FDA Grants Rare Pediatric Disease Designation to Small Molecule for Systemic Juvenile Idiopathic Arthritis

PIF Partners announced that FDA officials granted Rare Pediatric Disease Designation (RPDD) to its proprietary investigational therapeutic 101-PCG-005, which is being researched for the treatment of systemic juvenile idiopathic arthritis (sJIA) flares. The treatment is a type IA prodrug of dexamethasone that targets CD206+ macrophages.¹

Child sick with a fever | Image credit: Alexis Scholtz/peopleimages.com | stock.adobe.com

“By targeting activated macrophages with the most powerful anti-inflammatory compounds, we enhance efficacy and reduce toxicity,” said Alec Goldberg, CEO of PIF Partners, in a news release. “Critically, we have demonstrated no suppression of the [hypothalamic-pituitary-adrenal] 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

sJIA is a rare autoinflammatory disease that affects children younger than 16, characterized by high spiking fevers, rash, enlargement of the liver and spleen, and inflammation of the lining of the heart and lungs, as well as persistent joint inflammation. sJIA accounts for between 10% and 20% of all cases of juvenile idiopathic arthritis.¹

Sometimes called Still’s disease, sJIA most commonly starts around 2 years of age and affects boys and girls equally. It is the only subtype of JIA to be considered an autoinflammatory disease rather than autoimmune.²

The most severe potential complication of sJIA is macrophage activation syndrome (MAS), in which the body is overwhelmed by a massive inflammatory response. Known triggers for MAS include viral infections, medication changes, and flares. Although it can occur in any rheumatic disease, an estimated 80% of MAS cases are associated with sJIA.² Other health effects can include lung and heart problems, damaged bones and joints, and hypertension.²

There is currently no cure for sJIA, but the goals of treatment are to reduce symptoms and treat the underlying inflammation. Treatments include corticosteroids as well as other immunosuppressive medications. Biologic medications can also target specific inflammatory molecules, including anakinra (Kineret; Sobi), canakinumab (Ilaris; Novartis), and tocilizumab (Tofidence; Biogen).³ 

The standard approach to treating sJIA is to initiate nonsteroidal anti-inflammatory drugs and progress to stronger treatments over time, if necessary. Other steps may include high doses of oral of intravenous corticosteroids, which can rapidly relieve fevers and other systemic symptoms, as well as joint pain and swelling.²

In recent years, the FDA has approved newer medications that target specific inflammatory proteins, including interleukin (IL)-1 and IL-6, which are overactive in sJIA. Because these biologics can cause dramatic improvements in some children, some physicians are now using these are first-line therapies, avoiding corticosteroids and their associated adverse effects.²

The investigative therapeutic 101-PCG-005 is designed to reduce flares in sJIA. It is also in phase 3 clinical trials across 9 centers in India for the treatment of acute respiratory distress syndrome induced by COVID-19.¹

“We are thrilled the FDA has recognized the potential of ‘005 to address the significant unmet medical needs of children suffering from sJIA,” Goldberg said. “This designation is a pivotal milestone that reinforces our commitment to developing innovative therapies for rare pediatric inflammatory diseases.”¹

REFERENCES

1. PIF Partners Granted FDA Rare Pediatric Disease Designation for Its Proprietary Small Molecule in Treating Systemic Juvenile Idiopathic Arthritis (sJIA) Flares. News release. BusinessWire. December 3, 2024. Accessed December 3, 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. Systemic Juvenile Idiopathic Arthritis. Arthritis Foundation. Arthritis Foundation. Accessed December 3, 2024. https://www.arthritis.org/diseases/systemic-juvenile-idiopathic-arthritis

3. Taber SF. Systemic Juvenile Idiopathic Arthritis. Hospital for Special Surgery. Accessed December 3, 2024. https://www.hss.edu/conditions_systemic-juvenile-idiopathic-arthritis.asp