Final Report Summary - PROFNAIT (Development of a prophylactic treatment for the prevention of fetal/neonatal alloimmune thrombocytopenia (FNAIT)) Executive Summary:PROFNAIT is a European Union-funded consortium (www.profnait.eu) consisting of eleven Northern European hospitals, blood banks and companies, which aim to prevent 1,000 cases per year in EU and the US of potentially lethal or disabling internal bleeding in at-risk babies due to fetal and neonatal alloimmune thrombocytopenia (FNAIT). In spite of FNAIT being a rare condition, 13 leading EU and US medical societies in the fields of obstetrics and gynaecology have expressed their support of the PROFNAIT project and confirmed the unmet medical need in FNAIT.The FNAIT prophylaxis PROFNAIT set out to develop, tentatively named NAITgam, must be manufactured from human plasma donated by women who has developed a certain antibody (immunoglobulin). Many of these women are mothers to FNAIT babies and the partners in PROFNAIT collaborated with the patient organization Naitbabies on recruiting their members as plasma donors. In parallel, thousands of existing plasma donors were screened to identify even more women who could donate this rare type of plasma. The identified donors were subsequently referred to one of the blood banks participating in PROFNAIT or to one of 50 authorized plasma collection sites in the US. In total, close to 1,000 plasma donations were made and a total of 650 litres of plasma were collected. It is estimated that more than 10,000 doses of NAITgam can be manufactured from the collected plasma.The manufacturing and clinical development of NAITgam were discussed with the European Medicines Agency (EMA), the US Food and Drug Administration (FDA) and the German Paul-Ehrlich-Institut (PEI) to ensure agreement on fundamental safety and efficacy questions. The agencies expressed strong support of the project and agreed that the generally excellent safety of plasma-derived hyperimmune drugs like NAITgam and the challenge of identifying study subjects in this orphan condition justified a relatively limited development program consisting of a phase 0 study control study, a phase 1/2 dose-finding study and a single pivotal phase 3 study.PROFNAIT consortium successfully completed the phase 0 study, which aimed to determine the natural rate of elimination transfused platelets. In the phase 1/2, it will be investigated if NAITgam is able to accelerate the elimination of a certain type of platelets, which simulate incompatible fetal platelets, and thereby presumably prevent the mother from being immunized and subsequently attack her foetus’ platelets. The phase 1/2 study will also be used to establish the dose to be used in phase 3.Many of the European academic and medical experts in FNAIT were partners in PROFNAIT, and a numerous peer-reviewed articles were published during the project. The project was also strengthened on the business side by the completion of US market research, a comprehensive health-economic study geared towards UK NICE and US Medicaid and a health-policy “roadmap” project that mapped the US stakeholders involved with the publication and implementation of new clinical guidelines on general testing pregnant women to identify those at risk of FNAIT and in need of NAITgam.PROFNAIT created a strong basis for further development of NAITgam and future prevention of FNAIT, and many of the partners will continue to be involved with the project after the completion of PROFNAIT.Project Context and Objectives:Please see the attached final publishable summaryProject Results:Please see the attached final publishable summaryPotential Impact:Please see the attached final publishable summaryList of Websites:http://www.profnait.eu/Address to the PROFNAIT project coordinators in Tromsø:Prophylix Pharma ASForskningsparkenNO-9294 TromsøNorwayEmail: plasma@prophylixpharma.com Related documents final1-profnait-final-report.pdf
