Cardiovascular diseases

Antithrombotic medications

• Asundexian
• Edaravone / dexborneol

Hemostasis promoting medication

• Damoctocog alfa pegol

  • Treatment and prophylaxis of bleeding in previously treated patients ≥ 7 years of age with haemophilia A (congenital factor VIII deficiency).
• Etranacogene dezaparvovec

• Turoctocog alfa pegol

  • 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

  • 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

• Enlicitide decanoate

• Evinacumab

  • Extension of indication for EVKEEZA to include the treatment of paediatric patients with homozygous familial hypercholesterolaemia aged 6 months to less than 5 years.

• Inclisiran

  • 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

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

Other medication for cardiovascular diseases

• Acoramidis
• Aficamten
• Baxdrostat
• Etripamil

• Finerenon

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

• Mavacamten

  • Mavacamten for treatment of symptomatic (NYHA class II or III) non-obstructive hypertrophic cardiomyopathy in adults.
• Neoatricon (dopamine)
• Selatogrel
• Seralutinib

• Tirzepatide

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

• 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.

• 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

• Benralizumab

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

• Concizumab

  • 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
  • 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.
• Crovalimab
• Danicopan
• Denecimig

• Eltrombopag

  • Extension of indication to include second-line treatment of paediatric patients aged 2 years and above with acquired severe aplastic anaemia (SAA).
• Etavopivat
• Exagamglogene autotemcel
• Ferrimaltol ^IND
• Giroctocogene fitelparvovec
• Inclacumab

• Marstacimab

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

• Mitapivat

  • 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.
  • Pyrukynd is indicated in adults for the treatment of anaemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassaemia
• Mozafancogene autotemcel

• Pegcetacoplan

  • Aspaveli is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria who have haemolytic anaemi.
• Pozelimab
• Rilzabrutinib
• Rusfertide