FDA Grants Rare Pediatric Disease Designation for Catalyst’s CB 4332 for Treatment of CFI Deficiency

The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation for Catalyst Biosciences’ CB 4332 for the treatment of Complement Factor I deficiency.

Photo: Nassim Usman, CEO of Catalyst Biosciences

Complement factor I deficiency (CFID) is a rare immunodeficiency caused by mutations in the CFI gene. This gene provides instructions for making a protein called complement factor I, which helps regulate a part of the body’s immune response known as the complement system, a group of proteins that work together to destroy foreign invaders, trigger inflammation, and remove debris from cells and tissues. This system must be carefully regulated so it targets only unwanted materials and does not attack the body’s healthy cells. Complement factor I and several related proteins protect healthy cells by preventing activation of the complement system when it is not needed.

Mutations in the CFI gene that cause complement factor I deficiency result in abnormal, nonfunctional, or absent complement factor I. The lack (deficiency) of functional complement factor I protein allows uncontrolled activation of the complement system, which decreases blood levels of another complement protein called C3, reducing the immune system’s ability to fight infections. In addition, the immune system may malfunction and attack its own tissues, resulting in autoimmune disorders. CB4332 is an engineered protease designed to increase functional complement factor I in patients with CFI deficiency.

“The granting of the Rare Pediatric Disease designation for CB 4332 underscores the significant unmet medical need in pediatric patients with CFID,” said Nassim Usman, CEO of Catalyst Biosciences. “We are focused on efficiently advancing the development of CB 4332 and our complement medicines portfolio in a number of complement-driven diseases in hematology, nephrology and ophthalmology.”

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children aged 18 years or younger and impact fewer than 200,000 people in the United States. If a New Drug Application in the United States for IMX-110 is approved, ImmixBio may be eligible to receive a Priority Review Voucher (PRV) from the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Mirum Pharmaceuticals sold its voucher for $110 million.

Author: Rare Daily Staff