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ATMPs are medicines which could offer new treatments for rare diseases.​

Advanced Therapy Medicinal Products (ATMPs)

What are ATMPs?​

Advanced Therapy Medicinal Products (ATMPs) are innovative medicines that create new treatment options fordiseases that are difficult, or sometimes impossible, to treat with traditional drugs, especially rare conditions.

Unlike conventional medicines, ATMPs work by using a patient’s own genes, cells, or tissues to repair damage orfight disease, allowing for highly targeted and personalized therapies. In simple terms, they aim to fix the problem atits source, rather than just relieving symptoms.

As with allauthorised medicines, theEuropean Medicines Agency (EMA)continuouslymonitorsthe safety and effectiveness of ATMPs once they have been approved and placed onthe market.

Types of ATMPs

There are three main types of ATMPs, based onhow they work:​

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Gene Therapy Medicinal Products (GTMP)

These treatments use genes to prevent or treat disease. They work by delivering new or modified genetic material into a patient’s cells, enabling the body to produce something it is missing, such as a protein required for normal function. For example, gene therapy can be used to treat rare inherited eye conditions, such as RPE65 deficiency, helping some patients to regain vision.
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Cell Therapy Medicinal Products (CTMP)​​

These therapies involve administering living cells to patients to help repair or replace damaged tissue. The cells are processed or modified in a laboratory so that they act in a specific way. They may be taken from the patient themselves (autologous) or from a donor (allogeneic). Some stem cell therapies fall into this category, but only when the cells have been substantially altered or used for a new purpose. For example, some cancer treatments use a patient’s own immune cells, modified in the lab, to better recognize and attack cancer cells.
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Tissue Engineered Products (TEP)​​​

These treatments aim to rebuild or replace damaged tissues. They combine cells with supporting materials, known as scaffolds, to create new tissue. This approach may one day help to repair a wide range of tissues, including cartilage, or even whole organs. For example, some treatments are already used to support the healing of severe burns.
ATMPs can sometimes include a medical device as an integral part of the treatment. These are known as combined ATMPs, for example when cells are embedded in a biodegradable scaffold used for tissue engineering.

Challenges in ATMPs development​

Although the potential of ATMPs is immense, several challenges must be addressed before these therapies canbecome widely available.​​

High Costs​

The production of ATMPs is both complex and expensive. The personalised nature of these therapies, particularly in cell therapy, makes them costly to manufacture. As a result, the price for patients can be extremely high. Government and healthcare systems need to find ways to ensure that these therapies are affordable and accessible to those who need them most.

Regulatory Obstacles​

The regulatory framework for ATMPs is still evolving due to their complexity and novelty. In the EU, the European Medicines Agency (EMA) plays a key role in ensuring these therapies meet strict standards of quality, safety, and efficacy before approval.​ Although the process can be complex and time-consuming, this rigorous evaluation helps ensure that innovative treatments are safe for patients.​

Manufacturing Challenges​

The production of ATMPs requires highly specialized facilities and advanced techniques. Expanding manufacturing to meet global patient demand will require significant investment in infrastructure. Currently, production sites are concentrated in only a few countries, highlighting the gaps in available manufacturing capacity.​

Disclaimer​

Like any medical treatment, ATMPs must be properly tested and approved. Unregulated or unapproved ATMPs can be dangerous, as they offer no proven benefit and may cause serious harm. Patients should always consult their national health authority before considering such treatments.

Full list of EU National Competent Authorities available here.

Sources: EMAAdvanced Therapy MedicinalProducts Overview; EATRIS ATMPPlatform; AEMPSAdvanced therapies;Clinical applications of genetherapy for rarediseases: Areview;​

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