“Pediatric patients living with SAVI face a significant unmet medical need with limited treatment options,” says Margareth Jorvid, Head of Regulatory Affairs, Ilya Pharma. “Obtaining Rare Pediatric Disease Designation is another acknowledgement of the serious and life-threatening manifestations of this rare disease, and supports our mission to provide emilimogene sigulactibac as a potential new treatment option.”

SAVI

STING-associated vasculopathy with onset in infancy (SAVI) is caused by Gain-of-function mutations in the STING1 gene, which encodes the transmembrane protein STING (Stimulator of Interferon Genes). This rare disease belongs to a newly defined class of autoinflammatory disorders referred to as type I interferonopathies that typically presents with neonatal or infantile onset of systemic inflammation and small vessel vasculopathies.

SAVI disease is associated with marked childhood morbidity and increased mortality, and is characterized by episodes of recurrent fevers, cold-induced skin vasculitis causing rashes which often progress to loss of fingers and toes, as well as ear and nose tissue, and/or interstitial lung disease. The response to conventional immunosuppressive therapies is minimal in SAVI patients, resulting in marked childhood morbidity and increased mortality, which demonstrates an unmet clinical need, describes Ilya Pharma.

emilimogene sigulactibac

The rationale for investigating topical emilimogene sigulactibac (INN), ILP100-Topical for the treatment of skin wounds in patients with SAVI is based on the results from previous published nonclinical and clinical studies showing accelerated wound healing, increased numbers of CXCR4-expressing tissue restorative macrophages in injured tissue improve vessel function and tissue restoration, and potent antimicrobial effects on bacterial wound pathogens, especially multi drug resistant bacteria, describes Ilya Pharma.