The Center for Drug Evaluation (CDE) in China has announced its intention to include AstraZeneca’s (AZ, NASDAQ: AZN) ALXN1850, known as efzimfotase alfa, in the patient-centered rare disease drug development pilot program, referred to as the Care Plan. This move signifies a significant step forward in the global efforts to address rare diseases and improve patient outcomes.
Efzimfotase Alfa: A Tissue Nonspecific Alkaline Phosphatase (TNSALP) Enzyme Replacement Therapy
Efzimfotase alfa, a therapy designed to replace the tissue nonspecific alkaline phosphatase (TNSALP) enzyme, was given the green light for study in February of this year. It is intended for the treatment of hypophosphatasia (HPP), a rare and debilitating disease characterized by low levels of the TNSALP enzyme. The inclusion of efzimfotase alfa in the Care Plan underscores China’s commitment to advancing treatments for rare conditions and enhancing patient care.
Enzyme Replacement Therapy for Hypophosphatasia (HPP)
As an enzyme replacement therapy (ERT), efzimfotase alfa has the potential to significantly improve the lives of patients suffering from HPP by addressing the root cause of the disease. The decision by the CDE to include this therapy in the pilot program highlights the importance of international collaboration in the development of treatments for rare diseases and the potential for efzimfotase alfa to become a standard of care for HPP patients.-Fineline Info & Tech
