FDA grants orphan drug designation for rare pediatric skeletal disease therapy
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Key takeaways:
- The FDA granted orphan drug designation and rare pediatric disease designation for a treatment for autosomal dominant osteopetrosis type 2.
- If approved, the therapy would be the first treatment for the disease.
The FDA granted orphan drug designation and rare pediatric disease designation for a therapy to treat a rare pediatric skeletal disorder, according to an industry press release.
SIS-101-ADO (SiSaf Ltd.) is a siRNA therapeutic for youths with autosomal dominant osteopetrosis type 2. The therapy uses siRNA that suppresses the expression of CLCN7, a mutant gene that is responsible for causing autosomal dominant osteopetrosis type 2. The siRNA is combined with SiSaf’s Bio-Courier delivery technology to create an RNA therapy that restores bone mass and quality to near normal levels, according to the release.
SIS-101-ADO received rare pediatric disease designation based on the serious or life-threatening manifestations of autosomal dominant osteopetrosis type 2 that primarily affect children, including blindness from optic nerve compression, anomalies in dental and craniofacial development, and scoliosis.
There are no currently approved treatments for autosomal dominant osteopetrosis type 2, and no other treatments are currently in clinical trials. If approved, the agent would be the first treatment for the disease, according to the release.
“Being granted orphan drug designation and rare pediatric disease designation is a major milestone in our drive to move our revolutionary siRNA treatment forward to alleviate the pain and suffering that autosomal dominant osteopetrosis type 2 inflicts,” Suzanne Saffie-Siebert, PhD, founder and CEO of SiSaf Ltd., said in a press release. “SIS-101-ADO ushers in the potential for a new era of personalized care and treatment options for autosomal dominant osteopetrosis type 2 and other rare bone and skeletal diseases.”
According to the press release, SiSaf Ltd. is currently preparing for first-in-human clinical trials with SIS-101-ADO.
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