The European Medicines Agency (EMA) has issued a warning about the dangers of unregulated advanced therapy medicinal products (ATMPs) being illegally offered to patients in Europe.
ATMPs encompass a wide range of treatments including those that are based on cells, genes, or tissues. Advanced therapies have become a promising option in recent years to treat rare diseases and certain types of cancer.
Whilst the EMA has approved multiple products for patient use, the agency said that individuals, companies, and clinics are also marketing unregulated ATMPs. There is little to no evidence these unregulated products work and can cause serious side effects, putting patients at risk. The EMA said that sellers exploit patient distress, selling treatments on websites or social media channels “as a last hope.”
The EMA pointed to quality-related risks in the manufacturing process for unregulated ATMPs, which can lead to contamination, inconsistent product composition, and improper storage.
A particular modality highlighted by the agency was dendritic cell therapies, a form of immunotherapy that has shown some promise in treating cancers. The therapy uses the patients’ own dendritic cells to initiate an immune response and attack cancer cells. Dendreon Pharmaceuticals used to make the only EMA-approved dendritic cell therapy called Provenge (sipuleucel-T), which received marketing authorisation in 2013. However, approval was withdrawn in May 2015 by Dendreon citing its poor commercial performance. The vaccine is still available in the US.
“It is important that patients are only offered ATMPs, including dendritic cell therapies, that have been authorised or approved by a regulator,” the EMA said in a statement.
“Authorities across the EU are working together to clamp down on those supplying unregulated ATMPs. The public are requested to report any suspicious cases to their national competent authorities,” the agency added.
Murky landscape for dendritic cell therapies
For ATMPs, there exists a grey zone between EMA-approved products and therapies regulated at the national level. There are various clinics in Germany that offer dendritic cell therapy, for example.
Booking Health, a German medical tourism operator, connects cancer patients with clinics that charge up to €38,000 for the treatment, though costs can increase depending on further lab tests and required medication. On the company’s website, the company lists its laboratory being regulated by the EMEA, the Paul Ehrlich Institute, and local regional councils, but does not mention therapy-specific regulatory approvals. The EMEA and other drug regulatory bodies like the US Food and Drug Administration provide approvals or clearances to laboratories and ATMPs through different pathways, each with its own set of data requirements.
IOZK, an immunological institution based in Cologne, Germany, claims it is the only laboratory in Europe to have received a manufacturing license to produce an individual vaccine – called IO-VAC – to combat cancer using dendritic cells. The EMA does not oversee IO-VAC’s use, though states that ATMPs can be given as part of a special exemption or clinical trial that is authorised at the national level. Germany is able to approve its own ATMPs through national regulatory pathways.
IOZK says on its website: “We can already make this available to tumour patients today – even if it is not yet a standard of care.”
The warning by the EMA is not the only instance in recent times that a health agency has had to clamp down on unregulated medicinal products. The World Health Organization (WHO) issued an alert in June last year relating to falsified batches of popular type 2 diabetes drug Ozempic (semaglutide). The agency said the unregulated products could have harmful effects on patient health.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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