The National Health Commission (NHC) of China, through its Center for Drug Evaluation (CDE), has announced that Pfizer (NSE: PFIZER, NYSE: PFE, ETR: PFE)’s Marstaximab (PF-06741086) has been included in the “Care Plan” for patient-centered rare disease drug development. The publicity period for this decision ends on October 12, 2024.
Marstaximab is a novel therapy for hemophilia, indicated for patients aged 12 and above with hemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors or hemophilia B (congenital factor IX deficiency) without factor IX inhibitors. The treatment differentiates itself by targeting the tissue factor pathway inhibitor (TFPI) and requires only a once-weekly subcutaneous injection. This approach aims to address challenges such as the production of inhibitors and efficacy decline in previous clinical treatments. Marstaximab’s new drug application was accepted by the CDE in August 2024.
The “Care Plan” was introduced in May 2024 to guide and assist pharmaceutical companies in integrating patient experience data into the clinical development of rare disease drugs, enhancing the scientific rigor, standardization, and rationality of the process.- Flcube.com
