SYRACUSE, NY / ACCESS Newswire / May 12, 2026 / Repair Biotechnologies, Inc., a biotechnology company developing first-in-class therapies capable of rapidly regressing atherosclerotic plaque, today announced that the U.S. Food and Drug Administration (FDA) has granted eligibility for the Rare Disease Evidence Principles (RDEP) program for its investigational messenger RNA (mRNA) therapy, REP-0003, in the treatment of homozygous familial hypercholesterolemia (HoFH).
REP-0003 was granted Orphan Drug Designation by the FDA in May 2025 for the treatment of severe atherosclerosis in patients with HoFH. This RDEP eligibility further strengthens the Company's collaborative relationship with the FDA as it advances this first-in-class, disease-modifying therapy toward the treatment of patients in need.
This milestone aligns closely with the FDA leadership's public emphasis on mechanism of action (MOA) driven approvals and efficient development pathways for therapies addressing high unmet need rare diseases. Under RDEP, substantial evidence of effectiveness can be demonstrated with one adequate and well-controlled clinical study, supported by confirmatory evidence. This offers a streamlined, science-based route to approval.
REP-0003 is an mRNA therapy based on Repair Biotechnologies' proprietary Cholesterol Degrading Platform (CDP). It encodes an enzyme that selectively and safely degrades the previously undruggable target of toxic excess free cholesterol inside cells into a non-toxic, readily excreted metabolite. In preclinical studies using LDLR-knockout mouse models of HoFH, addressing free cholesterol toxicity with REP-0003 produced rapid regression of established atherosclerotic plaque, marked improvements in liver function, and enhanced exercise capacity.
HoFH is a very rare, life-threatening genetic disorder caused primarily by mutations in both copies of the LDLR gene. Patients exhibit extremely elevated LDL cholesterol from birth, leading to accelerated atherosclerosis, myocardial infarction, and premature cardiovascular death, often in childhood or early adulthood. Current treatments do not reliably or adequately control the condition, and carry significant tolerability, adherence, or access burdens.
"Receiving RDEP eligibility is a transformative validation of our science and the therapeutic potential of REP-0003 for patients with HoFH," said Dr. Mourad Topors, the Chief Scientific Officer of Repair Biotechnologies. "Our Cholesterol Degrading Platform directly targets the intracellular free cholesterol toxicity that drives accelerated plaque formation when LDLR function is impaired. Our compelling preclinical data shows rapid plaque regression and functional recovery. This, combined with RDEP's flexible evidence framework, positions us to deliver a truly disease-modifying therapy that could dramatically improve outcomes and quality of life for patients."
Reason, CEO of Repair Biotechnologies, added: "The FDA decision is very exciting. It derisks and accelerates our development program, emphasizing the strength and capability of our CDP platform. With Orphan Drug Designation in hand and now RDEP eligibility, we have a clear, efficient path forward that aligns with the Agency's evolving focus on mechanism-based innovation. This milestone not only brings us closer to patients who have waited far too long for better options but also represents a major value-creating opportunity for Repair Biotechnologies and the investors who share our vision of delivering transformative cardiovascular and rare-disease therapies."
The company plans to open an Investigational New Drug (IND) application to enable continued close collaboration with the FDA at key development milestones.
About Repair Biotechnologies:
Repair Biotechnologies develops first-in-class therapies that break down the undruggable target of toxic free cholesterol inside cells to rapidly reduce arterial plaque in atherosclerosis and familial hypercholesterolemia, reverse liver fibrosis in metabolic disorders, and which have the potential to treat numerous other conditions associated with aging and obesity.
https://www.repairbiotechnologies.com
Medial Contact:
Reason, CEO, Repair Biotechnologies, Inc.
info@repairbiotechnologies.com
SOURCE: Repair Biotechnologies, Inc.
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