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 proteinDiseaseCandidate statusTerminated
candidates directed to target
Phase 1Phase 2Phase 3Approved
Factor VIIaFactor VII deficiency---44
Factor VIIIHemophilia A2-6252 (P2)35
Factor IXHemophilia B-14131 (P1)19
Factor XFactor X deficiency--112
Factor XIFactor XI deficiency---22
Factor XIIIFactor XIII deficiency--112
vWFvon Willebrand disease--156
Protein CProtein C deficiency---22
Antithrombin IIIAntithrombin deficiency1--78
FibrinogenFibrinogen deficiency---22
C1-esterase inhibitorHereditary angioedema---55
Alpha-1 proteinase inhibitor (α1-PI)α1-PI deficiency---42 (P2)6
GlucocerebrosidaseGaucher disease--131 (P1)5
Alpha-L-iduronidaseMucopolysaccharidosis I---11
Iduronate sulfataseMucopolysaccharidosis II---11
N-acetylgalactosamine-4-sulfataseMucopolysaccharidosis VI---11
N-acetylgalactosamine-6-sulfataseMucopolysaccharidosis IVA--1-1
Heparan sulfate sulfataseMucopolysaccharidosis IIIA-1--1
Alpha-galactosidase AFabry disease---21 (P2)3
Alpha-glucosidasePompe disease1--21 (P1), 1 (P2)5
Acid sphingomyelinaseNiemann-Pick type B disease1---1
Arylsulphatase AMetachromatic leukodystrophy----1 (P2)1
Lysosomal acid lipase (LAL)LAL deficiency-1--1
Sucrase-isomaltaseSucraseisomaltase deficiency---11
Adenosine deaminase (ADA)ADA deficiency---11
Insulin-like growth factor 1 (IGF-1)Primary IGF-1 deficiency---22
Alkaline phosphataseHypophosphatasia-1--1
Porphobilinogen deaminaseAcute intermittent porphyria----1 (P2)1
Total: 29 protein targets for 29 monogenic diseases 55158511121