ATMP

Origin

This regulatory term was first formalized within the European Union to classify and streamline the approval of these highly complex, living-material-based therapies that fall outside the traditional definitions of drugs or devices. The scientific foundation is deeply rooted in advancements in molecular biology and regenerative medicine, particularly the understanding of cellular signaling and differentiation. The specialized classification was necessary due to the unique risk profile and the inherent manufacturing challenges associated with biological starting materials.

Mechanism

ATMPs function by introducing genetic material, functional cells, or engineered tissues directly into the patient’s physiological system to correct a molecular or cellular defect underlying an endocrine pathology. This targeted intervention aims to restore homeostatic hormonal balance rather than merely managing symptoms. The mechanism often involves the long-term expression of a missing hormone or the functional replacement of a non-secreting gland, offering a potential cure for certain endocrine deficiencies.