FDA grants Rare Pediatric Disease to 101-PGC-005 for systemic juvenile idiopathic arthritis | Image Credit: © Araki Illustrations - © Araki Illustrations - stock.adobe.com.

PIF Partners recently announced that 101-PGC-005 (‘005), a proprietary investigational therapeutic for the treatment of systemic juvenile idiopathic arthritis (sJIA) flares, has received Rare Pediatric Disease Designation from the FDA. The treatment is a Type IA prodrug of dexamethasone that targets CD206+ macrophages that is also currently in phase 3 clinical trials in India to treat acute respiratory distress syndrome induced by COVID-19.¹

The rare and severe autoinflammatory disease that impacts children under 16 years of age is characterized by systemic symptoms such as rash, high spiking fevers, enlargement of the liver and spleen, and inflammation of the heart and lung linings, as well as joint inflammation.¹

The federal agency grants Rare Pediatric Disease Designation to promote the development of new drugs and biologics for serious or life-threatening diseases that mainly impact children under 18 years of age.¹

“We are thrilled the FDA has recognized the potential of ‘005 to address the significant unmet medical needs of children suffering from sJIA,” said Alec Goldberg, CEO of PIF Partners, in a press release.¹

“This designation is a pivotal milestone that reinforces our commitment to developing innovative therapies for rare pediatric inflammatory diseases," said Goldberg. "By targeting activated macrophages with the most powerful anti-inflammatory compounds, we enhance efficacy and reduce toxicity. Critically, we have demonstrated no suppression of the HPA system, thus supplementing rather than replacing the natural anti-inflammatory response. We are eager to advance our clinical program and expand our research into other orphan diseases characterized by systemic inflammation.”¹

Related news:

Johnson & Johnson this month submitted a supplemental Biologics License Application (sBLA) for guselkumab (Tremfya) to treat active active juvenile psoriatic arthritis (jPsA) in children aged 5 years and older, as part of a dual submission.²

For the jPsA indication, the submission was based on the PK extrapolation analyses from the adult PsA studies, Discover 1 (NCT03162796) and Discover 2 (NCT03158285), while safety and efficacy data was from the Protostar study, according to Johnson & Johnson.²

Click here for more details on guselkumab and the sBLA submissions for jPsA and moderate-to-severe plaque psoriasis (PsO) in children aged 6 years and up.²

References:

1. PIF Partners Granted FDA Rare Pediatric Disease Designation for Its Proprietary Small Molecule in Treating Systemic Juvenile Idiopathic Arthritis (sJIA) Flares. PIF Partners. Press release. December 3, 2024. Accessed December 4, 2024.https://www.businesswire.com/news/home/20241120350128/en/PIF-Partners-Granted-FDA-Rare-Pediatric-Disease-Designation-for-Its-Proprietary-Small-Molecule-in-Treating-Systemic-Juvenile-Idiopathic-Arthritis-sJIA-Flares

2. Fitch, J. J&J submits applications for guselkumab to treat plaque psoriasis, jPsA in children. Contempoary Pediatrics. December 3, 2024. Accessed December 4, 2024. https://www.contemporarypediatrics.com/view/j-j-submits-applications-for-guselkumab-to-treat-plaque-psoriasis-jpsa-in-children