Cardiovascular diseases

Antithrombotic medications

• Andexanet alfa

• Apixaban

  • Pevention of venous thromboembolic events (VTE) in adult patients who have undergone elective hip or knee replacement surgery. Prevention of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation (NVAF), with one or more risk factors, such as prior stroke or transient ischaemic attack (TIA); age ≥ 75 years; hypertension; diabetes mellitus; symptomatic heart failure (NYHA Class ≥ II). Treatment of deep vein thrombosis (DVT) and pulmonary embolism (PE), and prevention of recurrent DVT and PE in adults.
  • Extension to the existing indication to include treatment of venous thromboembolism (VTE) and prevention of recurrent VTE in children, associated with the addition of new pharmaceutical forms and new strengths (0.15mg granules in capsules for opening, as well as 0.5mg, 1.5mg and 2mg coated granules in sachet).
• Asundexian
• Betrixaban
• Ciraparantag
• Dabigatran ^G
• Dabigatran etexilaat ^IND
• Dabigatran etexilate ^IND
• Edaravone / dexborneol

• Macitentan

  • Extension of Indication to include treatment of patients with inoperable chronic thromboembolic pulmonary hypertension (CTEPH)
• Proteïne C ^IND

• Ravulizumab

  • Extension of Indication to include the treatment of patients with atypical hemolytic uremic syndrome (aHUS).

• Rivaroxaban

  • Treatment of venous thromboembolism (VTE) and prevention of VTE recurrence in children and adolescents aged less than 18 years and weighing from 30 kg to 50 kg after at least 5 days of initial parenteral anticoagulation treatment.
  • In combinatie met acetylsalicylzuur, voor de preventie van atherotrombotische complicaties bij volwassen patiënten met stabiel coronair arterieel vaatlijden (CAV) of symptomatisch perifeer arterieel vaatlijden (PAV), met een verhoogd risico op ischemische events.
• Tenecteplase ^IND

Cardiovascular diseases

• Prasugrel

Heart failure

• Autologous stem cells
• Ularitide

Hemostasis

• Human fibrinogen / Human thrombin

Hemostasis promoting medication

• Damoctocog alfa pegol

  • Behandeling en preventie van bloedingen bij eerder behandelde patiënten (previously treated patients, PTP's) van 12 jaar en ouder met hemofilie A (aangeboren factor VIII deficiëntie).
  • Treatment and prophylaxis of bleeding in previously treated patients ≥ 7 years of age with haemophilia A (congenital factor VIII deficiency).

• Emicizumab

  • Hemlibra is indicated for Routine prophylaxis of bleeding episodes in patients with haemophilia A without factor VIII inhibitors who have moderate disease with severe bleeding phenotype.
  • Hemlibra is geïndiceerd voor de routinematige profylaxe van bloedingen bij patiënten met hemofilie A zonder remmers tegen factor VIII.
  • Hemlibra is geïndiceerd voor de routineprofylaxe van bloedingen bij patiënten met hemofilie A met remmers tegen factor VIII. Hemlibra kan bij alle leeftijdsgroepen worden gebruikt.

• Eptacog alfa (activated)

  • Preventie en behandeling van bloedingen bij operaties of invasieve ingrepen bij: 1) overgeërfde hemofilie met remmers tegen stollingsfactor VIII of IX (> 5 BU) of bij een verwachte hoge anamnestische respons op factor VIII of factor IX 2) verworven hemofilie 3) overgeërfde factor VII-deficiëntie 4) ziekte van Glanzmann (trombasthenie) met eerdere of huidige ongevoeligheid voor bloedplaatjestransfusie of wanneer bloedplaatjes niet direct beschikbaar zijn.
  • Extension of Indication to extend patient population of NovoSeven for use in patients with Glanzmann’s thrombasthenia with or present refractoriness to platelet transfusions, or where platelets are not readily available.
• Eptacog beta (activated)
• Etranacogene dezaparvovec
• FLT180a
• Fibrinogen
• Fidanacogene elaparvovec
• Nonacog beta pegol ^IND
• Peboctocogene camaparvovec
• Rurioctocog alfa pegol

• Turoctocog alfa pegol

  • Treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia A (congenital factor VIII deficiency).
  • Extension of indication to include children below 12 years of age for treatment and prophylaxis of bleeding with haemophilia A for Esperoct, including previously untreated patients (PUPs).
• Valoctocogene roxaparvovec

• Vonicog alfa

  • Extension of indication for the prevention and treatment of haemorrhage or surgical bleeding in adults (age 18 years and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or contraindicated.
  • VEYVONDI is geïndiceerd voor volwassenen (18 jaar en ouder) met de ziekte van von Willebrand, wanneer behandeling met desmopressine (DDAVP) alleen niet doeltreffend is of niet geïndiceerd is voor de - behandeling van hemorragie en chirurgische bloeding - preventie van chirurgische bloeding. VEYVONDI mag niet worden gebruikt bij de behandeling van hemofilie A.
  • Treatment of haemorrhage in children (aged less than 18 years) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or contraindicated.

Lipid-lowering medications

• Alirocumab

  • Extension of indication to include treatment of paediatric patients 8 years of age and older with heterozygous familial hypercholesterolemia (HeFH) as an adjunct to diet, alone or in combination with other LDL -C lowering therapies.
  • Indicated in adults with established atherosclerotic cardiovascular disease to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors: • in combination with the maximum tolerated dose of a statin with or without other lipid-lowering therapies or, • alone or in combination with other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contraindicated.
• Bempedoic acid

• Bempedoic acid / ezetimibe

  • Extension of indication to include treatment of adults with established or at high risk for atherosclerotic cardiovascular disease to reduce cardiovascular risk.
  • Nustendi is indicated in adults with primary hypercholesterolaemia (heterozygous familial and non-familial) or mixed dyslipidaemia, as an adjunct to diet: in combination with a statin in patients unable to reach LDL-C goals with the maximum tolerated dose of a statin in addition to ezetimibe, alone in patients who are either statin-intolerant or for whom a statin is contraindicated, and are unable to reach LDL-C goals with ezetimibe alone or in patients already being treated with the combination of bempedoic acid and ezetimibe as separate tablets with or without statin.
• Enlicitide decanoate

• Evinacumab

  • Extension of indication to include the treatment of paediatric patients with homozygous familial hypercholesterolaemia (HoFH) aged 5 years and older
  • Evkeeza is indicated as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adult and adolescent patients aged 12 years and older with homozygous familial hypercholesterolaemia (HoFH).
  • Extension of indication for EVKEEZA to include the treatment of paediatric patients with homozygous familial hypercholesterolaemia aged 6 months to less than 5 years.

• Evolocumab

  • Adults with established atherosclerotic cardiovascular disease (myocardial infarction, stroke or peripheral arterial disease) to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors
  • Extension of indication to include one new paediatric indication in paediatric patients aged 10 years and over with heterozygous and homozygous familial hypercholesterolaemia as an adjunct to diet, in combination with a statin or statin with other lipid-lowering therapies in patients unable to reach LDL‑C goals with the maximum tolerated dose of a statin or, alone or in combination with other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contraindicated.

• Inclisiran

  • Leqvio is indicated in adults with primary hypercholesterolaemia (heterozygous familial and non‑familial) or mixed dyslipidaemia, as an adjunct to diet: in combination with a statin or statin with other lipid‑lowering therapies in patients unable to reach LDL‑C goals with the maximum tolerated dose of a statin, or alone or in combination with other lipid‑lowering therapies in patients who are statin‑intolerant, or for whom a statin is contraindicated.
  • Extension of indication to include the treatment of paediatric patients aged 12 to less than 18 years with homozygous or heterozygous familial hypercholesterolaemia (HoFH/HeFH).

• Lerodalcibep

  • Is indicated in adults with primary hypercholesterolaemia (heterozygous familial (HeFH) and non-familial) or mixed dyslipidaemia as an adjunct to diet.
  • Cardiovascular disorder in adults and elderly on a stable diet and oral LDL-C lowering drug therapy with history of CVD, monotherapy for add-on treatment.
• Lomitapide ^IND
• Obicetrapib
• Obicetrapib / ezetimibe

• Olezarsen

  • Tryngolza is indicated as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS).
  • Olezarsen monotherapy for add-on treatment of severe hypertriglyceridemia in adults and elderly.
• Pegozafermine
• Pelacarsen
• Plozasiran
• Pravastatine / ​fenofibraat ^IND

• Volanesorsen

  • Waylivra is geïndiceerd als een aanvulling op het dieet bij volwassen patiënten met genetisch bevestigd familiair chylomicronemiesyndroom (FCS) met een hoog risico van pancreatitis bij wie de respons op dieet en triglyceriden verlagende behandeling onvoldoende was.

Other medication for cardiovascular diseases

• Acoramidis
• Aficamten
• Allogenic immunomodulatory progenitor cells
• Angiotensin II
• Apabetalon
• Baxdrostat

• Canakinumab

  • Secundaire preventie van cardiovasculaire incidenten bij patiënten met een doorgemaakt myocard infarct en een residuaal inflammatoir risico.

• Dapagliflozine

  • Forxiga is indicated in adults for the treatment of symptomatic chronic heart failure with reduced ejection fraction.
  • dapagliflozin is indicated in adults for the treatment of symptomatic chronic heart failure
• Emiplacel

• Empagliflozine

  • Jardiance is indicated in adults for the treatment of symptomatic chronic heart failure with reduced ejection fraction.
  • Chronic Heart Failure With Preserved Ejection Fraction (HFpEF, LVEF >40%).
• Etripamil
• Ferric carboxymaltose
• Fibrinogen / Thrombin ^IND

• Finerenon

  • Extension of indication to include the treatment of symptomatic chronic heart failure with left ventricular ejection fraction (LVEF) ≥ 40% in adults.
• Glutathione Sodium
• Icosapent ethyl
• Lorundrostat

• Macitentan

  • Fontan-palliated pulmonary hypertension in adults.
  • Opsumit, as monotherapy or in combination, is indicated for the long-term treatment of pulmonary arterial hypertension (PAH) in paediatric patients aged less than 18 years and bodyweight ≥ 40 kg with WHO Functional Class (FC) II to III.
• Macitentan / tadalafil

• Mavacamten

  • Camzyos is indicated for the treatment of symptomatic (New York Heart Association, NYHA, class II III) obstructive hypertrophic cardiomyopathy (oHCM) in adult patients.
  • Mavacamten for treatment of symptomatic (NYHA class II or III) non-obstructive hypertrophic cardiomyopathy in adults.
• Neoatricon (dopamine)
• Omecamtiv mecarbil
• Regadenoson ^IND
• Rexlemestrocel-L

• Rilonacept

  • In paediactric and adult patients with Recurrent Pericarditis ≥12 years
  • Treatment of idiopathic pericarditis.
• Selatogrel
• Seralutinib

• Sotagliflozine

  • Cardiovascular Events in Patients With Type 2 Diabetes Post Worsening Heart Failure
• Tafamidis ^IND

• Ticagrelor

  • Extension of indication to include, in co-administration with acetylsalicylic acid (ASA), the prevention of stroke in adult patients with acute ischaemic stroke or transient ischaemic attack (TIA) in the previous 24 hours.
  • Extension of indication to include, in co administration with acetylsalicylic acid (ASA), the prevention of atherothrombotic events in adult patients with coronary artery disease (CAD) and type 2 diabetes mellitus (T2DM) without a history of myocardial infarction who have undergone percutaneous coronary intervention (PCI).

• Tirzepatide

  • Extension of indication to include treatment of symptomatic chronic heart failure with preserved ejection fraction (HFpEF) in adults with obesity.

• Treprostinil

  • Treatment of adults with chronic thromboembolic pulmonary hypertension (CTEPH) who cannot have surgery, and in patients whose CTEPH continues or has come back after surgery.
  • Behandeling van idiopatische of erfelijke primaire pulmonale arteriële hypertensie bij matig-ernstig hartfalen (NYHA-klasse III).
• Udenafil

• Valsartan / sacubitril

  • Post acute Myocardial Infarction
  • Entresto is indicated in adult patients for treatment of symptomatic chronic heart failure with ejection fraction below 55/60%
  • Extension of indication to include treatment of children and adolescents aged one year or older with chronic heart failure with left ventricular systolic dysfunction.

• Vericiguat

  • Verquvo is indicated for the treatment of symptomatic chronic heart failure in adult patients with reduced ejection fraction who are stabilised after a recent decompensation event requiring IV therapy
  • Extension of indication for treatment of symptomatic chronic Heart failure with reduced ejection fraction in adults and elderly who have not had a recent hospitalization for heart failure or need for outpatient intravenous diuretics.
  • Verquvo is indicated for the treatment of symptomatic chronic heart failure in adult patients with reduced ejection fraction who are stabilised after a recent decompensation event requiring IV therapy

• Vutrisiran

  • Amvuttra is indicated for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
• Zalunfiban
• Ziltivekimab

Other non-oncological hematological medications

• Anselamimab
• Apadamtase alfa
• Aprocitentan
• Avatrombopag

• Benralizumab

  • Extension of indication to include treatment of adults and adolescents with hypereosinophilic syndrome (HES).
• Bentracimab

• Betibeglogene autotemcel

  • Zynteglo is geïndiceerd voor gebruik bij patiënten in de leeftijd van 12 jaar en ouder met transfusie-afhankelijke β-thalassemie (transfusion-dependent β-thalassaemia - TDT) die geen β0/β0-genotype hebben, voor wie transplantatie van hematopoëtische stamcellen (HSC) gepast is, maar geen humaan leukocytenantigeen (HLA)-compatibele gerelateerde HSC-donor beschikbaar is.
  • Behandeling van patiënten jonger dan 12 jaar met transfusie-afhankelijke β-thalassemie (TDT) die geen β0/β0-genotype hebben en voor wie hematopoïetische stamceltransplantatie (HSC) gepast is, maar voor wie geen humaan leukocyt antigeen (HLA)-compatibele verwante HSC-donor beschikbaar is.
  • Behandeling van patiënten met transfusie-afhankelijke β-thalassemie (TDT) die een β0 mutatie op beide allelen van het β-globuline (HBB) hebben en voor wie hematopoïetische stamceltransplantatie (HSC) gepast is, maar voor wie geen humaan leukocyt antigeen (HLA)-compatibele verwante HSC-donor beschikbaar is
• Caplacizumab

• Concizumab

  • Routine prophylaxis of bleeding in patients with; haemophilia A (congenital factor VIII deficiency) with FVIII inhibitors and of 12 years of age or more; haemophilia B (congenital factor IX deficiency) with FIX inhibitors and of 12 years of age or more.
  • Alhemo is indicated for routine prophylaxis of bleeding in patients 12 years of age or more with severe haemophilia A (congenital factor VIII deficiency, FVIII <1%) without FVIII inhibitors and moderate/severe haemophilia B (congenital factor IX deficiency, FIX ≤2%) without FIX inhibitors
• Crizanlizumab
• Crovalimab
• Danicopan
• Daprodustat

• Deferasirox

  • Geïndiceerd voor de behandeling van: 1) chronische ijzerstapeling als gevolg van veelvuldige bloedtransfusies bij patiënten van 6 jaar en ouder met bètathalassemie major. 2) chronische ijzerstapeling als gevolg van bloedtransfusies wanneer deferoxamine behandeling gecontra-indiceerd of inadequaat is in de volgende patiëntengroepen: pediatrische patiënten met bètathalassemie major met ijzerstapeling als gevolg van veelvuldige bloedtransfusies in de leeftijd van 2 tot 5 jaar, volwassen en pediatrische patiënten met bètathalassemie major met ijzerstapeling als gevolg van occasionele bloedtransfusies (<7 ml/kg/maand rode bloedcellen) in de leeftijd van 2 jaar en ouder en volwassen en pediatrische patiënten met andere anemieën in de leeftijd van 2 jaar en ouder. 3) chronische ijzerstapeling waarbij chelatietherapie noodzakelijk is en wanneer deferoxaminebehandeling gecontra-indiceerd of inadequaat is bij patiënten in de leeftijd van 10 jaar en ouder met niet-transfusie-afhankelijke thalassemiesyndromen.
  • Geïndiceerd voor de behandeling van: 1) chronische ijzerstapeling als gevolg van veelvuldige bloedtransfusies bij patiënten van 6 jaar en ouder met bètathalassemie major. 2) chronische ijzerstapeling als gevolg van bloedtransfusies wanneer deferoxamine behandeling gecontra-indiceerd of inadequaat is in de volgende patiëntengroepen: pediatrische patiënten met bètathalassemie major met ijzerstapeling als gevolg van veelvuldige bloedtransfusies in de leeftijd van 2 tot 5 jaar, volwassen en pediatrische patiënten met bètathalassemie major met ijzerstapeling als gevolg van occasionele bloedtransfusies (<7 ml/kg/maand rode bloedcellen) in de leeftijd van 2 jaar en ouder en volwassen en pediatrische patiënten met andere anemieën in de leeftijd van 2 jaar en ouder. 3) chronische ijzerstapeling waarbij chelatietherapie noodzakelijk is en wanneer deferoxaminebehandeling gecontra-indiceerd of inadequaat is bij patiënten in de leeftijd van 10 jaar en ouder met niet-transfusie-afhankelijke thalassemiesyndromen.
• Denecimig

• Eculizumab

  • Paroxysmal nocturnal haemoglobinuria
  • Paroxysmal nocturnal haemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS)
  • Paroxysmal nocturnal haemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS)
  • Paroxysmal nocturnal haemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).
• Efanesoctocog alfa ^IND

• Efgartigimod

  • Idiopathic thrombocytopenic purpura (ITP)

• Eltrombopag

  • Extension of indication to include treatment of adult patients with primary immune thrombocytopenia (ITP) who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) irrespective of time since initial diagnosis
  • Eltrombopag in combinatie met standaard immunosuppressieve therapie voor de behandeling van patiënten met ernstige aplastische anemie (SAA) als eerstelijnstherapie.
  • Extension of indication to include second-line treatment of paediatric patients aged 2 years and above with acquired severe aplastic anaemia (SAA).
• Eptacog alfa ^IND
• Etavopivat
• Exagamglogene autotemcel
• Ferrimaltol ^IND
• Ferumoxytol
• Giroctocogene fitelparvovec
• Horse anti-human T lymphocyte immunoglobulin

• Hydroxycarbamide

  • Geïndiceerd voor de preventie van recidiverende, pijnlijke vaso-occlusieve crises waaronder het 'acute chest syndrome' bij volwassenen, adolescenten en kinderen ouder dan 2 jaar met symptomatische sikkelcelziekte.
  • Extension of indication to include treatment of severe chronic anemia (haemoglobin level <6 g/dL or <7 g/dL with poor clinical or functional tolerance) in adults, adolescents and children older than 2 years suffering from sickle cell syndrome.
  • Xromi is indicated for the prevention of vaso-occlusive complications of Sickle Cell Disease in patients over 9 months of age
• Inclacumab

• Iptacopan

  • Treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) to treat haemolytic anaemia.
• L-Glutamine

• Luspatercept

  • Adult patients with Beta-thalassemia, who are transfusion dependent.

• Marstacimab

  • Routinematige profylactische behandeling van bloedingen bij patiënten twaalf jaar en ouder met ernstige hemofilie A en B zonder remmers
  • Routinematige profylactische behandeling van bloedingen bij patiënten twaalf jaar en ouder met ernstige hemofilie A en B met remmers

• Mepolizumab

  • Adolescent and Adult Subjects With Severe Hypereosinophilic Syndrome (HES) refractory to current standard of care.
• Methoxy polyethylene glycol-epoetin beta ^IND

• Mitapivat

  • Pyrukynd is indicated in adults for the treatment of anaemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassaemia
  • Pyruvate kinase deficiency in transfusion-dependent and transfusion-independent adults
  • Mitapivat monotherapy for treatment of pyruvate kinase deficiency anaemia in infants and toddlers over 1 year of age, children and adolescents who are not regularly receiving blood transfusion.
• Mozafancogene autotemcel

• Pegcetacoplan

  • Paroxysmal nocturnal haemoglobinuria (PNH)
  • Aspaveli is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria who have haemolytic anaemi.
• Pozelimab

• Ravulizumab

  • Treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) in patients with haemolysis with clinical symptom(s) indicative of high disease activity and in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months.
  • Uitbreiding van de behandeling naar pediatrische patiënten met een lichaamsgewicht van 10kg of meer met paroxismale nachtelijke hemoglobinurie (PNH): - bij patiënten met hemolyse met klinische symptomen die wijzen op een hoge ziekteactiviteit. - bij patiënten die klinisch stabiel zijn nadat ze ten minste de afgelopen 6 maanden behandeld zijn met eculizumab.
• Rilzabrutinib
• Rivipansel sodium

• Romiplostim

  • Extension of indication to add the use of romiplostim in adult patients who have had ITP for ≤ 12 months and who have had an insufficient response to corticosteroids or immunoglobulins.
• Roxadustat
• Rusfertide
• Thrombomodulin alfa

• UM171

  • Patients With Sickle-Cell Disease
• Vadadustat
• Voxelotor

Unknown

• Anacetrapib