EMA Orphan Drug Designations – July 2014

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Orphan Designations July

Click on the indication to find more details of each designation

Find the FDA’s orphan designation and approvals for July here.

EMA Orphan Drug Designations

Fabry Disease – Genzyme
Duchenne – NDA Group
Schnitzler syndrome – Les Laboratoires Servier
Cystic fibrosis – Vertex
Congenital factor VII deficiency – Richardson Associates Regulatory Affairs
Haemophilia A – Richardson Associates Regulatory Affairs
Haemophilia B – Richardson Associates Regulatory Affairs
Myelodysplastic syndromes – IDEA Innovative Drug European Associates
Apolipoprotein A-I deficiency – Cerenis Therapeutics
ATP-binding cassette transporter A1 deficiency – Cerenis Therapeutics
Bronchopulmonary dysplasia – Dr Philipp Heinrich Novak
Autosomal dominant polycystic liver disease – JJGConsultancy
Acute pancreatitis – BSV BioScience
Small cell lung cancer – DualTpharma
Acute myeloid leukaemia – Roche
Crigler-Najjar – Fondazione Telethon
Cutaneous T-cell lymphoma – Innate Pharma
Pyruvate kinase deficiency – Centro de Investigación Biomédica en Red
Ischaemia reperfusion injury – Caroline Systems
Large B-cell lymphoma – Roche
Mastocytosis – Medical university of Vienna
Paroxysmal nocturnal haemoglobinuria – Amyndas Pharmaceuticals
Biliary cirrhosis – Diamond BioPharm
Mitochondrial neurogastrointestinal encephalomyopathy – Vall d’Hebron Insitute of Research
Diffuse large B-cell lymphoma – Clinipace
Acute myeloid leukaemia – Clinipace
Pancreatic cancer – Synovo

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