TROMSØ, Norway--(BUSINESS WIRE)--Prophylix Pharma today announced that it will take its novel prophylactic NAITgam into Phase I/II trials in Q3 2015. NAITgam protects newborns against the rare but potentially crippling and fatal bleeding disorder called Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT). This condition occurs when a mother develops antibodies against her baby’s platelets. One of the most feared consequences of FNAIT is intracranial bleeding and this affects about 1000 pregnancies annually in the US and Europe. The decision follows FDA-approval of the company’s regulatory strategy to proceed with a greatly reduced clinical program. Prophylix will apply for Fast Track Designation and marketing authorization in 2018 is a realistic target.
NAITgam comprises specific IgG antibodies against HPA-1a manufactured from the plasma of rare donors recruited from European and US blood banks. The donors are women who previously have given birth to a child who suffered from FNAIT. The antibodies in the prophylactic drug remove fetal HPA-1a positive platelets from the mother’s circulation before her immune system has had time to develop antibodies against the fetal platelets.
All that is needed is a single injection within the first 6 hrs after an HPA-1a negative woman has given birth to an HPA-1a positive child. Dr. Jens Kjeldsen-Kragh, Chief Scientific Officer Prophylix Pharma AS comments, “It should also be noted that NAITgam is similar to Rh immune globulin, which for almost 50 years has proven safe and effective in almost eliminating a related condition, Hemolytic Disease of the Fetus and Newborn. We are delighted with the encouraging response from the FDA and will now file for an IND with a view to commencing clinical trials mid-2015.”
Thea Palmer, founder and trustee of naitbabies.org, a non-profit organization created to raise knowledge and awareness of FNAIT, also welcomed the news: “FNAIT is a devastating disease, currently without a cure, that may cause fetal death or lifelong disability. We were delighted to assist Prophylix with their recent submission to the FDA and the news that NAITgam will now go into clinical trials is extremely encouraging. We will be looking to help recruit plasma donors through our growing international network.”