Introduction to ATMPs QP Forum Trinity College Dublin

Introduction to ATMPs QP Forum Trinity College Dublin 12 April 2018 Hugh Mc. Mahon Principal Consultant/QP WHEN YOU NEED TO MEET A HIGHER STANDARD

ADVANCED THERAPY MEDICINAL PRODUCTS - DEFINITIONS • Tissue engineered (TE) products - growth and development of biomaterials using engineering principles and techniques, especially the synthesis of body tissues and whole organs for the restoration or replacement of a damaged or diseased body part. • Gene therapy (GT) medicinal products - the treatment of certain medical disorders, especially those caused by genetic anomalies or deficiencies, by introducing specific engineered genes into a patient's cells. • Somatic Cell therapy (SCT) - where living cells are injected into patient e. g. T- cells capable of fighting cancer cells via Cell –Mediated immunity may be injected in the course of immunotherapy • Combined ATMPs contain one or more medical devices as an integral part of the medicine, such as cells embedded in a biodegradable matrix or scaffold. • FDA defines ATMPs as: Cellular therapy products (that) include cellular immunotherapies and other types of both autologous and allogeneic cells for certain therapeutic indications, including adult and embryonic stem cells. Human gene therapy refers to products that introduce genetic material into a person’s DNA to replace faulty or missing genetic material, thus treating a disease or abnormal medical condition. WHEN YOU NEED TO MEET A HIGHER STANDARD 2

GENE THERAPY – MECHANISM OF INSERTION OF FUNCTIONAL GENE WHEN YOU NEED TO MEET A HIGHER STANDARD

LUXTURNA - GENE THERAPY FOR RPE 65 RELATED VISION LOSS WHEN YOU NEED TO MEET A HIGHER STANDARD

CHIMERIC ANTIBODY RECEPTOR - CAR-T CELL THERAPY How CAR-T cells work WHEN YOU NEED TO MEET A HIGHER STANDARD

COMPOSITION OF EU ATMP ORPHAN DRUG DESIGNATIONS SINCE 2001 (185) WHEN YOU NEED TO MEET A HIGHER STANDARD

CAR-T CELL CLINICAL TRIALS OVER TIME AND GEOGRAPHICAL DISTRIBUTION Jessica Hartmann et al. EMBO Mol Med. 2017; emmm. 201607485 WHEN YOU NEED TO MEET A HIGHER STANDARD

COST OF ATMP THERAPIES AND POTENTIAL PATIENT POPULATIONS WHEN YOU NEED TO MEET A HIGHER STANDARD

MOORE’S LAW VS COST OF GENOMIC SEQUENCING COST OF ATMP THERAPIES AND POTENTIAL PATIENT POPULATIONS

TABLE OF ATMP’S APPROVED BY EMA WHEN YOU NEED TO MEET A HIGHER STANDARD

OVERVIEW OF FIRST 12 MONTHS OF EMA PRIORITY MEDICINE DESIGNATION WHEN YOU NEED TO MEET A HIGHER STANDARD

Thank you for your attention Questions? WHEN YOU NEED TO MEET A HIGHER STANDARD 12

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