Medicure is developing MC-1, an investigational therapy for PNPO deficiency, an ultra-rare pediatric neurometabolic disorder with significant unmet medical need, and the program has received multiple FDA and EMA designations.

If MC-1 is approved, Medicure may be eligible for a Priority Review Voucher (PRV), a valuable FDA incentive that can accelerate future drug reviews or be sold to generate substantial reinvestment funding. Together, MC-1's clinical progress and potential PRV eligibility represent a meaningful opportunity to advance patient care while supporting Medicure's long-term growth and innovation strategy.

MC-1, PNPO Deficiency, and the PRV Program

PNPO Deficiency

Medicure is actively investigating MC-1, a pharmaceutical-grade formulation of pyridoxal-5'-phosphate (PLP), for the treatment of PNPO deficiency, an ultra-rare genetic neurometabolic disorder that often presents with neonatal-onset seizures.¹

Key Highlights

Phase 3 Clinical Trial: Medicure is conducting a Phase 3 clinical trial (MEND-PNPO) evaluating MC-1 for PNPO deficiency²
Regulatory Designations:
- The FDA has granted both a Rare Pediatric Disease Designation and an Orphan Drug Designation for MC-1 for PNPO deficiency³
- The European Medicines Agency (EMA) has granted Orphan Drug Designation to MC-1 for PNPO deficiency³
- The FDA has granted Fast Track designation for MC-1, supporting expedited development and review⁴
PRV Eligibility: Upon approval of the NDA for MC-1 for patients with PNPO deficiency, Medicure may be eligible to receive a Priority Review Voucher (PRV) from the FDA ⁴

How the Priority Review Voucher (PRV) Program Supports Rare Disease Innovation

Developing therapies for very small patient populations is scientifically difficult, financially risky, and often not viable without policy incentives. To address this gap, the U.S. FDA created the Rare Pediatric Disease Priority Review Voucher (PRV) Program in 2012.

What the PRV Does

When a company receives FDA approval for a rare pediatric disease therapy, it may be awarded a Priority Review Voucher.
This voucher can:
- Shorten FDA review of another drug application from ~12 months to ~6 months
- Be sold to another company, often for $150–$200M or more, generating critical reinvestment funding for rare-disease research

Impact of the PRV Program

Since its inception:

More than 60 priority review vouchers have been issued for innovative treatments across over 40 rare pediatric diseases
The program has helped enable therapies in 47 rare pediatric indications, according to industry analysis
The availability of vouchers has driven over $4 billion in reinvestment dollars back into rare-disease pipelines-funding that fuels new discoveries and supports ongoing clinical trials

The Opportunity Ahead

Medicure's work in PNPO deficiency represents both a commitment to patients and a strategic opportunity for the company's future. The potential approval of MC-1, combined with eligibility for a Priority Review Voucher - could:

Accelerate future development programs
Strengthen Medicure's rare-disease portfolio
Enable reinvestment into new therapeutic areas
Support long-term growth and innovation

Our focus remains on delivering meaningful therapies for patients with rare diseases while building a sustainable foundation for Medicure's future.

References

1. Plecko B, Mills P. PNPO Deficiency. 2022 Jun 23. In: Adam MP, Feldman J, Mirzaa GM, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2024. Available from: https://www.ncbi.nlm.nih.gov/books/NBK581452/

2 MEDICURE ANNOUNCES FDA PROVIDES COMPLETE APPROVAL TO ENROLL PATIENTS IN ITS PIVOTAL PHASE 3 TRIAL FOR TREATMENT OF RARE PEDIATRIC DISEASE. Medicure Inc. (2023, November 23). www.medicure.com

3 MEDICURE ANNOUNCES UPDATE ON ENROLLMENT IN ITS MEND-PNPO PHASE 3 CLINICAL TRIAL AND PRESENTATION OF THE TRIAL DESIGN AND BASELINE DATA AT THE 2025 CHILD NEUROLOGY SOCIETY CONFERENCE (2025, September 25) www.medicure.com

3 MEDICURE RECEIVES US FDA FAST TRACK DESIGNATION FOR MC-1 FOR PNPO DEFICIENCY. Medicure Inc. (2024, April 23). www.medicure.com

MC-1, PNPO Deficiency, and the PRV Program

PNPO Deficiency

Key Highlights

How the Priority Review Voucher (PRV) Program Supports Rare Disease Innovation

What the PRV Does

Impact of the PRV Program

The Opportunity Ahead

References

Our Strategic Commitment to Rare-Disease Therapeutics

MC-1, PNPO Deficiency, and the FDA's PRIORITY REVIEW VOUCHER (PVR) PROGRAM

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