Advanced therapy medicines

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Drugs for novel therapies (also ATMPs for Advanced Therapy Medicinal Products ) is the umbrella term for three drug product classes, the somatic cell therapeutics , the gene therapeutics and the biotechnologically processed tissue preparations [also tissue engineering products (TEP) ]. These drugs contain or consist mostly of living cells or tissue and are therefore characterized by a high level of complexity. The cells used are often taken from a patient, processed in the laboratory (for example, reproduced or genetically modified), and then given back to the same patient. Because of this, ATMPs are often an example of “ personalized medicine ”.

definition

The classification of an ATMP into one of the three product classes is determined by the legal and regulatory requirements that apply to the ATMP. The following properties of the drug determine the classification in the respective product class:

Somatic cell therapeutic agent

  • It has been substantially tampered with so that biological characteristics, physiological functions or structural properties have been changed for the intended clinical application.
  • The cells or tissues contained do not fulfill the same essential functions in the recipient as in the donor (so-called non-homologous use).

Gene therapeutic

  • It contains an active substance, consisting of or containing a recombinant nucleic acid , with the aim of adding, regulating, repairing, replacing or removing a genetic sequence in a cell.
  • The therapeutic, prophylactic or diagnostic effect can be traced back directly to the recombinant nucleic acid.

Exception: Medicines that fulfill both properties but are intended to be used as a vaccine against infectious diseases are not classified as gene therapeutic agents.

Tissue engineering products

  • It contains cells or tissues used to regenerate, repair, or replace human tissue.
  • It can contain living or dead cells and tissues of both human and animal origin in the presence or absence of other substances. Dead cells that have no pharmacological, immunological or metabolic mode of action are not tissue engineering products.
  • The cells or tissues contained do not fulfill the same function in the recipient as in the donor (non-homologous use).

Development and characteristics of the marketing authorization

The development of ATMPs is mostly characterized by a strongly experimental character and often has its origin in academic research environments such as universities and their clinics. This and the individual production in often small numbers for the treatment of the most severe, but also less common, diseases has so far meant that the classic (financially strong) pharmaceutical industry is almost not represented in this pharmaceutical sector. As a result, therapy concepts cannot reach market maturity due to a lack of financial support for the implementation of clinical studies as well as limited regulatory experience (e.g. compliance with legal requirements for drug approval ). Unlike most conventional drugs in individual countries within the European Union can be admitted (EU), ATMPs have EU-wide ( centralized ) on the European Medicines Agency - (EMA European Medicines Agency are allowed).

In German-speaking countries, the national authorities are responsible for approving clinical studies with ATMPs:

Examples of therapy concepts

The following concepts have been classified by the EMA, among others:

Somatic cell therapeutics

Gene therapeutics

Tissue engineering products

  • Cartilage-forming cells ( chondrocytes ) are removed, propagated in vitro and administered to those joints for the treatment of symptomatic cartilage defects.
  • Keratinocytes from the skin are removed, multiplied and retransplanted to a patient with burned skin .
  • Cells of the cornea of the eye that contain stem cells are multiplied and retransplanted if the cornea is damaged.
  • Skeletal muscle- derived cells are multiplied and used to treat urinary incontinence .
  • Oral mucous membrane cells are removed, multiplied in vitro and placed on an absorbable carrier membrane . The support membrane with the oral mucous membrane cells is sewn into the urethra to widen the urethra in the case of urethral strictures.

Web links

Individual evidence

  1. ↑ Law on the circulation of medicinal products (Medicines Act - AMG), Section 4, Paragraph 9 .
  2. ↑ Directive 2009/120 / EC of the Commission of September 14, 2009 amending Directive 2001/83 / EC of the European Parliament and of the Council on the creation of a Community code for medicinal products for human use with regard to advanced therapy medicinal products.
  3. ↑ Regulation (EC) No. 726/2004 of the European Parliament and of the Council of March 31, 2004 laying down community procedures for the approval and monitoring of medicinal products for human and veterinary use and establishing a European Medicines Agency (PDF) .
  4. ↑ Summary of ATMP classifications by the EMA .