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FDA Grants Rare Pediatric Disease Designation to 101-PGC-005 for Systemic Juvenile Idiopathic Arthritis

• The FDA has granted Rare Pediatric Disease Designation to PIF Partners' 101-PGC-005 for treating systemic juvenile idiopathic arthritis (sJIA) flares, a severe autoinflammatory condition in children. • 101-PGC-005, a Type IA prodrug of dexamethasone, targets CD206+ macrophages and is currently in Phase 3 trials in India for COVID-19-induced acute respiratory distress syndrome. • This designation aims to incentivize the development of new therapies for rare, life-threatening diseases affecting children under 18, addressing a significant unmet medical need. • PIF Partners aims to advance its clinical program and research into other orphan diseases characterized by systemic inflammation, leveraging the drug's efficacy and reduced toxicity.

PIF Partners' investigational therapeutic 101-PGC-005 has received Rare Pediatric Disease Designation from the FDA for the treatment of systemic juvenile idiopathic arthritis (sJIA) flares. This designation highlights the potential of 101-PGC-005 to address the unmet medical needs of children suffering from this severe autoinflammatory disease.
sJIA is a rare and severe condition affecting children under 16, characterized by systemic symptoms including rash, high fevers, liver and spleen enlargement, inflammation of heart and lung linings, and joint inflammation. The FDA grants this designation to encourage the development of new drugs and biologics for serious or life-threatening diseases primarily affecting children under 18.

Mechanism of Action and Clinical Development

101-PGC-005 is a Type IA prodrug of dexamethasone that targets CD206+ macrophages. It is currently in Phase 3 clinical trials in India for treating acute respiratory distress syndrome induced by COVID-19. The drug's mechanism focuses on enhancing efficacy and reducing toxicity by targeting activated macrophages with a potent anti-inflammatory compound, while demonstrating no suppression of the hypothalamic-pituitary-adrenal (HPA) axis.

Company Statement

Alec Goldberg, CEO of PIF Partners, stated, "We are thrilled the FDA has recognized the potential of ‘005 to address the significant unmet medical needs of children suffering from sJIA. This designation is a pivotal milestone that reinforces our commitment to developing innovative therapies for rare pediatric inflammatory diseases." He further added, "By targeting activated macrophages with the most powerful anti-inflammatory compounds, we enhance efficacy and reduce toxicity... We are eager to advance our clinical program and expand our research into other orphan diseases characterized by systemic inflammation."
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Reference News

[1]
FDA grants Rare Pediatric Disease to 101-PGC-005 for systemic juvenile idiopathic arthritis
contemporarypediatrics.com · Dec 4, 2024

PIF Partners' 101-PGC-005, a Type IA prodrug of dexamethasone targeting CD206+ macrophages for systemic juvenile idiopat...

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