Tables
- Table 1. MPRTs that have entered clinical development or received regulatory approval.
The number of candidates and development or approval status is listed by biological target. Plasma-derived products identified here may be approved in the EU at the national level and sold in individual EU countries. The full data set analyzed is in table S1.
Replacement protein Disease Candidate status Terminated
(phase)Total
candidates directed to targetPhase 1 Phase 2 Phase 3 Approved Factor VIIa Factor VII deficiency - - - 4 4 Factor VIII Hemophilia A 2 - 6 25 2 (P2) 35 Factor IX Hemophilia B - 1 4 13 1 (P1) 19 Factor X Factor X deficiency - - 1 1 2 Factor XI Factor XI deficiency - - - 2 2 Factor XIII Factor XIII deficiency - - 1 1 2 vWF von Willebrand disease - - 1 5 6 Protein C Protein C deficiency - - - 2 2 Antithrombin III Antithrombin deficiency 1 - - 7 8 Fibrinogen Fibrinogen deficiency - - - 2 2 C1-esterase inhibitor Hereditary angioedema - - - 5 5 Alpha-1 proteinase inhibitor (α1-PI) α1-PI deficiency - - - 4 2 (P2) 6 Glucocerebrosidase Gaucher disease - - 1 3 1 (P1) 5 Alpha-L-iduronidase Mucopolysaccharidosis I - - - 1 1 Iduronate sulfatase Mucopolysaccharidosis II - - - 1 1 N-acetylgalactosamine-4-sulfatase Mucopolysaccharidosis VI - - - 1 1 N-acetylgalactosamine-6-sulfatase Mucopolysaccharidosis IVA - - 1 - 1 Heparan sulfate sulfatase Mucopolysaccharidosis IIIA - 1 - - 1 Alpha-galactosidase A Fabry disease - - - 2 1 (P2) 3 Alpha-glucosidase Pompe disease 1 - - 2 1 (P1), 1 (P2) 5 Acid sphingomyelinase Niemann-Pick type B disease 1 - - - 1 Alpha-mannosidase Alpha-mannosidosis - 1 - - 1 Arylsulphatase A Metachromatic leukodystrophy - - - - 1 (P2) 1 Lysosomal acid lipase (LAL) LAL deficiency - 1 - - 1 Sucrase-isomaltase Sucraseisomaltase deficiency - - - 1 1 Adenosine deaminase (ADA) ADA deficiency - - - 1 1 Insulin-like growth factor 1 (IGF-1) Primary IGF-1 deficiency - - - 2 2 Alkaline phosphatase Hypophosphatasia - 1 - - 1 Porphobilinogen deaminase Acute intermittent porphyria - - - - 1 (P2) 1 Total: 29 protein targets for 29 monogenic diseases 5 5 15 85 11 121
Additional Files
- Supplementary Material for:
Protein Replacement Therapies for Rare Diseases: A Breeze for Regulatory Approval?
Jennifer A. Gorzelany* and Mark P. deSouza*Corresponding author. E-mail: jen{at}laurelwoodbio.com
Published 27 March 2013, Sci. Transl. Med. 5, 178fs10 (2013)
DOI: 10.1126/scitranslmed.3005007This PDF file includes:
- Methods
- Table S1
