Emergent BioSolutions to Manufacture Prophylix AS Developmental Drug for Fetal-Neonatal Alloimmune Thrombocytopenia.
May 21, 2019
Prophylix AS today announced a manufacturing collaboration with Emergent BioSolutions Inc. (NYSE:EBS) whereby Emergent will manufacture Prophylix’s developmental drug NAITgam [Human Platelet Antigen 1a Immunoglobulin (anti-HPA-1a)]. The collaboration will leverage Emergent’s proven platform for manufacturing of plasma-derived hyperimmune drug products to enable the development of the first drug for the prevention of the rare but potentially chronically disabling or life-threatening pediatric condition fetal-neonatal alloimmune thrombocytopenia (FNAIT). Today, no drug is approved for the prevention or treatment of FNAIT.
“Emergent is pleased to assist Prophylix AS with the advancement of their NAITgam program,” said Sean Kirk, EVP, Manufacturing and Technical Operations and Contract Development and Manufacturing Organization (CDMO) Business Unit Head at Emergent. “Our proven technology and rich history of hyperimmune development is a unique capability that we can leverage to help make an impact in addressing this unmet medical need. This is consistent with and supportive of Emergent’s mission – to protect and enhance life.”
“The agreement with Emergent marks a turning point in Prophylix’s efforts to develop NAITgam for the prevention of FNAIT,” said Søren Weis Dahl, CEO, Prophylix AS. “FNAIT is a rare but potentially devastating pediatric condition, and Emergent’s platform is ideally suited for manufacturing of this niche product. We are looking forward to a long and mutually beneficially partnership. Now that the manufacturing timeline is all set, we anticipate to initiate a phase 1/2 study of NAITgam early next year. The source plasma we are using to manufacture NAITgam was in part donated by members of the patient organization Naitbabies, and I would like to use this opportunity to express my gratitude for their enduring support.”
NAITgam is a close analogue to anti-D. Both drugs are IgG preparations derived from human plasma, but whereas anti-D is used routinely to prevent RhD-immunization in RhD-negative pregnant women and new mothers in case of exposure to RhD-positive fetal red blood cells, NAITgam is developed to prevent new mothers negative for Human Platelet Antigen-1a (HPA-1a) from being immunized by HPA-1a-positive fetal platelets. HPA-1a-immunized pregnant women may cause their fetus to be thrombocytopenic, which in turn may result in intracranial hemorrhage, intrauterine death or chronic disability. NAITgam has orphan drug status in Europe and the US and was granted rare pediatric disease designation by the U.S. Food and Drug Administration.
The PROFNAIT project is being conducted by 11 European hospitals, universities and businesses and aims to develop a prophylatic treatment of FNAIT.
The project is supported by the EU’s 7th Framework Programme (2012-2018).
The project aims to convert plasma donated by immunized women from the USA, Germany, Norway, Sweden and Holland into a new, safe medicine for the prevention of FNAIT. To do this, test medicine is first made from the collected plasma and used to document that it is safe and effective in preventing FNAIT in fetuses and newborns. Based on this documentation, the European Medicines Agency (EMA) will be asked to approve the new FNAIT medicine.
If you are from the above countries and know or suspect that you have antibodies to HPA-1a and would like to donate plasma for this project please click the following link for further information:
26 February 2016
Prophylix has with the continuous support of naitbabies.org members and our plasma collection partners in the US succeeded in collecting enough plasma for the first 2 batches of NAITgam. With these two batches we can complete the clinical development of NAITgam. A third batch will not be required until the full development program is completed and Prophylix is ready to apply for marketing authorizations with the regulatory authorities in Europe and North America.
Since plasma is a biological product with a limited shelf-life and because we do not need more plasma now, Prophylix has decided to put the collection of further plasma on hold. Prophylix is currently discussing the endpoints of last collections with our collaborate blood establishments and we will reach out to the donors with more specific information. We at Prophylix and our PROFNAIT partners highly appreciate the huge efforts that everybody involved has made to bring us a very important step closer to preventing FNAIT and a deep felt thank you goes to all of you donors and to naitbabies.org. We very much hope to see you all again when it is time to resume the plasma collection program. Naitbabies.org will notify all of its members when that happens.