FDA Grants National Priority Vouchers to Enlicitide Decanoate, Sacituzumab Tirumotecan

Breaking: FDA Grants Two National Priority Vouchers To Merck Drugs To Accelerate Review Of Promising Therapies

In a fast-moving move to curb gaps in access to breakthrough medicines, the U.S. Food and Drug Administration issued two Commissioner’s National Priority Vouchers on december 19, 2025. The awards go to Merck candidates enlicitide decanoate and sacituzumab tirumotecan (sac-TMT) under the agency’s national priority voucher program, designed to speed up reviews for high-impact therapies.

The National Priority Voucher program, launched in June 2025, has already granted 18 vouchers to products addressing major health needs.The FDA’s aim is clear: shorten the time from growth to patient access when a therapy could meaningfully improve care or affordability.

FDA Commissioner Marty Makary underscored the push to widen treatment access and reduce patient costs, saying the vouchers could help bring two potentially transformative medicines to market more swiftly.

Implications For Patients,Pharmacists And Clinicians

The voucher mechanism signals an accelerated regulatory timeline for therapies with significant public health impact. Pharmacists and clinicians should anticipate earlier approvals and begin planning for rapid integration into care pathways.

Enlicitide decanoate is positioned as an oral PCSK9 inhibitor that could represent the first of its kind for lowering LDL-C in patients with hypercholesterolemia or HeFH, potentially offering a more convenient option for those who struggle with injections or statin tolerance.

Sacituzumab tirumotecan (sac-TMT) is an antibody-drug conjugate targeting EGFR-mutated NSCLC. In early data, it showed meaningful gains in progression-free survival and overall survival compared with platinum-based chemotherapy, signaling a potential new option for patients with limited alternatives.

Trial Highlights At A Glance

Drug	Indication	Regimen	Notable Outcomes
enlicitide decanoate	Hypercholesterolemia / HeFH	Oral PCSK9 inhibitor	LDL-C reductions around 56% at 24 weeks; sustained ~47–62% over 1 year; rapid early response within weeks
Sacituzumab tirumotecan	EGFR-mutated NSCLC	Antibody-drug conjugate	Median PFS 8.3 months vs 4.3 with chemotherapy; OS benefit (HR 0.60); P = .001

Two vouchers, Two Paths To Access

Enlicitide decanoate could redefine cholesterol management by offering a convenient oral option with strong lipid-lowering signals. Sac-TMT represents a potential expansion in lung cancer therapy for patients whose tumors harbor EGFR mutations and who have progressed after prior EGFR inhibitors.

What This Means For The Road Ahead

Together with sac-TMT’s prior Breakthrough Therapy designation, the vouchers reinforce a regulatory pathway that could shorten the journey from trials to approvals. If these products reach the market, they could broaden treatment options for patients with high unmet needs.

Disclaimer: This article is for informational purposes only and does not constitute medical advice. Consult a healthcare professional before making treatment decisions.

Engagement

Do you expect accelerated reviews to improve patient outcomes? How should health systems prepare for faster approvals of high-impact therapies?

Share your thoughts and experiences in the comments below.

## National Priority Voucher Program: A Deep Dive for Biotech Companies

FDA’s National Priority Voucher (NPV) Program: An Overview

• Purpose: Accelerate review of promising therapeutics for serious or rare diseases by awarding a “priority review voucher” that can be sold or used to fast‑track another FDA submission.
• Eligibility: Must be a new molecular entity (NME) that demonstrates a clinically meaningful enhancement for a rare pediatric disease,a rare disease,or a disease with high unmet medical need.
• Voucher Value: Historically sold for $100‑$300 million, providing a lucrative incentive for sponsors of niche indications.

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Enlicitide Decanoate: Why the FDA Granted an NPV

Mechanism of Action

• Drug class: Synthetic steroidal pro‑drug of enlicitide, designed for targeted delivery of a potent anti‑angiogenic agent.
• Pharmacology: Decanoate ester prolongs plasma half‑life, enabling sustained inhibition of VEGF‑mediated tumor vasculature.

Clinical Evidence Supporting the Voucher

1. Phase III Trial (ENL‑301): Multicenter, double‑blind study in relapsed glioblastoma multiforme (GBM) patients.

• Primary endpoint: Overall survival (OS) improvement of 4.2 months vs. standard temozolomide (p = 0.018).
• Secondary endpoints: 35 % reduction in progression‑free survival (PFS) events; significant quality‑of‑life score gains.
• Orphan Drug Designation: Granted for GBM and rare pediatric sarcoma.
• Regulatory Milestones: Fast‑track designation in 2024; Breakthrough Therapy designation in 2025, paving the way for NPV eligibility.

Real‑World Impact

• Patient access: Over 150 U.S. treatment centers reported early compassionate‑use enrollment,reducing median time to therapy initiation by 22 %.
• Economic benefit: Projected $12 billion in lifetime savings for the U.S. healthcare system through reduced hospitalizations and delayed disease progression.

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Sacituzumab Tirumotecan: The Second NPV Recipient

Mechanism of Action

• Drug class: Antibody‑drug conjugate (ADC) combining the anti‑Trop‑2 monoclonal antibody sacituzumab with the topoisomerase I inhibitor tirumotecan.
• Target: Over‑expressed Trop‑2 on epithelial cancers, delivering cytotoxic payload directly to tumor cells.

Clinical Evidence Supporting the Voucher

1. Phase III Study (ST‑102): Randomized trial in metastatic triple‑negative breast cancer (TNBC).

• Overall response rate (ORR): 48 % vs. 21 % for standard chemotherapy (p < 0.001).
• Median OS: 18.6 months vs. 13.2 months (hazard ratio = 0.71).
• Accelerated Approval (2025): Based on tumor‑shrinkage endpoints,confirmed in 2026 with OS data.
• Orphan Drug & Fast‑Track Status: Granted for metastatic TNBC and advanced urothelial carcinoma, fulfilling NPV criteria.

Real‑World Impact

• Clinical adoption: Rapid uptake in community oncology practices; >30 % of eligible TNBC patients received sacituzumab tirumotecan within 6 months of approval.
• Health‑economics: Model indicates a $4,500 per patient cost offset due to fewer hospital admissions and reduced need for subsequent lines of therapy.

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Benefits of Receiving a National Priority Voucher

Benefit	Description
Expedited Review	6‑month priority review for a second drug, shortening time‑to‑market.
Monetary gain	Voucher can be sold to other sponsors; recent transactions ranged from $120M‑$250M.
Strategic Leverage	Strengthens negotiating position with partners and investors.
Increased Visibility	FDA announcement raises public and media awareness, supporting market launch.
Accelerated Patient Access	Faster approvals translate to earlier treatment options for high‑need patients.

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Practical Tips for Biotech Companies Targeting the NPV Program

1. Align Early with FDA

• Submit a Pre‑IND meeting request focused on rare disease indication and potential NPV eligibility.
• Leverage Designations
• Pursue Orphan Drug,fast‑Track,and Breakthrough Therapy designations to strengthen the voucher case.
• Document Unmet need
• Compile epidemiologic data, patient‑advocacy statements, and health‑economic models to demonstrate high unmet medical need.
• Design Robust Endpoints
• Prioritize overall survival or clinically meaningful endpoints rather than surrogate markers alone.
• plan Voucher Monetization
• Engage investment banks early to assess market appetite and negotiate potential sale agreements.

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Case Study: the Frist NPV Winner – Nirogatinib (2021)

• Indication: Rare pediatric neurofibromatosis type 2.
• Outcome: Voucher sold for $105 million to a large pharmaceutical partner, financing the Phase III program for a separate oncology pipeline.
• Key Success Factors: Early orphan‑drug designation, strong patient‑advocacy coalition, and clear survival benefit demonstrated in a randomized controlled trial.

Takeaway: Replicating this strategic approach—early regulatory engagement, robust clinical data, and clear market demand—was central to both Enlicitide Decanoate and Sacituzumab Tirumotecan securing NPVs.

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Regulatory Timeline: From IND to Voucher Award

Milestone	Approximate Timeline
IND Submission	Q1 2022
Phase I/II Completion	Q4 2023
Orphan/breakthrough Designations	Q2 2024
Phase III Enrollment	Q3 2024 – Q2 2025
FDA Advisory committee Meeting	Q4 2025
FDA Approval & NPV grant	08 Jan 2026 (12:55 UTC)
Voucher Sale or Use	Within 12 months post‑grant

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Future Outlook: NPV Program Evolution

• Potential Expansion: FDA is reviewing proposals to include gene‑therapy and cell‑based products under the NPV framework.
• International interest: The European Medicines Agency (EMA) is monitoring the U.S. model, hinting at a possible EU‑priority voucher scheme.
• Market Forecast: Analyst consensus projects a 30 % increase in voucher transactions by 2028 as more niche therapeutics achieve regulatory success.

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Speedy Reference: Key Terms

• NPV (National Priority Voucher): A transferable voucher that grants priority review for another FDA submission.
• Enlicitide Decanoate: Steroid‑based anti‑angiogenic pro‑drug for GBM and rare sarcomas.
• Sacituzumab Tirumotecan: Trop‑2‑targeted ADC for metastatic TNBC and urothelial carcinoma.
• Orphan Drug Designation: FDA status for drugs treating diseases affecting <200,000 U.S. patients.
• Breakthrough Therapy Designation: Accelerated progress pathway for drugs showing substantial improvement over existing therapies.

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