Voydeya - danicopan Voydeya is indicated as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia (see section 5.1 of the SmPC). European authorization on: 25/04/2024

ZYNYZ - retifanlimab ZYNYZ is indicated as monotherapy for the first line treatment of adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC) not amenable to curative surgery or radiation therapy. European authorization on: 24/04/2024

Filspari - Sparsentan Filspari is indicated for the treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion > 1.0 g/day (or urine protein-to-creatinine ratio ≥ 0.75 g/g, see section 5.1). European authorization on: 23/04/2024

Casgevy - exagamglogene autotemcel β thalassemia Casgevy is indicated for the treatment of transfusion dependent β thalassemia (TDT) in patients 12 years of age and older for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA) matched related HSC donor is not available. Sickle cell disease Casgevy is indicated for the treatment of severe sickle cell disease (SCD) in patients 12 years of age and older with recurrent vaso occlusive crises (VOCs) for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA) matched related HSC donor is not available. European authorization on: 12/02/2024

Skyclarys - omaveloxolone Skyclarys is indicated for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. European authorization on: 12/02/2024