Catalyst Biosciences Inc. (NASDAQ: CBIO) Receives Rare Paediatric Diseases Designation for CB 4332 in CFID Treatment 

Catalyst Biosciences Inc. (NASDAQ: CBIO) has announced that the FDA has granted CB 4332 a Rare Paediatric Disease Designation (RPDD) for Complement Factor 1 deficiency (CFID) treatment.

CB 4332 granted RPDD

CEO of the company Nassim Usman,” The granting of the Rare Pediatric Disease Designation for CB 4332 underscores the significant unmet medical need in pediatric patients with CFID. 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.”

Usman stated that they are delighted with the FDA’s evaluation of CB 4332 as a possible treatment for an underserved pediatric group of CFID patients and the rare pediatric disease designation grant.

The FDA may provide a priority review coupon to a company that achieves a product clearance for a rare pediatric ailment via the FDA’s RPDD  program. A rare pediatric disease is described as a severe or life-threatening ailment that affects fewer than 200,000 people in the United States each year, most of whom are under the age of 18.

Catalyst halts MarzAA development 

Recently the company announced a clinical development hold of MarzAA and planned to look for a buyer for its hemophilia assets. The company plans to repurpose its resources on the complement therapeutics and protease treatments platform.

Usman stated, “We have made a strategic decision to stop the clinical development of MarzAA (engineered FVIIa) and focus solely on our complement programs and protease medicines platform. Based on several factors including a recently updated feasibility assessment, we determined that we cannot continue to develop MarzAA through completion of the ongoing trials.”

Several issues, including COVID-19-related logistical problems, patient competitiveness, and increased availability of prophylactic medicine globally, have hampered recruitment in the MarzAA clinical studies. Due to these circumstances, the company will no longer be able to supply topline results in 2022. However, it will discuss the results of the Crimson-1 study so far, which demonstrate that it has successfully treated bleeding with subcutaneous (SQ) MarzAA, and there haven’t been significant treatment-related negative or thrombotic events.