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news.The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Papillon Therapeutics’ PPL-001, aimed at treating Friedreich's ataxia, a rare neurodegenerative disorder.
PPL-001 is a gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy. Credit: PublicDomainPictures from Pixabay.
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This designation will offer potential benefits such as tax credits and fee waivers to facilitate the drug’s development.
The ODD status is intended for potential treatments for diseases affecting fewer than 200,000 people in the US.
This programme is designed to encourage the development of therapies for rare diseases by providing financial incentives.
Papillon’s PPL-001 has demonstrated a novel, multi-systemic method that simultaneously targets multiple organ systems.
A gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, PPL-001 targets the GAA repeat expansion in the FXN gene, which is associated with Friedreich’s ataxia.
Preclinical studies have shown that PPL-001 has the potential to improve disease phenotype across multiple affected tissues to potentially modify and reverse disease progression.
Friedreich’s ataxia impacts various body tissues, including the central nervous system and heart, leading to progressive loss of coordination and muscle strength.
Symptoms often begin in childhood or adolescence, and patients experience increasing disability over time.
Papillon Therapeutics is focused on developing genetic medicines for inherited diseases. Its technology platform offers durable expression of functional proteins, potentially addressing the root causes of genetic disorders.
Papillon Therapeutics CEO Carter Cliff said: “We are pleased that the FDA has granted orphan drug designation to our programme PPL-001.
“This significant milestone underscores our commitment to developing groundbreaking therapies for patients with rare, debilitating diseases such as Friedreich’s ataxia. We believe this therapy has the potential to treat multiple symptoms and address the unmet treatment needs of patients and their families.”
