PROFNAITProject reference: 305986
Funded under :
Development of a prophylactic treatment for the prevention of fetal/neonatal alloimmune thrombocytopenia (FNAIT)
Total cost:EUR 7 837 467,18
EU contribution:EUR 5 986 000
Topic(s):HEALTH.2012.2.4.4-1 - Preclinical and/or clinical development of substances with a clear potential as orphan drugs
Call for proposal:FP7-HEALTH-2012-INNOVATION-1See other projects for this call
Funding scheme:CP-FP - Small or medium-scale focused research project
Objective--The project aims to develop an anti HPA-1a immunoglobulin (IgG), Tromplate® that can prevent post delivery immunisation of the mother against the Human Platelet Antigen-1a (HPA-1a). This prophylactic treatment will prevent Fetal/Neonatal Alloimmune Thrombocytopenia (FNAIT) in the fetus/newborn in subsequent pregnancies.
FNAIT--FNAIT is a rare but potentially serious condition which affects about 4,000 fetuses and newborns a year in EU-27 and which may result in severe bleeding, intracranial hemorrhage, fetal death or lifelong disability. FNAIT may develop when the mother and the fetus have different platelet surface antigens, most commonly HPA-1a. Transferral of HPA-1a antigen from the fetus may cause immunisation of the mother and the anti-HPA-1a antibodies she develops may in turn destroy the platelets of subsequent HPA-1a positive fetuses/newborns and cause FNAIT. Today, no good treatment of FNAIT exists.
Rationale--Previously, it was believed that the pregnant woman develops antibodies early in the first pregnancy with the implication that FNAIT could not be prevented. However, a study conducted by the applicants of more than 100,000 pregnancies revealed that HPA-1a antibody formation most often takes place in association with or after delivery. Therefore, anti-HPA-1a IgG given to the mother shortly after birth should prevent her immune system from generating harmful anti-HPA-1a antibodies. This concept is analogous to the use of anti(D) for Rhesus D prophylaxis.
Activities--The project elements: manufacturing of Tromplate® using the process for producing anti(D); screening for HPA-1a negative and pregnant women and clinical Phase II/III studies investigating the efficacy and safety of Tromplate®.
Relevance to the work program−The prophylactic Tromplate® treatment has obtained orphan drug status in Europe. FNAIT represents a significant health care expense for the society and a great burden for the affected children and their families.
EU contribution: EUR 2 719 082,6
SYKEHUSVEGEN 23 FORSKNINGSPARKEN
Tel.: +45 4260 5260
EU contribution: EUR 78 389,98
Niels Bohrs Vej 30
Tel.: +45 99 32 11 25
EU contribution: EUR 4 107,9
LANDSTEINER STRASSE 5
Tel.: +49 6103 801 262
EU contribution: EUR 553 903,2
Tel.: +49 174 3377 345
EU contribution: EUR 287 401,44
104 22 STOCKHOLM
EU contribution: EUR 709 737,24
TEMPOVEJ 44 - 1st floor
EU contribution: EUR 165 613,26
Tel.: +46 46 2223207
Fax: +46 46 176006
EU contribution: EUR 237 439,22
KIRKEVEIEN 166 TARNBYGGET
EU contribution: EUR 108 326,4
HANSINE HANSENS VEG 14
Tel.: +47 48141286
EU contribution: EUR 585 193,06
EU contribution: EUR 18 900
291 89 KRISTIANSTAD
EU contribution: EUR 517 905,7