BOSTON, Jan. 21, 2026 (GLOBE NEWSWIRE) — BPGbio, Inc., a leading biology-first, AI-powered clinical-stage biopharma company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead candidate, BPM31510, for the treatment of primary coenzyme Q10 (CoQ10) deficiency (PCQD).
Primary CoQ10 deficiency is an ultra-rare, life-threatening mitochondrial disorder affecting fewer than one in 100,000 individuals. The condition severely impairs cellular energy production, leading to developmental delays, muscle weakness, seizures, and renal alterations, urinary incontinence and potentially fatal organ damage. Current oral CoQ10 supplements often fail to reach therapeutic levels in critical organs, leaving a significant unmet medical need.
“Orphan Disease Designation marks a critical landmark in the path toward advancing closer to developing a potent precision therapy for PCQD,” said Marni Falk, MD, Executive Director of the Mitochondrial Medicine Frontier Program at Children’s Hospital of Philadelphia and Professor, Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, who serves as a compensated medical advisor to BPGbio.
The designation was supported by data-driven insights generated through BPGbio’s proprietary NAi Interrogative Biology® platform, which uniquely utilizes Bayesian AI to map thousands of human biological data points. Unlike traditional “black box” AI, BPGbio’s Bayesian-based approach allows the company to establish causal relationships in mitochondrial pathways, significantly reducing the uncertainty inherent in rare disease drug development.
“Regulatory milestones like this send an important signal to patients, clinicians, and researchers across the mitochondrial disease community that therapeutic development in rare, inherited mitochondrial disorders is advancing,” said Philip Yeske, Ph.D., Science & Alliance Officer of the United Mitochondrial Disease Foundation. “The designation of BPM31510 reflects increasing alignment between scientific insight, clinical urgency, and regulatory support for diseases that have historically been underserved.”
Key Highlights of BPM31510 for Primary CoQ10 Deficiency:
- Targeted Delivery: BPM31510 is a CoQ10-lipid nanoparticle conjugate designed for intravenous administration to deliver therapeutic levels of CoQ10 directly to the mitochondria to effectuate supraphysiological levels.
- Clinical Momentum: BPGbio recently presented encouraging preliminary clinical data from multiple compassionate use cases at various mitochondrial conferences globally and has engaged the FDA on an expedited development path.
- AI-Driven Discovery: The therapeutic potential of BPM31510 was validated using BPGbio’s proprietary NAi platform, which models patient biology to reveal new applications for mitochondrial-targeting agents.
“Receiving Orphan Drug Designation for BPM31510 in primary CoQ10 deficiency is a vital milestone in our mission to bring transformative therapies to patients with rare mitochondrial diseases,” said Niven R. Narain, Ph.D., President and CEO of BPGbio. “This designation, following our previous Rare Pediatric Disease Designation, reinforces the potential of our NAi Interrogative Biology platform to identify and develop treatments for conditions where traditional approaches have failed”.
About Orphan Drug Designation
The FDA’s Orphan Drug Designation program provides incentives to companies developing treatments for rare diseases affecting fewer than 200,000 people in the U.S. Benefits include tax credits for clinical testing, exemptions from certain FDA fees, and seven years of market exclusivity upon regulatory approval.
About BPGbio, Inc.
BPGbio is a biology-first AI-powered biopharma company focused on mitochondrial biology and protein homeostasis. Headquartered in greater Boston, the company leverages its NAi Interrogative Biology® platform and one of the world’s largest clinically annotated biobanks to develop a deep pipeline in oncology, rare diseases, and neurology.
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