

Aakash Desai: A summary of What Biopharma Should Learn

Aakash Desai, Assistant Professor and Associate Director of Phase 1 and Precision Oncology Program at the UAB O’Neal Comprehensive Cancer Center, shared a post on LinkedIn:

“FDA ODAC Decisions in the past few weeks have been very informative!

Let’s talk about “What Biopharma Should Learn”! Here’s a summary of valuable regulatory lessons:

1) Talazoparib plus Enzalutamide (Pfizer) for mCRPC (All-Comers).

ODAC: 8–0 Against Approval.

Benefit driven by HRR-mutated subgroup.

No formal statistical plan for non-HRRm group.

Safety concerns without offsetting benefit.

Lesson: Indication expansion requires prespecified subgroup justification and efficacy across full population. Toxicity without clear benefit weakens the case.

2) UGN-102 (UroGen) for LG-IR NMIBC.

ODAC: 4–5 Against Approval.

Single-arm design (ENVISION) lacked comparator.

ATLAS trial terminated early.

Lesson: For recurrent, non-lethal conditions, comparative data is essential. Single-arm studies carry regulatory risk unless in ultra-rare diseases.

3) Glofitamab plus GemOx (Genentech) for R/R DLBCL.

ODAC: 8–1 Against Applicability to U.S. Population.

STARGLO trial showed efficacy, but North American subgroup lacked benefit.

Only 9% U.S. enrollment; prognostic imbalances.

Lesson: Global trials must include representative U.S. cohorts. Regional disparities undermine applicability—FDA needs U.S.-relevant data.

4) Daratumumab SC (Janssen) for High-Risk Smoldering Myeloma.

ODAC: 6–2 In Favor of Approval.

AQUILA trial showed PFS benefit vs. observation.

Tolerable safety, neutral QoL.

Lesson: In early/pre-malignant conditions, delay in progression can justify approval—if supported by strong prospective data in clearly defined high-risk groups.

Cross-Cutting Takeaways for Biopharma.

Predefine and power subgroup analyses—don’t extrapolate post hoc.

Prioritize RCTs and real-world relevance—especially for the U.S.

Single-arm trials are fragile—use them strategically.

Target the right population—clinical and biomarker precision matters.

Be proactive with FDA—especially when your trial hinges on population nuances.

These decisions are a timely reminder: great science must meet rigorous, patient-representative regulatory standards. Designing trials with FDA strategy in mind is essential.”

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Aakash Desai AQUILA trial Biopharma cancer Daratumumab Enzalutamide FDA fight against cancer Genentech Global fight against cancer mCRPC ODAC OncoDaily Oncology pfizer smoldering myeloma STARGLO trial Talazoparib UGN-102

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