EC Regulation 1394/2007 established the regulatory framework for Atmps and made amendments to Directive 2001/83 EC and EC Regulation 726/2004. Directive 2009/120/EC further updated the definitions and scientific and technical requirements for these medicines. In addition, developments of Atmps must comply with Directive 2001/20/EC on good clinical practice in the testing of medicines for human use.

Research numbers

The latest Alliance Regenerative Medicines (ARM) report indicates that there are nearly 1,900 ongoing clinical trials worldwide, 98 of which are already in phase 3 (91 of these are being conducted by the biopharmaceutical industry while 7 are being conducted by academies, governments or other institutions).

Specifically, in Europe, there are 341 active clinical trials of which about 50 are in the pre-authorization phase.

Clinical trials of gene therapies for rare diseases show high efficacy. ARM estimates that orphan gene therapies entering phase 1 are 3.5 times more likely to be approved than the average drug (included in the BIO New Clinical Development Success Rates 2011-2020 report). The success rates recorded by IQVIA confirm the higher efficacy of the three gene therapies.

What’s new on the treatment front

The most recent Italian report on advanced therapies is dated 2023 and takes a snapshot of several aspects: the state of the art related to advanced therapies in the world and in Italy, the timing of their arrival in regional formularies, and health care spending, which is now 0.34 percent of the total.

Between 2022 and 2023, several Atmps were approved in the United States including the first topical gene therapy and a treatment for Duchenne disease. In addition, the world’s most expensive drug to combat hemophilia B has been introduced.

In Italy, eight advanced therapies are currently marketed and reimbursed while six are under evaluation. Fondazione Telethon has become responsible for the production and distribution of a drug against Ada-Scid congenital immunodeficiency, marking a major shift in manufacturing in the field of Atmps. In addition, the field is characterized by mergers and acquisitions between companies to combine expertise and resources, and politics has also shown interest with the establishment of a National Ethics Committee for Atmp trials and a specific Technical Table at the Ministry of Health.

Therapies approved in Europe

According to the Advanced Therapies Observatory, updated to July 2024, there are 27 advanced treatments approved by Ema so far. Among them, gene therapies account for the majority with 18 preparations that have been given the green light; followed by five cell therapies, three tissue engineering products and one genomic edit.

Notably, 17 of the 27 cleared therapies are orphan therapies, that is, intended for the treatment of a rare disease.

Four Atmps are made in Italy
>> Strimvelis (for ADA-SCID in cases of absence of a consanguineous donor),
>> Libmeldy (for metachromatic leukodystrophy),
>> Zalmoxis (for adjunctive treatment in cases of stem transplantation in counteracting high-risk hematologic malignancies),
>> Holoclar (to combat moderate to severe limbal stem cell deficiency caused by eye burns from physical or chemical agents).

The most recent advanced therapies approved in Europe are:

  • Casgevy (exagamglogene autotemcel), approved for transfusion-dependent beta-thalassemia and severe sickle cell anemia characterized by vaso-occlusive crises-February 2024;
  • Durveqtix (fidanacogene elaparvovec) orphan drug approved for severe/moderately severe hemophilia B (congenital Factor IX deficiency) – July 2024.