Axicabtagene ciloleucel (Yescarta)

Used for aggressive diffuse large B-cell lymphoma, which is a serious cancer of the immune system’s lymph cells. Used when the disease quickly returns or does not respond well enough to previous treatment. Subtype: CAR-T Main type: Gene therapy Source: Danish Medicines Council, 2nd line + 3rd line Source: EMA

Lisocabtagene maraleucel (Breyanzi)

Used for aggressive diffuse large B-cell lymphoma (DLBCL) when the disease returns or does not respond to previous treatments. The treatment directs the patient’s own immune cells against the cancer cells. Subtype: CAR-T Main type: Gene therapy Source: Danish Medicines Council, 2nd line, 3rd line Source: EMA

Tisagenlecleucel (Kymriah)

Used for B-cell acute lymphoblastic leukemia, a serious blood cancer—especially in children and young people, where the disease does not respond to treatment or has returned. Subtype: CAR-T Main type: Gene therapy Source: Danish Medicines Council, Tisagenlecleucel (Kymriah) Source: EMA

Ciltacabtagene autoleucel (Carvykti)

Used for multiple myeloma that has returned or no longer responds to previous treatments (especially lenalidomide). Multiple myeloma affects plasma cells in the bone marrow and can cause anemia, pain, and risk of infection. Subtype: CAR-T Main type: Gene therapy Source: Danish Medicines Council: Later line & 2nd line or later Source: EMA

Exagamglogene autotemcel (Casgevy)

Used for transfusion-dependent beta-thalassemia, an inherited blood disorder in which the body does not produce enough normal hemoglobin, and sickle cell anemia, in which red blood cells become rigid and can block small blood vessels. This can cause severe pain crises, anemia, and organ damage. Both patient groups often need regular blood transfusions throughout their lives. Main type: Gene therapy Subtype: ex vivo gene editing Source: Danish Medicines Council, sickle cell disease Source: Danish Medicines Council, beta-thalassemia Source: EMA

Etranacogene dezaparvovec (Hemgenix)

Used for hemophilia B, an inherited bleeding disorder in which the body lacks factor IX. Patients may experience spontaneous or prolonged bleeding and often need preventive factor treatment. Main type: Gene therapy Subtype: in-vivo Source: Danish Medicines Council, hemophilia B Source: EMA

Onasemnogene abeparvovec (Zolgensma)

Used for spinal muscular atrophy in infants, a serious inherited muscle-wasting disease. The disease weakens the muscles and can affect breathing and movement early in life. Main type: Gene therapy Source: Danish Medicines Council, spinal muscular atrophy Source: EMA

Voretigene neparvovec (Luxturna)

Used for inherited vision loss due to RPE65-related retinal disease. The disease causes progressively worsening vision and can lead to severe vision loss or blindness. Main type: Gene therapy Source: Danish Medicines Council, retinal dystrophy Source: EMA

Limbal stem cells (Holoclar)

(partially recommended) Used for severe limbal stem cell deficiency after chemical or physical eye injuries. The condition means that the surface of the eye cannot heal normally and can cause severe visual impairment. Main type: Tissue-engineered product Source: Danish Medicines Council, limbal stem cell deficiency Source: EMA

CCIT (Center for Cancer Immune Therapy, Herlev Hospital)

CCIT has a strong national position and an increasingly visible European profile within advanced cancer immunotherapies. The center is characterized by an integrated structure with laboratory research, GMP-compliant cell production, and early clinical testing, which makes it possible to rapidly translate knowledge between research and patient care and supports the development and clinical translation of next-generation cell therapies, including T cell-based treatments.

Rigshospitalet is Denmark’s leading tertiary hospital and provides advanced treatment and research to a large target group for advanced therapies, with more than 900 patients treated across 13 departments and 8 specialties. The hospital has solid experience with early clinical trials and clinical implementation of ATMPs and serves both as sponsor and as a trial site for a range of academic and industry-initiated studies. Research areas include, among others, CAR T-cell therapy in hematology and pediatrics, gene therapy for neurological diseases, and mesenchymal stem cell-based approaches to cardiac regeneration.

Aalborg University Hospital

Aalborg University Hospital has established a clinical trials unit that supports both commercial and non-commercial studies and offers GCP monitoring for non-commercial studies. The unit is part of Aalborg Hospital Science and Innovation Center and provides researchers with a single point of entry for guidance and support for preclinical trials, clinical trials, and innovation. Preclinical research includes, among other things, diagnostics and monitoring in immunotherapy using modern techniques in gene editing, sequencing, and PCR.

Phase IV units (University of Copenhagen/Bispebjerg Hospital, University of Southern Denmark, and Aarhus University)

University of Copenhagen (Bispebjerg Hospital), University of Southern Denmark, and Aarhus University have dedicated Phase IV units. Denmark is internationally strong in registry-based drug research with documented expertise in pharmacoepidemiology and post-authorization studies for both EMA and FDA.

Novo Nordisk Foundation Center for Stem Cell Medicine (reNEW) (University of Copenhagen)

reNEW was established with a grant of more than €300 million from the Novo Nordisk Foundation and focuses on stem cell medicine and regenerative therapies, including the social and ethical aspects of ATMP treatments.

CITCO (Centre for Cellular ImmunoTherapy of Haematological Cancer, Odense University Hospital, OUH)

CITCO is among the leading environments in chimeric antigen receptor therapy (CAR) and T cell engineering, with a focus on in vivo delivery of therapeutic products.

Odense University Hospital (OUH)

OUH is one of Denmark’s largest trial sites for CAR T-cell therapy and stem cell therapy, with several ongoing protocols, both commercial and the hospital’s own ATMPs. The hospital has access to large cleanroom facilities for clinical production of CAR T and is also planning a vector laboratory. OUH and the University of Southern Denmark are involved in several local and national partnerships with Danish and international medical and pharma companies, including AstraZeneca (Gothenburg), AlphaLyse (Odense), and Samplix (Birkerød), as well as the innovation network Life Science Fyn.

CeBIL (Center for Advanced Studies in Bioscience Innovation Law, University of Copenhagen)

CeBIL is an interdisciplinary research center focused on regulation, incentives, and policies for ATMPs as well as related areas such as medicines for rare diseases, personalized medicine, production of biological medicines, health data, and technology transfer.

CGCT (Center for Gene and Cellular Therapy, Aarhus University, AU)

CGCT focuses on regenerative medicine and immunomodulation with mesenchymal stem cells (MSC). The center has ongoing clinical ATMP trials in three indications and two additional ones under approval – primarily early-phase trials and investigator-initiated studies – as well as preclinical programs in gene editing of hematopoietic stem cells.

Aarhus University Hospital (AUH)

AUH is strongly engaged in the development and translation of advanced therapies, rooted in the close collaboration between CGCT and Aarhus University. AUH conducts several investigator-initiated ATMP trials, including CRISPR-based therapies and the development of next-generation CAR T, and contributes internationally through participation in, among others, the European University Hospital Alliance and Join4ATMP.

Advanced Therapy Medicinal Products

ATMP stands for Advanced Therapy Medicinal Products. In Danish, we often refer to them as advanced therapies. They are medicines based on genes, cells, or tissues.

What are ATMPs?

When we become ill, it is sometimes because something very fundamental in the body is not working as it should. It can be in our genes, cells or tissues. ATMPs are a new type of medicine that starts right there. In the body’s own building blocks.

While many medicines help the body by relieving symptoms or slowing a disease, ATMPs can in some cases work more directly on what is wrong. For example, they can be used to change, repair or replace functions in the body that have been lost or are not working normally.

That is why ATMPs are especially relevant for diseases where there are no good treatment options. This can include certain cancers, rare inherited diseases or diseases where cells or tissues have been damaged.

Three types

There are three main types of ATMPs, which are based on genes, cells or tissue. What they have in common is that they use biological material to treat disease.

GTMP

Gene therapy

Treatment that works by adding, changing, or regulating the body’s genetic material.

TEP

Tissue therapy

Treatment in which cells or tissue are used to repair, replace, or rebuild damaged tissue.

sCTMP

Cell therapy

Treatment in which living cells are a central part of the medicine. The cells can come from the patient or from a donor.

A term from the EU

New advanced therapies required common rules in the EU.

Advanced Therapy Medicinal Products (ATMP) is the EU’s common term for advanced biological medicinal products based on genes, cells, or tissues.

The term was introduced in EU legislation (Regulation (EC) No 1394/2007) because these therapies did not fit into the existing rules for traditional medicines. Therefore, a common European framework was created that both protects patients and makes it possible to develop and use these new treatments.

Visit EMA's website to learn more

Denmark is strongly positioned in the ATMP area

Denmark has a strong foundation for the development and implementation of advanced therapies. We combine highly specialized clinical environments, unique health data, and a well-functioning life science sector. This creates good conditions for bringing new treatments from research to patients.

Strong professional environments
Denmark has a highly specialized healthcare system and strong professional environments at hospitals and universities that are active in international collaborations and, in several areas, leading within Europe. This means that new advanced treatments can be developed and tested close to the patients who can benefit from them.
Data and follow-up
Denmark has solid experience with clinical trials and research carried out close to everyday patient care. At the same time, our health registries and CPR system make it possible to follow patients over the long term and document whether the treatments work and are safe.
Collaboration across sectors
Denmark has a strong life science sector with experience in developing, producing, and implementing ATMPs. At the same time, we have a long tradition of collaboration between hospitals, researchers, authorities, and companies. This provides good frameworks for developing, producing, and putting ATMPs into use.

From European approval to treatment in Denmark

Before a new medicine can be used in Denmark, it must first be approved at the European level. This takes place at the European Medicines Agency (EMA), which reviews all documentation on the medicine’s quality, efficacy, and safety.

If the EMA gives the medicine a positive assessment, the European Commission grants official authorization to market the medicine. This means that, in principle, it can be used in all EU countries.

In Denmark, it is the Danish Medicines Council that prepares recommendations on which new medicines should be possible standard treatments in Danish hospitals. The Council assesses whether there is a reasonable balance between the clinical value of the medicine and the costs of treatment with the medicine.

Health in Denmark

The Danish healthcare system

The Danish healthcare system is responsible for ensuring good, fast, and safe treatment for all citizens - with high quality and equal access.

Every year, 9 out of 10 Danes come into contact with the healthcare system. This may be with their general practitioner, at the hospital, with a specialist, or at the dentist.

Denmark spends around DKK 150 billion annually on the healthcare system. Most of it is funded through taxes. This means that, as a citizen, you generally do not pay out of pocket to see a doctor or receive treatment at the hospital.

The healthcare system is divided among the state, the regions, and the municipalities. The state sets the rules and has the overall responsibility. The regions operate the hospitals and are responsible for specialized treatment. The municipalities are responsible for local health services such as prevention, care, and rehabilitation.

Source: Ministry of Health and Ecclesiastical Affairs of Denmark

Learn more about the Danish healthcare system

Recommendation

Recommended ATMPs in Denmark

Here you will find an overview of the ATMPs that the Danish Medicines Council has recommended in Denmark.

Axicabtagene ciloleucel (Yescarta)
Used for aggressive diffuse large B-cell lymphoma, which is a serious cancer of the immune system’s lymph cells. Used when the disease quickly returns or does not respond well enough to previous treatment.
Subtype: CAR-T
Main type: Gene therapy

Source: Danish Medicines Council, 2nd line + 3rd line
Source: EMA
Lisocabtagene maraleucel (Breyanzi)
Used for aggressive diffuse large B-cell lymphoma (DLBCL) when the disease returns or does not respond to previous treatments. The treatment directs the patient’s own immune cells against the cancer cells.
Subtype: CAR-T
Main type: Gene therapy

Source: Danish Medicines Council, 2nd line, 3rd line
Source: EMA
Tisagenlecleucel (Kymriah)
Used for B-cell acute lymphoblastic leukemia, a serious blood cancer—especially in children and young people, where the disease does not respond to treatment or has returned.
Subtype: CAR-T
Main type: Gene therapy
Source: Danish Medicines Council, Tisagenlecleucel (Kymriah)

Source: EMA
Ciltacabtagene autoleucel (Carvykti)
Used for multiple myeloma that has returned or no longer responds to previous treatments (especially lenalidomide). Multiple myeloma affects plasma cells in the bone marrow and can cause anemia, pain, and risk of infection.
Subtype: CAR-T
Main type: Gene therapy
Source: Danish Medicines Council: Later line & 2nd line or later
Source: EMA
Exagamglogene autotemcel (Casgevy)
Used for transfusion-dependent beta-thalassemia, an inherited blood disorder in which the body does not produce enough normal hemoglobin, and sickle cell anemia, in which red blood cells become rigid and can block small blood vessels. This can cause severe pain crises, anemia, and organ damage.
Both patient groups often need regular blood transfusions throughout their lives.
Main type: Gene therapy
Subtype: ex vivo gene editing
Source: Danish Medicines Council, sickle cell disease
Source: Danish Medicines Council, beta-thalassemia
Source: EMA
Etranacogene dezaparvovec (Hemgenix)
Used for hemophilia B, an inherited bleeding disorder in which the body lacks factor IX. Patients may experience spontaneous or prolonged bleeding and often need preventive factor treatment.
Main type: Gene therapy
Subtype: in-vivo
Source: Danish Medicines Council, hemophilia B
Source: EMA
Onasemnogene abeparvovec (Zolgensma)
Used for spinal muscular atrophy in infants, a serious inherited muscle-wasting disease. The disease weakens the muscles and can affect breathing and movement early in life.

Main type: Gene therapy

Source: Danish Medicines Council, spinal muscular atrophy
Source: EMA
Voretigene neparvovec (Luxturna)
Used for inherited vision loss due to RPE65-related retinal disease. The disease causes progressively worsening vision and can lead to severe vision loss or blindness.
Main type: Gene therapy

Source: Danish Medicines Council, retinal dystrophy
Source: EMA
Limbal stem cells (Holoclar)
(partially recommended)

Used for severe limbal stem cell deficiency after chemical or physical eye injuries. The condition means that the surface of the eye cannot heal normally and can cause severe visual impairment.
Main type: Tissue-engineered product
Source: Danish Medicines Council, limbal stem cell deficiency
Source: EMA

A look into the future - ATMPs on the way

These ATMPs are currently being evaluated by EMA.

Adstiladrin	Gene therapy
What disease?	How does it work?
Bladder cancer (non-muscle-invasive) with carcinoma in situ. A type of bladder cancer in which the cancer cells are located in the lining of the bladder and have not grown into the muscle of the bladder wall. It is usually treated with BCG instillation (a tuberculosis vaccine derivative), but in some patients BCG no longer works. These patients have faced the choice between removal of the entire bladder or having few other options.	A harmless virus is used as a "delivery vehicle" to deliver a gene into the mucosal cells of the bladder. The gene causes the cells to produce an immune-boosting substance (interferon) that attacks the cancer from within.
Who can get it?	How far along is the process?
Adults with bladder cancer where BCG no longer works. Treatment: Instilled into the bladder via a catheter - once every three months.	EMA's Committee for Medicinal Products for Human Use (CHMP) has recommended that the medicine be approved. The European Commission usually makes the final decision within 2-3 months after the recommendation.
Itvisma	Gene therapy
What disease?	How does it work?
Spinal muscular atrophy (SMA) A rare, inherited disease in which the nerve cells that control the muscles slowly die. This leads to increasing muscle weakness and, in more severe cases, problems with breathing and swallowing. SMA is caused by a defect in the SMN1 gene. The disease has so far been one of the most debilitating neuromuscular diseases in children.	A harmless virus (AAV9) is used as a "delivery vehicle" to deliver a healthy copy of a missing or defective gene (SMN1) directly into the nerve cells. The cells can thereby produce the protein they lack. The treatment is given only once.
Who can get it?	How far along is the process?
Children from 2 years, adolescents and adults with SMA The gene therapy Zolgensma had previously only been approved for children under 2 years. Treatment: Injection into the spinal canal – once for life.	EMA's Committee for Medicinal Products for Human Use (CHMP) has recommended that the medicine be approved. The European Commission usually makes the final decision within 2-3 months after the recommendation.

Zopapogene imadenovec	Gene therapy (immunotherapy)
What disease?	How does it work?
Recurrent respiratory papillomatosis (RRP) A rare disease in which HPV virus (type 6 or 11) causes repeated, benign warts in the larynx, trachea and lungs. The warts obstruct breathing and require frequent operations. Some patients must undergo surgery several times a year. There has so far been no medical treatment.	A harmless virus is used to "show" the immune system what the HPV virus proteins look like. The immune system thereby learns to recognize and attack the cells infected with HPV.
Who can get it?	How far along is the process?
Adults with RRP who need frequent operations. Treatment: Four injections under the skin over 12 weeks.	EMA has begun its first review of the documentation. There are typically 1-2 years until a possible recommendation. The product name and company will only be disclosed in the event of a positive assessment. (The product has already been approved by the FDA in the US under the name Papzimeos in August 2025).
TIL cell therapy (lifileucel)	Cell therapy
What disease?	How does it work?
Advanced melanoma The most serious form of skin cancer that has spread to other parts of the body. It is usually treated with immunotherapy (so-called checkpoint inhibitors) and possibly targeted treatment, but in some patients the disease stops responding, and there are limited treatment options left.	The patient's own immune cells are removed from the tumor through surgery, multiplied in the laboratory into billions of cancer-fighting cells, and then given back to the patient via a blood transfusion. The patient's own immune system thus becomes a "tailor-made" treatment.
Who can get it?	How far along is the process?
Adults with advanced melanoma where other treatment no longer works. Treatment: One-time treatment: surgery to collect cells, 3-4 weeks of laboratory work, then infusion.	EMA has begun its first review. The product name and company will only be disclosed in the event of a positive assessment. It is expected to be Iovance Biotherapeutics' Amtagvi, which is already approved in the US and Canada. A previous EU application was withdrawn and has now been resubmitted.
Zamtocabtagene autoleucel	CAR-T cell therapy (gene therapy + cell therapy)
What disease?	How does it work?
Expected: cancer of the lymphatic system (B-cell cancer) This CAR-T therapy is aimed at B-cell cancers. That is, cancer in the white blood cells that produce antibodies. It covers, among other things, certain forms of lymphoma and leukemia. CAR-T is typically used when chemotherapy and other treatment no longer work.	The patient's own T cells (a type of immune cell) are removed, genetically modified in the laboratory so that they get an artificial "radar" (CAR) that can recognize cancer cells, multiplied, and given back to the patient. The cells then hunt the cancer cells in the body.
Who can get it?	How far along is the process?
Expected: adults with certain B-cell cancers (exact indication to be determined). Treatment: One-time treatment: blood sample to collect cells, about 3-4 weeks of laboratory work, then infusion.	EMA has begun its first review. The product name, company and exact indication will only be disclosed in the event of a positive assessment. There are typically 1-2 years until a possible recommendation.

This overview was prepared based on Amgros' Horizon Scanning from April 2026 and publicly available information from EMA and FDA. The stated timeframes are estimates and may change.

Research

Advanced therapies are developed and tested here

Denmark has strong environments for advanced therapies – from research in the laboratory to the first clinical studies with patients.

CCIT (Center for Cancer Immune Therapy, Herlev Hospital)
CCIT has a strong national position and an increasingly visible European profile within advanced cancer immunotherapies. The center is characterized by an integrated structure with laboratory research, GMP-compliant cell production, and early clinical testing, which makes it possible to rapidly translate knowledge between research and patient care and supports the development and clinical translation of next-generation cell therapies, including T cell-based treatments.
Rigshospitalet
Rigshospitalet is Denmark’s leading tertiary hospital and provides advanced treatment and research to a large target group for advanced therapies, with more than 900 patients treated across 13 departments and 8 specialties. The hospital has solid experience with early clinical trials and clinical implementation of ATMPs and serves both as sponsor and as a trial site for a range of academic and industry-initiated studies. Research areas include, among others, CAR T-cell therapy in hematology and pediatrics, gene therapy for neurological diseases, and mesenchymal stem cell-based approaches to cardiac regeneration.
Aalborg University Hospital
Aalborg University Hospital has established a clinical trials unit that supports both commercial and non-commercial studies and offers GCP monitoring for non-commercial studies. The unit is part of Aalborg Hospital Science and Innovation Center and provides researchers with a single point of entry for guidance and support for preclinical trials, clinical trials, and innovation. Preclinical research includes, among other things, diagnostics and monitoring in immunotherapy using modern techniques in gene editing, sequencing, and PCR.
Phase IV units (University of Copenhagen/Bispebjerg Hospital, University of Southern Denmark, and Aarhus University)
University of Copenhagen (Bispebjerg Hospital), University of Southern Denmark, and Aarhus University have dedicated Phase IV units. Denmark is internationally strong in registry-based drug research with documented expertise in pharmacoepidemiology and post-authorization studies for both EMA and FDA.
Novo Nordisk Foundation Center for Stem Cell Medicine (reNEW) (University of Copenhagen)
reNEW was established with a grant of more than €300 million from the Novo Nordisk Foundation and focuses on stem cell medicine and regenerative therapies, including the social and ethical aspects of ATMP treatments.
CITCO (Centre for Cellular ImmunoTherapy of Haematological Cancer, Odense University Hospital, OUH)
CITCO is among the leading environments in chimeric antigen receptor therapy (CAR) and T cell engineering, with a focus on in vivo delivery of therapeutic products.
Odense University Hospital (OUH)
OUH is one of Denmark’s largest trial sites for CAR T-cell therapy and stem cell therapy, with several ongoing protocols, both commercial and the hospital’s own ATMPs. The hospital has access to large cleanroom facilities for clinical production of CAR T and is also planning a vector laboratory. OUH and the University of Southern Denmark are involved in several local and national partnerships with Danish and international medical and pharma companies, including AstraZeneca (Gothenburg), AlphaLyse (Odense), and Samplix (Birkerød), as well as the innovation network Life Science Fyn.
CeBIL (Center for Advanced Studies in Bioscience Innovation Law, University of Copenhagen)
CeBIL is an interdisciplinary research center focused on regulation, incentives, and policies for ATMPs as well as related areas such as medicines for rare diseases, personalized medicine, production of biological medicines, health data, and technology transfer.
CGCT (Center for Gene and Cellular Therapy, Aarhus University, AU)
CGCT focuses on regenerative medicine and immunomodulation with mesenchymal stem cells (MSC). The center has ongoing clinical ATMP trials in three indications and two additional ones under approval – primarily early-phase trials and investigator-initiated studies – as well as preclinical programs in gene editing of hematopoietic stem cells.
Aarhus University Hospital (AUH)
AUH is strongly engaged in the development and translation of advanced therapies, rooted in the close collaboration between CGCT and Aarhus University. AUH conducts several investigator-initiated ATMP trials, including CRISPR-based therapies and the development of next-generation CAR T, and contributes internationally through participation in, among others, the European University Hospital Alliance and Join4ATMP.

What are ATMPs?

Strong professional environments

Data and follow-up

Collaboration across sectors

A look into the future - ATMPs on the way

Adstiladrin

Gene therapy

Itvisma

Gene therapy

Zopapogene imadenovec

Gene therapy (immunotherapy)

TIL cell therapy (lifileucel)

Cell therapy

Zamtocabtagene autoleucel

CAR-T cell therapy (gene therapy + cell therapy)