FDA Grants Rare Pediatric Disease Designation to PIF Partners' Small Molecule for sJIA Flares

PIF Partners announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational therapeutic 101-PGC-005 (‘005) for treating systemic juvenile idiopathic arthritis (sJIA) flares. The compound, a Type IA prodrug of dexamethasone, targets CD206+ macrophages. Currently, ‘005 is in Phase 3 clinical trials in India, focusing on treating ARDS induced by COVID-19.

According to Alec Goldberg, CEO of PIF Partners, the FDA's recognition of ‘005 is a significant milestone in addressing the unmet medical needs of children suffering from sJIA. Goldberg emphasized the compound’s ability to enhance efficacy and reduce toxicity by targeting activated macrophages with powerful anti-inflammatory agents. Importantly, the therapeutic does not suppress the hypothalamic-pituitary-adrenal (HPA) system, thereby supplementing the body's natural anti-inflammatory response. The company is eager to advance their clinical program and extend their research into other rare diseases characterized by systemic inflammation.

RPDDs are granted by the FDA to promote the development of new treatments for serious or life-threatening conditions that primarily affect children under 18 and impact fewer than 200,000 people in the U.S. This designation makes PIF Partners eligible for a Priority Review Voucher (PRV) upon approval of ‘005. A PRV can accelerate the review process for a subsequent marketing application from the standard 10 months to six months.

Systemic juvenile idiopathic arthritis (sJIA) is a severe and rare autoinflammatory disease affecting children under 16. Unlike other juvenile arthritis forms, sJIA is marked by systemic symptoms like high fevers, rash, liver and spleen enlargement, and serositis, along with persistent joint inflammation. The condition accounts for approximately 10-20% of all juvenile idiopathic arthritis cases.

Children with sJIA face significant challenges, including chronic pain, growth retardation, joint damage, and potentially life-threatening complications like macrophage activation syndrome (MAS). Current treatments are limited and often involve long-term use of corticosteroids and immunosuppressive drugs, which can have serious side effects. There is an urgent need for new therapies that effectively manage both systemic and joint-related symptoms with improved safety profiles.

PIF Partners holds exclusive rights to ‘005 and other therapeutics using their macrophage targeting platform. The company is actively seeking a partner to advance the development and commercialization of novel therapies for sJIA and other rare diseases characterized by systemic inflammation. Their goal is to address critical gaps in treatment and advance a pipeline of therapeutic candidates targeting CD206+ macrophages.