The US Food and Drug Administration (FDA) has granted Orphan Drug Designation in the US to Cantargia’s CAN10 for the treatment of systemic sclerosis. This designation provides a range of incentives in the continued clinical development of CAN10 in systemic sclerosis.
“The Orphan Drug Designation by the FDA is another crucial milestone in the clinical development of CAN10, highlighting the urgent need for new treatment options for systemic sclerosis patients, as well as the potential of CAN10. We look forward to advancing the program and presenting initial data from the ongoing phase I safety trial for CAN10 in 2024,” said Göran Forsberg, CEO of Cantargia.
The FDA Office of Orphan Products Development provides orphan status to drugs and biologics which are intended to treat, diagnose, or prevent rare diseases that affect fewer than 200,000 people in the US. Orphan Drug Designation qualifies sponsors for incentives, including tax credits for qualified clinical trials, exemption from user fees, and potential seven years of market exclusivity after approval.
Systemic sclerosis is a life-threatening autoimmune disease characterized by inflammation and subsequent fibrosis, i.e. uncontrolled scar tissue formation, in skin and various internal organs. The medical need for systemic sclerosis is high, with few approved drugs currently available.
The CAN10 antibody strongly binds IL1RAP and simultaneously blocks the function of the signaling molecules IL-1, IL-33 and IL-36, which play key roles in several autoimmune and inflammatory diseases. CAN10 has previously shown promising effects in several models of such diseases, including the lead indications systemic sclerosis and myocarditis. A clinical phase I trial investigating the safety and tolerability of CAN10 in healthy volunteers and psoriasis patients is ongoing. Up to 80 subjects may be included in the trial, and initial data from the trial are expected in 2024.
