Closed programs

Commercial name Active substance Indication
Ozurdex® Intravitreal implant with dexamethasone 700 microgram Diabetic macular edema
Vargatef® Nintédanib In combination with docetaxel adult patients with locally advanced, metastatic or locally recurrent non-small cell lung cancer of adenocarcinoma tumour histology progressing after one prior line of chemotherapy
Imbruvica® Ibrutinib Patients suffering from relapsed or refractory lymphocytic leukaemia/small lymphocytic lymphoma
Enbrel® Etanercept Psoriatic arthritis in children aged 12-17 years
Gazyvaro® Obinutuzumab In combination with chlorambucil for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL) and with comorbidities making them unsuitable for full-dose fludarabine based therapy
Entyvio® Vedolizumab Patients with ulcerative colitis that have failed at least 2 previous anti-TNF or for whom anti-TNF are contra-indicated
Mepolizumab® Mepolizumab Eosionophylic granulomatosis with polyangitis (EGPA or churg-Strauss Syndrom)
Xtandi® Enzalutamide Asymptomatic or mildly symptomatic metastatic castration-resistant prostate cancer in adult patients whose disease progressed during or after androgen deprivation therapy (ADT) but for whom chemotherapy is not yet clinically indicated
Zydelig® Idelalisib In combination with rituximab for relapsed chronic lymphocytic leukemia in adult patients who cannot be treated with chemotherapy
Avastin ® Bevacizumab in combination with paclitaxel and cisplatin or, alternatively, paclitaxel and topotecan in patients who cannot receive platinum therapy, in adult patients with persistent, recurrent, or metastatic carcinoma of the cervix
Zydelig® Idelalisib Follicular lymphoma (FL) refractory to two prior lines of therapy
Entyvio® Vedolizumab Patients with Crohn disease that have failed at least 2 previous anti-TNF or for whom anti-TNF is contra-indicated
Keytruda®
 
Pembrolizumab – MK 3475 Unresectable or metastatic melanoma in patients who are refractory to treatment with ipilimumab
Keytruda® Pembrolizumab – MK 3475 Advanced (unresectable or metastatic) melanoma in first line or after BRAF inhibitor 
Pixuvri® Pixantrone monotherapy for the 3rd or 4th line treatment of adult patients with multiply relapsed or refractory aggressive Non‐Hodgkin B‐cell Lymphomas (NHL) (DLBCL and transformed indolent lymphoma)
Raxone® Idebenone For the treatment of yung patients with Duchenne Muscular Dystrophy who are not taking contamitant glucocorticoid steroids
Humira ® Adalimumab Active moderate to severe Hidradenitis Suppurativa (HS) in adult patients with an inadequate response to conventional systemic HS therapy
Kyprolis® carfilzomib in combination with lenalidomide and dexamethasone (KRd) for the treatment of adult patients with relapsed multiple myeloma who did receive at least 1 prior therapy
Cobimetinib® Cobimetinib In combination with Zelboraf® (vemurafenib) in patients with unresectabel or metastatic BRAFV600 mutation-positive melanoma
Tagrisso® Osimertinib Tagrisso® is indicated for the treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC)
Opdivo® Nivolumab Medical Need Program with Opdivo (nivolumab) for the treatment of anti-PD-1-treatment naïve patients with advanced renal clear-cell carcinoma who have progressed after or failed at least one prior treatment with an anti-angiogenic agent
Zepatier® Elbasvir / grazoprevir Treatment of Chronic Kidney Disease stage 4/5 patients with hepatitis C  genotype 1 or 4 infection
Opdivo® Nivolumab locally advanced and metastatic Non-Small-Cell Lung Cancer (NSCLC) in patients who have documented progression on or after prior chemotherapy
Opdivo® Nivolumab Classical Hodgkin lymphoma in adult patients who have relapsed after ASCT and brentuximab vedotin treatment.
Zykadia® Ceritinib ALK positive NSCLC patients that have received prior therapy with crizotinib
Revolade® Eltrombopag Thrombocytopenia in patients with severe aplastic anemia
Darzalex® Daratumumab Patients who have relapsed and refractory multiple myeloma (MM), who have received at least 3 prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD) or whose disease is double refractory to both a PI and an IMiD
Orkambi® lumacaftor/ivacaftor Cystic fibrosis in patients 12yrs and older who are homozygous for the F508del CFTR mutation
Entresto® Sacubitril/Valsartan
 
Medical Need Program with Entreso (Sacubitril/Valsartan) for the treatment symptomatic chronic heart failure with reduced ejection fraction in adult patients.
Abraxane® paclitaxel in combination with gemcitabine for the first line treatment of metastatic adenocarcinoma of the pancreas
KEYTRUDA® pembrolizumab First‐Line treatment of metastatic Non‐Small Cell Lung Cancer (NSCLC) in adults whose tumors express PD‐L1 with a ≥50% tumor proportion score (TPS) with no EGFR‐ or ALK‐ positive tumor mutations
Repatha® Evolocumab Severe uncontrolled familial hypercholesterolemia (FH), despite treatment with a maximum tolerated dose of a statin in combination with ezetimibe, or with ezetimibe without a statin in case of statin intolerance or contra‐indication
Humira®  Adalimumab Active enthesitis-related arthritis in patients, 6 years of age and older, who have had an inadequate response to, or who are intolerant to, conventional therapy
Humira®  Adalimumab Treatment of moderately to severely active Crohn's disease in paediatric patients (from 6 years of age) who have had an inadequate response to conventional therapy including primary nutrition therapy and a corticosteroid and/or an immunomodulator, or who are intolerant to or have contraindications for such therapies
Kyprolis®

carfilzomib

in combination with dexamethasone (Kd) for the treatment of adult patients with relapsed multiple myeloma who did receive 3 or more prior therapies

Stelara® Ustekinumab

Treatment of moderate to severe active Crohn’s disease who have failed TNFα antagonists (infliximab/infliximab biosimilar, adalimumab) and the integrin inhibitor vedolizumab, or who are intolerant to, or have a contraindication to these treatments.

Humira®

Adalimumab

Treatment of non-infectious intermediate, posterior and pan-uveitis

Revolade® Eltrombopag

Thrombocytopenia in patients with myelodisplastic syndrome.

Ofev® Nintedanib Idiopathic pulmonary fibrosis (IPF) in adult patients
Venclyxto® Venetoclax Treatment of chronic lymphocytic leukaemia in adult patients who are unsuitable for or have failed other treatments.
Kisqali®

Ribociclib

In combination with letrozole for adult postmenopausal women as initial endocrine-based therapy for hormone receptor-positive (HR+) HER2-negative (HER2-) locally advanced or metastatic breast cancer.

Alprolix®

Eftrenacog alfa

Treatment and prophylaxis of bleedings in hemophilia B patients (deficiency= Factor IX coagulation factor)

Lartruvo® Olaratumab

In combination with doxorubicin for the treatment Advanced soft tissue sarcoma not amenable to curative treatment with surgery or radiotherapy and not previously treated with doxorubicin

Cabometyx® Cabozatinib Advanced renal cell carcinoma in adults following prior vascular endothelial growth factor (VEGF)- targeted therapy
Tecentriq®

Atezolizumab

locally advanced or metastatic urothelial carcinoma in adult, previously untreated patients who are considered cisplatin ineligible
Trisenox® Arsenic trioxide In combination with alltrans retinoic acid (ATRA) for the first‐line treatment of low or intermediate risk acute promyelocytic leukemia (APL)
Blincyto®

Blinatumomab

Adults with Philadelphia chromosome negative relapsed or refractory B-precursor acute lymphoblastic leukaemia (ALL)

Wakix® Pitolisant Second-line treatment of adult patients with narcolepsy with or without cataplexy
Lenvima®

Lenvatinib

Radioiodine-refractory differentiated thyroid cancer.

Halaven®

Eribulin

Treatment of adult patients with unresectable liposarcoma who have received prior anthracycline containing therapy (unless unsuitable) for advanced or metastatic disease.

Blincyto®

Blinatumomab

Adults with Philadelphia chromosome negative relapsed or refractory B-precursor acute lymphoblastic leukaemia (ALL)

Xalkori®

Crizotinib

ROS1-positive non-small cell lung cancer (NSCLC)

Ilaris®

Canakinumab

Hereditary periodic fever

Ibrance®

palbociclib

patients suffering from advanced/metastatic breast cancer hormone receptor (HR)‑positive, human epidermal growth factor receptor 2 (HER2)‑negative with endocrine therapy

Bavencio

Avelumab

Treatment of adult patients with metastatic Merkel cell carcinoma whose disease has progressed after receiving at least one prior chemotherapy or who are ineligible for chemotherapy

Spinraza® Nusinersen Infantile-onset (Type I) Spinal Muscular Atrophy (SMA)
Alecensa® Alectinib

First-line treatment of adult patients with anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC)

Zytiga®

Abiraterone acetate

Treatment of newly diagnosed with high risk Metastatic hormone sensitive prostate cancer (mHSPC) in adult men in combination with androgen deprivation therapy (ADT) and prednisone or prednisolone.

Rydapt® Midostaurin (PKC412) Newly diagnosed FLT3 mutated acute myeloid leukemia patients eligible for standard induction and consolidation chemotherapy.

Xermelo

 

Telotristat etiprate

Treatment of carcinoid syndrome diarrhea in combination with somatstatin analogue (SSA) therapy in adults inadequately controlled by SSA therapy.

Tafinlar + Mekinist

Dabrafenib + Trametinib

Traitement de patients atteints d’un cancer bronchique non à petites cellules (CBNPC) avancé porteur d’une mutation BRAF V600.

Humira Adalimumab Treatment of paediatric chronic non-infectious anterior uveitis patients (from 2 years of age) who have had an inadequate response to conventional therapy including topical corticosteroid and an immunomodulator, or who are intolerant to or have contraindications for such therapies.
Zavicefta®

Ceftazidime/Avibactam

Treatment of infections due to aerobic Gram-negative organisms in adult patients with no other treatment options available.

Ocrevus® Ocrelizumab Primary progressive multiple sclerosis.
Keytruda® Pembrolizumab - MK 3475 In combination with chemotherapy (carboplatin + paclitaxel) for the first-line treatment of metastatic squamous non-small cell lung cancer (NSCLC) in adults whose tumours express PD-L1 with a tumour proportion score (TPS) between l%-49% or no PD-L1 (TPS <1%).
Imfinzi

durvalumab

Treatment of locally advanced (stage III), unresectable non-small cell lung cancer (NSCLC) in adults whose tumours express PD-L1 on ≥ 1% of tumour cells and whose disease has not progressed following platinum-based chemoradiation therapy.

Dupixent Dupilumab Treatment of moderate-to-severe atopic dermatitis in adult patients coming from the Open Label Extension study (OLE)R668-AD-1225 and who are candidates for systemic therapy
Dupixent® Dupilumab Treatment of severe atopic dermatitis in adult patients, uncontrolled with immunosuppressive systemic therapy during the last year.
Vimpat®

Lacosamide

Partial-onset or generalized tonic clonic seizures in patients ≥ 16 years of age coming from the SP0994 clinical trial

Mylotarg ®

Gemtuzumab Ozogamicin

CD33-positive relapsed or refractory acute myeloid leukemia (AML) or CD33-positive relapsed or refractory acute promyelocytic leukemia (APL).

Perjeta® Pertuzumab In combination with Herceptin® (trastuzumab) and chemotherapy for the adjuvant treatment of adult patients with HER2 positive early breast cancer at high risk of recurrence defined as lymph node positive disease.
Besponsa®

inotuzumab ozogamicin

relapsed or refractory CD22-positive B-Cell precursor acute lymphoblastic leukemia (ALL) in adult patients

Tafinlar + Mekinist

Dabrafenib  + Trametinib

adjuvant treatment of high-risk BRAF V600 mutation-positive stage III melanoma after complete surgical resection.

Cablivi caplacizumab

acquired thrombotic thrombocytopenic purpura.

Entresto

sacubitril / valsartan

heart failure with preserved fraction (HFpEF) for patients who participated in study CLCZ696D2301 (PARAGON-HF)

RoACTEMRA® Tocilizumab

Systemic sclerosis.

Xeljanz®

Tofacitinib citrate

Treatment of adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to either conventional therapy or a biologic agent.

Tecentriq

Atezolizumab

In combination with carboplatin and etoposide, for the first-line treatment of adult patients with extensive-stage small-cell lung cancer (ES-SCLC).

Raxone ®

Idebenone

Leber’s hereditary optic neuropathy (LHON)  

Lynparza®

Olaparib

Treatment of patients with deleterious or suspected deleterious germline BRCA1 or BRCA2 mutation and who have previously been treated with chemotherapy in the neoadjuvant, adjuvant or metastatic setting for TNBC or HER2-/ HR+ breast cancer unsuitable for further anti-hormonal therapy.

Onpattro

Patisiran-LNP

hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy

Gilenya®

Fingolimod

Relapsing multiple sclerosis (RMS) in for pediatric patients aged 10 to less than 18 years old of 40 kg or above.

Yervoy®

Ipilimumab

Treatment-Naïve Intermediate and Poor Risk Patients with Advanced or Metastatic Renal Cell Carcinoma in combination with Opdivo® (nivolumab)

Aimovig®

erenumab

Treatment of patients diagnosed with migraine, who are in need of prophylactic treatment and for whom no acceptable alternative prophylactic treatment is available.

RoActemra®

tocilizumab

Giant cell arteritis (GCA or temporal arteritis)

Hemlibra

Emicizumab

Routine prophylaxis of bleeding episodes in patients with hemophilia A with factor VIII inhibitors. Hemlibra can be used in all age groups.

Hemlibra

Emicizumab

Routine prophylaxis of bleeding episodes in patients with severe haemophilia A without factor VIII inhibitors and with inadequate response on their current treatment.

Tecentriq® Atezolizumab In combination with bevacizumab, paclitaxel and carboplatin, for the first-line treatment of adult patients with stage IV or recurrent metastatic non-squamous non-small cell lung cancer (NSCLC) and whose tumors express PD-L1 < 50% with no EGFR or ALK positive tumors.
Opdivo

nivolumab

Treatment of adult patients with unresectable Malignant Pleural Mesothelioma who have progressed after at least one line of treatment including a platinum-based doublet chemotherapy.

Polivy® Polatuzumab vedotin In combination with bendamustine and rituximab for relapsed or refractory adult patients with diffuse large B-cell lymphoma (DLBCL) who are not candidates for hematopoietic stem cell transplant and have been treated with at least two prior lines of therapy.
Signifor LAR®

Pasireotide

Patients with acromegaly who are inadequately controlled with 1st generation somatostatin analogues

LYNPARZA

olaparib

maintenance treatment of adult patients with advanced (FIGO stages III and IV) BRCA1/2-mutated (germline and/or somatic) high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) following completion of first-line platinum-based chemotherapy

Keytruda®

Pembrolizumab

In combination with platinum and 5-fluorouracil (5-FU) chemotherapy for the first-line treatment of recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) in adults with a PDL-1 CPS score=0.

COR-003 (2S,4R-ketoconazole)®

COR-003 (2S,4R-ketoconazole)

Endogenous Cushing’s Syndrome.

Xarelto Rivaroxaban Co-administered with acetylsalicylic acid (ASA), for the treatment of patients with coronary artery disease (CAD) at high risk of ischaemic events.
Briviact® Brivaracetam

Adjunctive treatment of partial-onset seizures with or without secondary generalisation in adults, adolescents and children from 4 years of age with epilepsy.

Ipatasertib (MO40153)

Ipatasertib (MO40153)

In combination with paclitaxel in PIK3CA/AKT1/PTEN altered, locally advanced or metastatic, triple-negative breast cancer or hormone-receptor-positive, HER2-negative breast cancer.

Lonsurf®

trifluridine/tipiracil

monotherapy for the treatment of adult patients with metastatic gastric cancer including adenocarcinoma of the gastroesophageal junction, who have been previously treated with at least two prior systemic treatment regimens for advanced disease.
Raxone®

Idebenone

Patients with Duchenne Muscular Dystrophy (DMD) who completed DELOS study (SNT-III-003/ EudraCT 2009-012037-30)

Vimpat®

lacosamide

Adjuvant therapy in the treatment of partial seizures with or without secondary generalization in adults, adolescents and children from 4 years old with epilepsy.

Lorlatinib Lorlatinib (PF-06463922) Metastatic NSCLC (Stage IV, AJCC v7.0) that carries an ALK rearrangement or that carries a ROS1 rearrangement
Bavencio avelumab first-line maintenance treatment of adult patients with locally advanced or metastatic urothelial carcinoma (UC) without disease progression following first-line platinum-based induction chemotherapy
Keytruda® pembrolizumab first-line treatment of adult patients with Microsatellite Instability-High (MSI-H) or Mismatch Repair-Deficient (dMMR) metastatic Colorectal Carcinoma (mCRC)
Ajovy® fremanezumab Prophylaxis of migraine in adult patients who suffer from at least 8 monthly migraine days and who previously failed on at least 3 preventive treatment classes and cannot be satisfactorily treated with the other approved and commercially available alternative treatments, in accordance with clinical guidelines, because of efficacy and/or safety issues.
Kadcyla trastuzumab emtansine as a single agent for the adjuvant treatment of adult patients with HER2 positive early breast cancer who have residual invasive disease, in the breast and/or lymph nodes, after neoadjuvant taxane-based and HER2-targeted therapy.
Yescarta® Axicabtagene ciloleucel Treatment of aggressive conditions in adults with primary mediastinal B-cell lymphoma (PMBCL) affecting your lymph tissue (part of the immune system) that affects a type of white blood cell called B lymphocytes and other organs in your body.
Tecentriq Atezolizumab Treatment of first line for adult patients with locally advanced or metastatic, positive PD-L1, triple negative breast cancer, in combination with nab-paclitaxel.
Tecentriq® Atezolizumab in combination with bevacizumab, is indicated for the first-line treatment of adult patients with advanced or unresectable hepatocellular carcinoma (HCC) who have received no prior systemic therapy
Libtayo Cemiplimab Treatment of patients with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for curative surgery or curative radiation.
Adcetris Brentuximab vedotin in combination with cyclophosphamide, doxorubicin and prednisone (CHP) for the treatment of adult patients with previously untreated systemic anaplastic cell lymphoma (sALCL)
Orkambi® lumacaftor 100mg / ivacaftor 125mg Treatment of cystic fibrosis (CF) in patients 6 through 11 years of age who are homozygous for the F508del mutation in the CFTR gene.
Vitrakvi Larotrectinib Treatment of adult and paediatric patients with locally advanced or metastatic solid tumors with a Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusion that cannot be treated satisfactorily with the available treatment options
Cyramza® ramucirumab treatment of adult patients with advanced or unresectable hepatocellular carcinoma who have a serum alpha fetoprotein (AFP) of ≥ 400 ng/ml and who have been previously treated with systemic therapy.
Gilteritinib (ASP2215) gilteritinib (ASP2215) treatment of patients with FMS-like tyrosine kinase 3 (FLT3)-mutated relapsed or refractory acute myeloid leukemia (AML) without access to comparable or alternative therapy.
RoActemra tocilizumab treatment of patients 50 years or older with active Giant Cell Arteritis who cannot be adequately treated with the current standard GC therapy.
Blincyto® Blinatumomab Adults with B-precursor acute lymphoblastic leukemia (ALL) in complete hematological remission defined as less than or equal to 5% blasts in the bone marrow after at least three intense chemotherapy blocks and presence of minimal residual disease (MRD) at a level≥10-4
Fremanezumab (TEV-48125) Fremanezumab (TEV-48125) Patients with episodic migraine or chronic migraine who have successfully completed (per protocol) Study TV48125‐CNS‐30051 or the treatment phase of Study TV48125‐CNS‐30068.
Vyndaqel® tafamidis treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
Metycor® Metyparone Endogenous Cushing’s syndrome in patients who have completed the study extension period of the PROMPT clinical trial with metyrapone.
Keytruda pembrolizumab in combination with platinum and fluoropyrimidine based chemotherapy as first-line treatment for patients with locally advanced unresectable or metastatic carcinoma of the esophagus or HER-2 negative gastroesophageal junction Siewert type I adenocarcinoma in adults whose tumours express PD-L1 with a CPS≥10
Givlaari® givosiran treatment of acute hepatic porphyria (AHP) in adults and adolescents (≥12 years of age)
Revolade eltrombopag patients aged 1 year and above with primary immune thrombocytopenia (ITP) lasting 6 months or longer from diagnosis and who are refractory to other treatments (e.g. corticosteroids, immunoglobulins)
VX-445/TEZ/IVA elexacaftor / tezacaftor / ivacaftor Cystic fibrosis (CF) patients in critical need who are 12 years of age and older, heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes)
VX-445/Tezacaftor/Ivacaftor and Ivacaftor VX-445/Tezacaftor/Ivacaftor and Ivacaftor Cystic fibrosis (CF) patients in critical need who are 12 years of age and older, homozygous for F508del (F/F genotypes).
Fycompa® perampanel Partial onset seizures for patients who have previously participated in Study 311 and are aged <12 years.
Erleada® Apalutamide in combination with androgen deprivation therapy (ADT) for the treatment of adult patients having castration resistant prostate cancer with PSADT ≤ 10 months and no detected metastases using imaging as per physician’s clinical practice.
Erleada® Apalutamide in combination with androgen deprivation therapy (ADT) for the treatment of adult patients with metastatic hormone-sensitive prostate cancer (mHSPC).
Alpelisib® Alpelisib In combination with fulvestrant or letrozole for postmenopausal women and men with endocrine resistant hormone receptor-positive (HR+) HER2-negative (HER2-) metastatic breast cancer, who have recurrence or progressed after at least 3 lines of systemic treatment for advanced or metastatic disease, and who harbor specific PIK3CA hotspot mutations.
Doptelet Avatrombopag severe thrombocytopenia in adult chronic liver disease (CLD) patients who are scheduled to undergo an invasive procedure.
Polivy® Polatuzumab vedotin In combination with bendamustine and rituximab for the treatment of adult patients with relapsed/refractory diffuse large B‐cell lymphoma (DLBCL) who are not candidates for haematopoietic stem cell transplant.
Entrectinib Entrectinib treatment for adult patients, with NTRK fusion-positive, locally advanced or metastatic solid tumors.
Entrectinib Entrectinib adult patients with locally advanced or metastatic Non-Small Cell Lung Cancer (NSCLC) with Central Nervous System (CNS) metastases, harbouring a ROS1 gene fusion, who have not received prior ROS1 inhibitor.
Spravato® Esketamine Indicatie (EN): In combination with a SSRI or SNRI, for treatment-resistant depression (Major Depressive Disorder) in adults who have not responded to at least two different treatments with antidepressants in the current moderate to severe depressive episode
Braftovi® encorafenib in combination with cetuximab, for the treatment of adult patients with metastatic colorectal carcinoma with a BRAFV600E mutation, who have received prior systemic therapy
Zejula® niraparib maintenance treatment of adult female patients with BRCA1/2 wild-type advanced epithelial high-grade ovarian, fallopian tube or primary peritoneal cancer in response following first-line platinumbased chemotherapy
Siponimod BAF312 Treatment for adult patients with Secondary Progressive Multiple Sclerosis (SPMS).
Opdivo® nivolumab first line treatment of adult patients with HER2 negative advanced or metastatic gastric, gastro oesophageal junction or oesophageal adenocarcinoma whose tumours express PD-L1 with a CPS ≥ 5 in combination with fluoropyrimidine- and platinum-based chemotherapy.
Nplate® romiplostim Treatment of adult patients with Primary Immune thrombocytopenia (ITP) within the first 12 months of diagnosis who are refractory to other treatments (e.g. corticosteroids, immunoglobulins)
Venclyxto® venetoclax in combination with azacitidine for the treatment of newly diagnosed acute myeloid leukemia (AML) in adult patients who are ineligible for intensive chemotherapy and who have no other suitable treatment option and are not eligible to participate in a clinical study for their serious condition.
Rinvoq® upadacitinib adult patients with severe Ankylosing Spondylitis who have no other suitable treatment option and are not eligible to participate in a clinical study for their serious condition
Blenrep belantamab mafodin multiple myeloma in adult patients, who have received at least four prior therapies and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy
Retsevmo Selpercatinib adults with advanced RET fusion-positive non-small cell lung cancer (NSCLC) following prior systemic therapy
Retsevmo® Selpercatinib treatment of adults with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require
systemic treatment following prior treatment
Nubeqa® darolutamide treatment of adult men with non-metastatic castration resistant prostate cancer (nmCRPC) who are at high risk of developing metastatic disease
Lynparza olaparib patients with metastatic castration resistant prostate cancer who have progressed on prior NHA and have a BRCA mutation (germline or somatic)
Sarclisa® isatuximab in combination with pomalidomide and dexamethasone (Isa-Pd) for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on the last therapy
Xtandi® enzalutamide treatment of adult men with metastatic hormone‐sensitive prostate cancer (mHSPC) in combination with androgen deprivation therapy (pending final marketing authorisation)
Opdivo and Yervoy nivolumab and ipilimumab First line treatment of adult patients with unresectable malignant pleural mesothelioma.
Aubagio® Teriflunomide Relapsing Remitting Multiple Sclerosis in the pediatric population.
Olumiant baricitinib moderate to severe atopic dermatitis (AD), defined as EASI score ≥16 before start of treatment, in adult patients that qualify for systemic treatment
Risdiplam® risdiplam treatment of patients who suffer from Type 1 SMA
risdiplam risdiplam Spinal Muscular Atrophy  Type 2 for patients ≥2 months.
Keytruda® pembrolizumab in combination with chemotherapy as neoadjuvant treatment and then continued as monotherapy as adjuvant treatment after surgery for the treatment of adults with locally advanced, or early-stage triple-negative breast cancer at high risk of recurrence
Keytruda® pembrolizumab in combination with chemotherapy with or without bevacizumab for the treatment of persistent, recurrent, or metastatic cervical cancer in adults whose tumours express PD-L1 with a CPS ≥1
Revlimid® Lenalidomide Diffuse large B cell lymphoma in patients who already received at least two prior treatment lines
Tecentriq® atezolizumab adjuvant treatment following resection and platinum‐based chemotherapy for adult patients with stage IIB‐III non‐small cell lung cancer (NSCLC) whose tumours have PD‐L1 expression on ≥50% of tumour cells (AL43776).
Ofev nintedanib adult patients with non-IPF (idiopathic pulmonary fibrosis) chronic fibrosing interstitial lung diseases (ILDs) with progressive phenotype (PF-ILD's).
Pemazyre pemigatinib adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that is relapsed or refractory after at least one line of systemic therapy
Remimazolam Remimazolam Sedation of Covid-19 patients under artificial ventilation in the Intensive Care Unit.
Remimazolam Remimazolam Induction and Maintenance of General Anesthesia.
Otezla apremilast Treatment of adult patients with oral ulcers associated with Behçet’s disease (BD) who are candidates for systemic therapy. The patient has received prior treatment with a topical therapy AND with colchicine. The patient has shown to be resistant or intolerant to colchicine or has a documented contraindication against colchicine.
Lenvima® lenvatinib in combination with pembrolizumab for the treatment of patients with advanced or recurrent endometrial carcinoma in adults who have disease progression on or following prior treatment with a platinum-containing therapy in any setting and who are not candidates for curative surgery or radiation. 
Vyxeos liposomal daunorubicin and cytarabine Newly diagnosed, treatment-related acute myeloid Leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC)
Tecartus autologous anti-CD19 CD3+ cells (CAR+ viable T cells) treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) after two or more lines of systemic therapy including a Bruton's tyrosine kinase (BTK) inhibitor
Forxiga® dapagliflozin Treatment of chronic kidney disease
Jemperli® dostarlimab as monotherapy for the treatment of adult patients with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) recurrent or advanced endometrial cancer (EC) that has progressed on or following prior treatment with a platinum-containing regimen.
Humira® adalimumab moderately to severely active Ulcerative Colitis in paediatric patients of 2 to 5 years old.
Soliris® eculizumab patients with refractory generalized myasthenia gravis (rgMG) who completed the study ECU-MG-302 (Open label extension) and, in the opinion and the clinical judgement of the treating physician would continue to benefit from a treatment with Eculizumab (Soliris®) which is not yet commercially available for this indication in Belgium.
LUMYKRAS sotorasib Treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with KRAS G12C mutation and who have progressed after at least one prior line of systemic therapy
Gilenya® fingolimod treatment of patients with relapsing-remitting multiple sclerosis (RRMS) who are included in medical need program CFTY720DBE02_C_IPS
Trodelvy® sacituzumab govitecan treatment in monotherapy of adult patients with unresectable, locally advanced or metastatic triple-negative breast cancer (mTNBC) who have received two or more prior systemic therapies, including at least one of them for advanced disease and with no adequate treatment options
Kaftrio ELX/TEZ/IVA and IVA alone Cystic fibrosis (CF) in patients from 6 to 11 years old who are homozygous for F508del (F/F genotype) or heterozygous for F508del and a minimal function (F/MF genotype) mutation.
Kerendia finerenone Adult patients with chronic kidney disease (CKD) (stage 3 & 4 with albuminuria) associated with type 2 diabetes (T2D) who cannot be satisfactorily treated with the standard of care.
Imfinzi durvalumab First-line Treatment for Patients with Locally Advanced or Metastatic Biliary Tract Cancers in combination with Gemcitabine Plus Cisplatin Therapies
tepotinib tepotinib Treatment of adult patients with advanced non-small cell lung cancer harboring mesenchymal-epithelial transition factor gene METexon14 (METex14) skipping alterations.
Skyrizi Risankizumab treatment of patients with active Crohn's Disease who have no other suitable treatment option and are not eligible to participate in a clinical study for their serious or life-threatening condition.
Padcev enfortumab vedotin locally advanced or metastatic urothelial cancer in patients who have previously received a platinum-containing chemotherapy and a programmed death receptor 1 (PD-1) or programmed death ligand 1 (PD-L1) inhibitor
Opdivo + Yervoy nivolumab + ipilimumab Treatment of adult patients with unresectable malignant pleural mesothelioma that have progressed after at least one line of treatment, including platinumbased doublet chemotherapy.
Aspaveli® pegcetacoplan Treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic despite treatment with a C5 inhibitor for at least 3 months.
Jardiance® empagliflozin treatment of adult patients with heart failure with preserved ejection fraction (ejection fraction >40%)
Verzenios abemaciclib in combination with Endocrine Therapy as the adjuvant treatment of adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, nodepositive early breast cancer at high risk of recurrence
Lumasiran lumasiran Treatment of primary hyperoxaluria type 1 (PH1) in patients who have reached at least 37 weeks estimated gestational age (full‐term infant) at consent (or assent).
Enhertu® trastuzumab deruxtecan adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens.
Olorofim F901318 Treatment of invasive fungal infections due to Lomentospora prolificans, Scedosporium spp., Aspergillus spp., and other resistant fungi in patients lacking suitable alternative treatment.
Verquvo® vericiguat Adult patients with symptomatic chronic heart failure (New York Heart Association (NYHA) Class II-III-IV) with a reduced ejection fraction who are stabilized after a previous decompensation event requiring IV therapy.
iNOmax® iNOmax patients who suffer from pulmonary hypertension associated with COPD and who participated in the PULSE-COPD-007 study
Rinvoq® upadacitinib the treatment of adolescents ≥ 12 years old with severe atopic dermatitis who have no other suitable treatment option and are not eligible to participate in a clinical study
Tecvayli teclistamab monotherapy, for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior lines of therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and who have exhausted all commercially approved and clinically appropriate treatment options, are ineligible for a clinical trial and have evidence of disease progression after the last therapy.
Inrebic® fedratinib treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis have been treated with ruxolitinib.
Sarclisa Isatuximab In combination with carfilzomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy and being lenalidomide exposed/refractory.
Nexviadyme  Avalglucosidase alfa treatment of adult Pompe disease patients who have participated and exit the clinical studies COMET (EFC14028) or NEO-EXT (LTS13769).
Minjuvi Tafasitamab the treatment of adult patients who have Relapsed or Refractory Diffuse Large B-Cell Lymphoma (R/R DLBCL) and who are not eligible for autologous stem cell transplantation (ASCT)
Darzalex daratumumab in combination with cyclophosphamide, bortezomib and dexamethasone for the treatment of adult patients with newly diagnosed systemic amyloid light chain (AL) amyloidosis
Adtralza tralokinumab Adolescent patients (12 ≤ 18 years) with severe atopic dermatitis (and a body weight < 60 kg) who are candidate for systemic treatment (including patients from the open label extension study ECZTEND LP0162-1337)
Tebentafusp tebentafusp HLA-A*02:01 positive patients with metastatic uveal melanoma
Cosentyx secukinumab Treatment of active moderate to severe hidradenitis suppurativa.
Nubeqa darolutamide Treatment of adult men with metastatic hormone-sensitive prostate cancer (mHSPC) in combination with ADT and docetaxel.
Fycompa Perampanel Seizures associated with Lennox Gastaut Syndrome (LGS) for pediatric patients under 12 years of age.
Evrenzo® roxadustat Treatment of symptomatic anaemia of Chronic Kidney Disease in non-dialysis and dialysis patients, and where use of available treatment (IV iron or injectable ESAs) is not appropriate or is expected not to be appropriate, or patients who have a known contraindication for the currently available treatment or who do not tolerate the currently available treatment, or are expected not to tolerate this product
Kapruvia difelikefalin treatment of moderate to severe chronic kidney disease-associated pruritus (CKD-aP) in adults undergoing hemodialysis (HD)
Rinvoq updacitinib the treatment of patients with active Crohn's Disease who have no other suitable treatment option and are not eligible to participate in a clinical study for their serious condition
tremelimumab tremelimumab as first-line treatment in patients with unresectable hepatocellular carcinoma in combination with Durvalumab (Imfinzi)
Vyvgart efgartigimod alfa add-on to standard therapy treatment for patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive and who demonstrate inadequate response to conventional treatments, are not clinically eligible or with contra-indication to treatment with approved and commercially available treatment and are not eligible to participate in a clinical trial
Brukinsa zanubrutinib For the treatment of adult patients with Marginal Zone Lymphoma (MZL) who have received at least one prior anti-CD20 based therapy, and for whom there are no available clinical studies
Ultomiris ravulizumab the treatment of atypical Haemolytic Uremic Syndrome in patients who completed the Open Label Extension of the phase III studies ALXN1210-aHUS-311 and ALXN1210-aHUS-312 (EudraCT Nr: 2016-002027-29) and ALXN1210-aHUS-312 (EudraCT Nr: 2016-002499-29)
Ultomiris ravulizumab treatment of Paroxysmal Nocturnal Haemoglobinuria in patients who completed the Open Label Extensions of the phase III studies ALXN1210-PNH-301 (EudraCT Nr: 2016-002025-11) or ALXN1210-PNH-303 (EudraCT Nr: 2017-002370-39)
Last updated on 23/02/2024