Indication: Lipoprotein lipase deficiency

Treatment population: adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD) and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions.adults

Classiffication: GTMP

Year of approval: 2012

Current MA status: MA not renewed

EPAR: 

On the market of RS: /

Indication: Melanoma

Treatment population: adults with melanoma (a type of skin cancer) that cannot be surgically removed and that has spread to other parts of the body (but not to bone, lung, brain and other internal organs).

Classiffication: GTMP

Year of approval: 2015

Current MA status: Authorised

EPAR: 

On the market of RS: /

Indication: Melanoma

Treatment population: Severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)

Classiffication: GTMP

Year of approval: 2016

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: High-grade B-cell lymphoma (HGBL);
diffuse large B-cell lymphoma (DLBCL);
primary mediastinal large B-cell lymphoma (PMBCL);
Follicular lymphoma (FL)

Treatment population: patients whose blood cancer has returned (recurrent) or has stopped responding to previous treatment (refractory)

Classiffication: GTMP

Year of approval: 2018

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: B-cell acute lymphoblastic leukaemia (ALL)
Diffuse large B-cell lymphoma (DLBCL)
Follicular lymphoma (FL)

Treatment population: ALL in children and young adults up to 25 years of age whose cancer did not respond to previous treatment, has come back two or more times, or has come back after a transplant of stem cells; DLBCL and FL in adults whose cancer has come back or did not respond after two or more previous treatments.

Classiffication: GTMP

Year of approval: 2018

Current MA status: Authorised

EPAR: 

On the market of RS: Yes

Indication: Loss of vision due to inherited retinal dystrophy

Treatment population: adults and children

Classiffication: GTMP

Year of approval: 2018

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: Beta thalassaemia

Treatment population: patients 12 years and older who require regular blood transfusions

Classiffication: GTMP

Year of approval: 2019

Current MA status: Withdrawn

EPAR:

On the market of RS: /

Indication: Spinal muscular atrophy

Treatment population: patients with inherited mutations affecting a gene known as SMN1, who have either been diagnosed with SMA type 1 (the most severe type) or have up to 3 copies of another gene known as SMN2.

Classiffication: GTMP

Year of approval: 2020

Current MA status: Authorised

EPAR:

On the market of RS: Yes

Indication: Metachromatic leukodystrophy (MLD)

Treatment population: children who have mutations in the arylsulfatase A (ARSA) gene.

Classiffication: GTMP

Year of approval: 2020

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: Mantle cell lymphoma (a cancer of B cells, a type of white blood cell) when the cancer has come back after two or more previous treatments, including a type of cancer medicine called a Bruton’s tyrosine kinase (BTK) inhibitor;
Acute lymphoblastic leukaemia (ALL) when the cancer has come back or did not respond to previous treatments

Treatment population: MCL in adults; ALL in adults 26 years of age and older

Classiffication: GTMP

Year of approval: 2020

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: Early cerebral adrenoleukodystrophy (CALD)

Treatment population: children under 18 years of age

Classiffication: GTMP

Year of approval: 2021

Current MA status: Withdrawn

EPAR:

On the market of RS: /

Indication: Relapsed or refractory multiple myeloma

Treatment population: adults who have received at least two prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti‑CD38 antibody and whose disease has worsened since the last treatment.

Classiffication: GTMP

Year of approval: 2021

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: Relapsed or refractory diffuse large B-cell lymphoma (DLBCL);
high-grade B-cell lymphoma (HGBCL);
primary mediastinal large B-cell lymphoma (PMBCL);
follicular lymphoma grade 3B (FL3B)

Treatment population: patients whose cancer came back (relapsed) or did not respond (refractory) after an initial treatment with chemoimmunotherapy (a combination of systemic therapy to kill or slow the growth of cancer cells and immunotherapy to stimulate or restore the immune system’s ability to fight the cancer). In patients with relapsed or refractory DLBCL, PMBCL or FL3B, it can also be used after two or more previous treatments with systemic therapy (treatment given by mouth or injection)

Classiffication: GTMP

Year of approval: 2022

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: Relapsed or refractory multiple myeloma

Treatment population: adults who have received at least one prior therapy, including an immunomodulatory agent and a proteasome inhibitor, whose disease has worsened since the last treatment, and for whom treatment with lenalidomide did not work (refractory)

Classiffication: GTMP

Year of approval: 2022

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: Severe aromatic L-amino acid decarboxylase (AADC) deficiency

Treatment population: adults and children aged 18 months and older

Classiffication: GTMP

Year of approval: 2022

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: Severe haemophilia A

Treatment population: adults who do not have inhibitors (antibodies) against factor VIII and who have no antibodies against adeno-associated virus serotype 5 (AAV5)

Classiffication: GTMP

Year of approval: 2022

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: Severe and moderately severe haemophilia B

Treatment population: adults who have not developed inhibitors (proteins made by the body’s natural defenses) against factor IX

Classiffication: GTMP

Year of approval: 2023

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: Castration resistant prostate cancer

Treatment population: adults man

Classiffication: CTMP

Year of approval: 2013

Current MA status: Withdrawn

EPAR:

On the market of RS: /

Indication: Add-on treatment in adults who have received a haematopoietic stem cell transplant (HSCT, a transplant of cells that can develop into different types of blood cells) from a partially matched donor (a so-called haploidentical transplant)

Treatment population: patients who have received a haploidentical HSCT because they have serious blood cancers, such as certain
leukaemias and lymphomas.Before receiving an HSCT, the patient will have received treatment to
remove existing cells from the bone marrow, including cancer cells and immune cells

Classiffication: CTMP

Year of approval: 2016

Current MA status: Withdrawn

EPAR:

On the market of RS: /

Indication: Complex anal fistulas 

Treatment population: adults with Crohn’s disease (an inflammatory condition of the gut) when a conventional or biological medicine has not worked well enough.

Classiffication: CTMP

Year of approval: 2018

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: Blood cancer called Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD)

Treatment population: adults and children from 2 years of age

Classiffication: CTMP

Year of approval: 2022

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: Beta thalassaemia and sickle cell disease

Treatment population: patients 12 years and older, who require regular blood transfusions

Classiffication: CTMP

Year of approval: 2024

Current MA status: Authorised

EPAR:

On the market of RS: /

Indication: To repair damage to the cartilage in the knee.

Treatment population: adults, who have a single defect in the cartilage of the femoral condyle (the bottom end of the thighbone) that is causing symptoms

Classiffication: TEP

Year of approval: 2009

Current MA status: Withdrawn

EPAR:

On the market of RS: /

Indication: Implant used to repair cartilage defects at the ends of the bones of the knee joint

Treatment population: adults who are experiencing symptoms (such as pain and problems moving the knee).

Classiffication: TEP

Year of approval: 2013

Current MA status: MA not renewed

EPAR:

On the market of RS: /

Indication: Stem-cell treatment used in the eye to replace damaged cells on surface (epithelium) of the cornea, the transparent layer in front of the eye covering the iris (the coloured part).

Treatment population: adult patients with moderate to severe limbal stem-cell deficiency caused by burns, including chemical burns, to the eyes.

Classiffication: TEP

Year of approval: 2015

Current MA status: Authorised

On the market of RS: /

Indication: To repair defects to the cartilage in the knee in patients who are experiencing symptoms (such as pain and problems moving the knee)

Treatment population: adults and in adolescents whose bones in the joints have finished growing

Classiffication: TEP

Year of approval: 2017

EPAR:

On the market of RS: /