All resolutions concerning metabolic medicinal products

Ivacaftor (11) Kalydeco® Vertex Pharmaceuticals Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Indication for non-quantifiable additional benefit
Asfotase alfa (2, reassessment >€50m) Strensiq® Alexion Pharma Germany GmbH Metabolic diseases METABOL ORPHAN Hypophosphatasia (HPP) 0.8% Indication for non-quantifiable additional benefit
Burosumab (2, reassessment) Crysvita® Kyowa Kirin GmbH Metabolic diseases METABOL ORPHAN Hypophosphatemia 100% Indication for non-quantifiable additional benefit
Ivacaftor (10, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Indication for non-quantifiable additional benefit
Ivacaftor (9, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Indication for minor additional benefit
Ivacaftor (8, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Indication for non-quantifiable additional benefit
Ivacaftor (7, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Indication for non-quantifiable additional benefit
Ivacaftor (6, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% no additional benefit
Ivacaftor (5, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% no additional benefit
Ivacaftor (4, reassessment >€50m) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 86% Indication for considerable additional benefit
Volanesorsen Waylivra® Akcea Therapeutics Germany GmbH Metabolic diseases METABOL ORPHAN Familial chylomicronemia syndrome (FCS) 100% Indication for non-quantifiable additional benefit
Pegvaliase Palynziq® BioMarin International Limited Metabolic diseases METABOL ORPHAN Phenylketonuria (PKU) 100% Indication for non-quantifiable additional benefit
Nintedanib (3, reassessment >€50m, Ofev®) Ofev® Boehringer Ingelheim Pharma GmbH & Co. KG Respiratory diseases METABOL ORPHAN Idiopathic pulmonary fibrosis 100% Indication for considerable additional benefit
Lumacaftor / Ivacaftor (3) Orkambi® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL Cystic fibrosis (CF) 100% Indication for non-quantifiable additional benefit
Glycerolphenylbutyrat (2) Ravicti® Swedish Orphan Biovitrum GmbH Metabolic diseases METABOL ORPHAN Urea cycle disorders (UCD) 100% Indication for non-quantifiable additional benefit
Tezacaftor / Ivacaftor Symkevi® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 91% Hint for considerable additional benefit
Patisiran Onpattro® Alnylam Germany GmbH Metabolic diseases METABOL ORPHAN Amyloidosis 100% considerable additional benefit
Inotersen Tegsedi® Akcea Therapeutics Germany GmbH Metabolic diseases METABOL ORPHAN Amyloidosis 100% non-quantifiable additional benefit
Metreleptin Myalepta® Aegerion Pharmaceuticals GmbH Metabolic diseases METABOL ORPHAN Lipodystrophy 100% Hint for non-quantifiable additional benefit
Vestronidase alfa Mepsevii® Ultragenyx Germany GmbH Metabolic diseases METABOL ORPHAN Mucopolysaccharidosis 100% non-quantifiable additional benefit
Velmanase alfa Lamzede® Chiesi GmbH Metabolic diseases METABOL ORPHAN α mannosidosis 100% Indication for non-quantifiable additional benefit
Burosumab Crysvita® Kyowa Kirin GmbH Metabolic diseases METABOL ORPHAN Hypophosphatemic rickets (XLH) 100% Hint for non-quantifiable additional benefit
Patiromer Veltassa® Fresenius Medical Care Nephrologica Deutschland GmbH Metabolic diseases METABOL Hyperkalemia 100% no additional benefit
Glycerolphenylbutyrat Ravicti® Swedish Orphan Biovitrum GmbH Metabolic diseases METABOL ORPHAN Urea cycle disorders (UCD) 100% Hint for non-quantifiable additional benefit
Lumacaftor / Ivacaftor (2) Orkambi® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL Cystic fibrosis (CF) 100% Indication for non-quantifiable additional benefit
Elosulfase alfa (2, reassessment) Vimizim® BioMarin Deutschland GmbH Metabolic diseases METABOL ORPHAN Mucopolysaccharidosis 100% minor additional benefit
Nusinersen Spinraza® Biogen GmbH Diseases of the nervous system METABOL ORPHAN Spinal muscular atrophy (SMA) 10% major additional benefit
Cerliponase alfa Brineura® BioMarin Deutschland GmbH Metabolic diseases METABOL ORPHAN Neuronal ceroid lipofuscinosis (CNL2) 100% non-quantifiable additional benefit
Etelcalcetid Parsabiv® Amgen GmbH Metabolic diseases METABOL Secondary hyperparathyroidism (sHPT) 100% no additional benefit
Obeticholsäure Ocaliva® Intercept Pharma Deutschland GmbH Diseases of the digestive system METABOL ORPHAN Hepatic cirrhosis 100% non-quantifiable additional benefit
Teduglutid (2) Revestive® Shire Deutschland GmbH Diseases of the digestive system METABOL ORPHAN Short bowel syndrome 100% non-quantifiable additional benefit
Afamelanotid Scenesse® Clinuvel Ltd. Metabolic diseases METABOL ORPHAN Erythropoietic protoporphyria (EPP) 100% non-quantifiable additional benefit
Ivacaftor (3) Kalydeco® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 75% Hint for minor additional benefit
Lumacaftor / Ivacaftor Orkambi® Vertex Pharmaceuticals (Europe) Limited Metabolic diseases METABOL Cystic fibrosis (CF) 100% Hint for considerable additional benefit
Sebelipase alfa Kanuma® Synageva BioPharma Limited Metabolic diseases METABOL ORPHAN Lysosomal lipase acid deficiency (LAL) 100% non-quantifiable additional benefit
Asfotase alfa Strensiq® Alexion Europe SAS Metabolic diseases METABOL ORPHAN Hypophosphatasia (HPP) 50% Hint for non-quantifiable additional benefit
Eliglustat Cerdelga® Genzyme GmbH Metabolic diseases METABOL ORPHAN Morbus Gaucher type 1 100% non-quantifiable additional benefit
Nintedanib (2, Ofev®) Ofev® Boehringer Ingelheim Pharma GmbH & Co. KG Respiratory diseases METABOL ORPHAN Idiopathic pulmonary fibrosis 100% Hint for minor additional benefit
Pasireotid (2) Signifor® Novartis Pharma GmbH Metabolic diseases METABOL ORPHAN Acromegalia 100% minor additional benefit
Ataluren Translarna® PTC Therapeutics International Limited Diseases of the musculoskeletal diseases METABOL ORPHAN Duchenne muscle dystrophia 100% minor additional benefit
Alipogentiparvovec Glybera® Chiesi GmbH Metabolic diseases METABOL ORPHAN Lipoprotein deficiency 100% non-quantifiable additional benefit
Teduglutid Revestive® NPS Pharma Germany GmbH Diseases of the digestive system METABOL ORPHAN Malabsorption syndrome 100% minor additional benefit
Ivacaftor (2) Kalydeco® Vertex Pharmaceuticals Limited Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 100% Hint for minor additional benefit
Elosulfase alfa Vimizim® BioMarin Deutschland GmbH Metabolic diseases METABOL ORPHAN Mucopolysaccharidosis / Morquio A syndrome 100% minor additional benefit
Cholsäure Orphacol® Laboratoires CTRS Metabolic diseases METABOL ORPHAN Heritage dysbalance of primary bilic acid synthesis 100% non-quantifiable additional benefit
Ivacaftor Kalydeco® Vertex Pharmaceuticals GmbH Metabolic diseases METABOL ORPHAN Cystic fibrosis (CF) 84% considerable additional benefit
Pasireotid Signifor® Novartis Pharma GmbH Metabolic diseases METABOL ORPHAN Dysfunction of pituitary gland 100% minor additional benefit
Tafamidis Vyndaqel® Pfizer Pharma GmbH Other METABOL ORPHAN Amyloidosis 100% minor additional benefit
Pirfenidon Esbriet® InterMune Deutschland GmbH Respiratory diseases METABOL ORPHAN Idiopathic pulmonary fibrosis 100% non-quantifiable additional benefit