Ilya Pharma’s investigational treatment receives rare pediatric disease designation
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Key takeaways:
- Ilya Pharma’s emilimogene sigulactibac treatment received the FDA’s rare pediatric disease designation.
- The treatment is being investigated for STING-associated vasculopathy with onset in infancy.
The FDA has issued a rare pediatric disease designation to Ilya Pharma for its topical emilimogene sigulactibac treatment for STING-associated vasculopathy with onset in infancy, according to a press release.
Stimulator of interferon genes (STING)-associated vasculopathy with onset in infancy (SAVI) is a rare autoinflammatory disease caused by gain-of-function mutations in the STING1 gene. Disease onset tends to occur during neonatal or infantile stages and can present as episodes of recurrent fevers and cold-induced skin vasculitis causing rashes which often progress to loss of fingers, toes, ear and nose tissue. Interstitial lung disease may also occur.
The disease is marked by increased childhood mortality.
“Pediatric patients living with SAVI face a significant unmet medical need with limited treatment options,” Margareth Jorvid, MSc Pharm, MSc MTRA, MBA, head of regulatory affairs at Ilya Pharma, said in the release. “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.”
Emilimogene sigulactibac (ILP100-Topical) is being investigated for the treatment of skin wounds in patients with SAVI. According to the release, previous results showed that this topical accelerates wound healing, increases numbers of restorative tissue and acts as a potent antimicrobial on bacterial wound pathogens.
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