Two Decades of Global Progress in Authorized Advanced Therapy Medicinal Products: An Emerging Revolution in Therapeutic Strategies

Abstract

The introduction of advanced therapy medicinal products (ATMPs) to the global pharma market has been revolutionizing the pharmaceutical industry and has opened new routes for treating various types of cancers and incurable diseases. In the past two decades, a noticeable part of clinical practices has been devoting progressively to these products. The first step to develop such an ATMP product is to be familiar with other approved products to obtain a general view about this industry trend. The present paper depicts an overall perspective of approved ATMPs in different countries, while reflecting the degree of their success in a clinical point of view and highlighting their main safety issues and also related market size as a whole. In this regard, published articles regarding safety, efficacy, and market size of approved ATMPs were reviewed using the search engines PubMed, Scopus, and Google Scholar. For some products which the related papers were not available, data on the relevant company website were referenced. In this descriptive study, we have introduced and classified approved cell, gene, and tissue engineering-based products by different regulatory agencies, along with their characteristics, manufacturer, indication, approval date, related regulatory agency, dosage, product description, price and published data about their safety and efficacy. In addition, to gain insights about the commercial situation of each product, we have gathered accessible sale reports and market size information that pertain to some of these products.

Keywords: ATMP; advanced therapy medicinal products; efficacy; market size; safety.

FIGURE 1

Number of authorized ATMPs worldwide, according to country. Each country has been determined by specific color, and based on the number of authorized ATMPs. United States, South Korea, and the European Union have the highest number of approved ATMPs.

FIGURE 2

The percent of approved cell, gene, and tissue engineering products, along with their related indications and product type (autologous or allogeneic). (A) The CTMPs with 34 members constitute the largest class of ATMPs. Most of these products have an autologous source. Hematologic disorders, along with skin and soft tissue related disorders and orthopedic disorders constitute the main fields. (B) The TEPs, with 20 products, represent the next largest class of ATMPs. The most emphasized indications in this class are skin and soft tissue related disorders, orthopedic disorders, and cardiovascular diseases, respectively, and the percentage of allogeneic products are more than autologous products. (C) GTMPs with 10 products comprise the last class. The first common indications are oncology, hematologic disorders, and cardiovascular diseases, respectively. Similar to CTMPs, GTMP members mostly have autologous sources.

FIGURE 3

The annual trend of approval for CTMPs, GTMPs, and TEPs worldwide. Most of the products were recently approved. (A) In terms of CTMPs, despite a decrease in the number of approved products in 2013 and 2014, a second increased trend can be observed with five products approved in 2017. (B) For TEPs, the year 2007 with five approved products showed an increase in the total trend; however, there was a subsequent decrease in the following years. (C) There is a noticeable growth trend with respect to gene therapy medicinal products (GTMPs).