The US FDA has granted Rare Paediatric Disease (RPD) designation for Satellite Bio’s SB-101 for the treatment of urea cycle disorders (UCDs).,
Satellite Bio is a biotechnology company developing off-the-shelf liver therapies designed to restore liver function in patients with severe liver diseases.
UCDs are devastating, life-threatening conditions with high infant mortality and significant risk of neurological injury, with no curative treatment available in the first weeks of life.
UCDs primarily affect individuals aged from birth to 18 years of age, with serious or life-threatening manifestations such as neurocognitive deficits, behavioural impairment, encephalopathy, coma, seizures, organ failure and mortality.
The current standard of care for UCDs remains inadequate, with mortality rates exceeding 25%, cognitive impairment affecting half of the patients and no curative treatment available in the first weeks of life.
“This designation marks an important milestone for Satellite Bio and reinforces the potential of our approach to restore essential liver function for patients with severe liver diseases,” said Tom Lowery, President and CEO of Satellite Bio. “UCDs are some of the most devastating diseases affecting infants with no curative treatments available in the first weeks of life. We look forward to advancing SB-101 in clinical trials this year and expanding treatment options for individuals with UCDs.”
Satellite Bio’s lead programme, SB-101, is a first-in-class, off-the-shelf liver therapy being developed as a treatment for infants with severe early onset UCD. The company plans to initiate a phase 1/2 clinical trial of SB-101 in 2026. Satellite Bio’s pipeline also includes additional drug candidates designed to improve liver function in other paediatric liver diseases and adult patients with chronic liver disease.
The liver performs more than 500 vital functions, including detoxifying harmful substances, processing nutrients and producing essential blood proteins. Severe liver diseases occur when hepatocytes, the liver’s functional cells, can no longer perform these critical activities.
RPD designation is granted to drug candidates intended to treat serious or life-threatening diseases that primarily affect children from birth to 18 years of age.