May 21, 2019
Emergent BioSolutions to Manufacture Prophylix AS Developmental Drug for Fetal-Neonatal Alloimmune Thrombocytopenia
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.
December 19, 2017
FDA designates NAITgam as a novel drug for a rare pediatric disease
Today, the US Food and Drug Administration designated NAITgam as a novel drug for prevention of the rare and potentially disabling or lethal pediatric condition Fetal Neonatal Alloimmune Thrombocytopenia. This pediatric rare disease (PRD) designation greatly increases Prophylix’s chance of also being granted a Pediatric Rare Disease Priority Review Voucher, most recently traded for around $125 million, upon approval of NAITgam by the FDA before October 2022, a key goal for the company.
February 15, 2016
General HPA-1a typing of pregnant women and prevention of FNAIT in at-risk pregnancies is highly cost-effective
Independent health-economic analyses of the cost-effectiveness of general HPA-1a typing of all pregnant women and prevention of FNAIT in at-risk pregnancies demonstrate that the typing and prophylaxis promoted by Prophylix will be highly cost-effective in both the US and the UK assuming realistic efficacy levels of the FNAIT prophylaxis for either postnatal or combined antenatal/postnatal administration.
UK and US advisory boards with representation of both clinicians and payers have validated the model structures, the input data and the required assumptions.
The favorable results of the health-economic analyses came as now surprise to Prophylix’s Chief Clinical Officer Dr. Jonas Lundahl, MMBS who comments: «The current use of massive doses of intravenous immunoglobulin to minimize the impact of maternal HPA-1a immunization, the transfusion of platelets to the fetus, the prolonged stays at neonatal intensive care units etc. impose huge costs to the health care system. In addition, the affected babies may need multiple rounds of surgery, disability equipment and special education. Both the medical side and the payer side see the potential of a prophylaxis».
August 26, 2015
12 key North American and European medical societies focused on obstetrics and gynecology confirms the unmet need in FNAIT and will support general HPA-1a typing of pregnant women and cost-effective FNAIT prophylaxis
After having been presented with a briefing package containing information about Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) and Prophylix’s efforts to develop a prophylaxis for FNAIT, the addressed medical societies unanimously agreed that FNAIT in their opinion represent an unmet need and cost-effective FNAIT prophylaxis was supported. The favorable statements were obtained from:
- American Congress of Obstetricians and Gynecologists
- Society for Maternal-Fetal Medicine (US)
- Canadian Society of Maternal Fetal Medicine
- Society of Obstetricians and Gynaecologists of Canada
- North American Fetal Therapy Network (NAFTNet)
- Royal College of Obstetricians and Gynaecologists
- British Maternal and Fetal Medicine Society
- German Society of Prenatal Medicine and Obstetrics
- Dutch Society of Obstetrics and Gynaecology
- Swedish Society of Obstetrics and Gynaecology
- Norwegian Society of Obstetrics and Gynaecology
- Danish Society of Obstetrics and Gynaecology
- Spanish Society of Blood Transfusion and Cellular Therapy
Prophylix’s Chief Scientific Officer, Dr. Jens Kjeldsen-Kragh, MD comments: «FNAIT is a very rare condition and which severely affects only 1 in 10,000 pregnancies. Still, these key medical societies have now confirmed that we as a society should aim to help the 1,000 babies and families who each year experience this devastating and potentially lethal condition.»
October 2, 2014
FDA accepts Prophylix’s clinical development plan for NAITgam (anti-HPA-1a immunoglobulin) for the prevention of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
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 which affects about 1,000 pregnancies annually in the US and Europe.
The US FDA has now approved Prophylix’s lean program for developing NAITgam, including the use of historical control data, primary and surrogate end-points and studies in an enriched patient population. Prophylix will apply for Fast Track Designation and work closely with the FDA throughout the program.
NAITgam comprises IgG antibodies against human platelet antigen-1a (HPA-1a). The drug will be manufactured by Emergent BioSolutions from the plasma of women who have given birth to a child with FNAIT. The antibodies in the prophylactic drug are intended to remove fetal HPA-1a positive platelets from the mother’s blood before her immune system has had time to develop a much stronger immune response 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 at Prophylix Pharma AS comments, «It should also be noted that NAITgam is an exact platelet analogue 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 that gives us a fast and feasible path to the market».
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 soon now go into clinical trials is extremely encouraging. We will be looking to help recruit plasma donors through our growing international network.»
October 1, 2014
New York Blood Center, Prophylix Pharma AS and BloodCenter of Wisconsin Collaborate to Fight Devastating Neonatal Bleeding Disorder
10,000 babies in the United States and European Union suffer each year from a potentially crippling and fatal bleeding disorder called Fetal and Neonatal Alloimmune Thrombocytopenia, or FNAIT. Currently no therapy exists to prevent FNAIT. FNAIT occurs when amother develops antibodies against her baby’s platelets, animportant blood component that stops bleeding. New York City’s top medical powerhouses and Blood Center of Wisconsin are joining with Prophylix Pharma and the EU-funded PROFNAIT consortium of ten universities, hospitals and research organizations to develop a product called NAITgam for prevention of FNAIT.
Prophylix Pharma and BloodCenter of Wisconsin will help identify blood plasma donors among women who have had babies affected by FNAIT. New York Blood Center will collect the donor’s plasma, and has applied to the Food and Drug Administration for a Source Plasma License, a necessary component to its collaboration. The collected plasma will be made into NAITgam which will be given to at-risk pregnant women so they do not make antibodies against their baby’s platelets. This may prevent FNAIT in that and future pregnancies.
NAITgam is very similar to Rh immune globulin which for almost 50 years has proven safe and effective in almost eliminating the related condition, Hemolytic Disease of the Fetus and Newborn – caused by mothers making antibodies against their babies’ red blood cells.
Dr. Søren Weis Dahl, Chief Business Officer and EU Project Coordinator, Prophylix Pharma AS comments, «Access to the specialty source plasma that New York Blood Center is now starting to collect for the PROFNAIT project is essential for our ability to manufacture and develop NAITgam.»
Dr. Beth H. Shaz, Chief Medical Officer, New York Blood Center adds, «Saving lives is what the New York Blood Center is all about. We’re excited to participate in a program that could alleviate so much of the suffering endured by families potentially affected by this bleeding disorder.»
September 1, 2014
Prophylix Pharma Signs Manufacturing Agreement with Emergent BioSolutions
Prophylix has signed an agreement with Emergent BioSolutions on the manufacture of the new FNAIT prophylaxis, NAITgam, which is developed by Prophylix and its partners in the EU-funded PROFNAIT consortium of ten universities, hospitals and research organizations. FNAIT is a potentially lethal or disabling bleeding disorder which every year affects 10,000 babies in EU and the US. 1,000 of these experience intracranial hemorrhage that leads to life-long disability or death.
NAITgam will be produced from human plasma using Emergent’s proprietary hyperimmune manufacturing process, which today is used to produce a number of similar FDA-licensed products such as the anti-D drug WinRho® SDF [Rho (D) Immune Globulin Intravenous (Human)].
Dr. Søren Weis Dahl, Chief Business Officer and EU Project Coordinator, Prophylix comments, «Emergent is the perfect manufacturing partner to complement the efforts of the PROFNAIT partners, New York Blood Center, Biotest Pharmaceuticals Corporation and BloodCenter of Wisconsin who all collaborate to identify plasma donors and collect their plasma for use in NAITgam manufacture.» In general, NAITgam can only be produced from plasma from women who have had a baby affected by the rare condition FNAIT.
Barry Labinger, Executive Vice President and President BiosciencesDivision at Emergent, adds, «We are pleased to leverage our hyperimmune manufacturing expertise and platform to produce clinical grade material for Prophylix Pharma’s upcoming clinical studies. We look forward to supporting the team at Prophylix in their efforts to address this unmet medical need.»
Norwegian TV reports on the PROFNAIT project
A HPA-1a negative mother and one of Norway’s leading medical doctors in FNAIT describe the risk of the condition, the PROFNAIT team’s efforts to prevent FNAIT and the need for HPA-1a screening of all pregnant women (NRK web cast, in Norwegian).
Consortium lead byProphylix Pharma AS receives up to €6 M inEU-funding
A proposal from Prophylix Pharma AS for developing a new therapy under the European Commission’s Seventh Framework Programme (FP7- HEALTH) has been favorably evaluated. A Grant Agreement providing funding of up to €6 million is now being negotiated (press release, in English)
Prophylix invited to negotiate a grant agreement of up to €6 M with the EUsFP
We are proud to announce that Prophylix, as the Coordinator of the project, has been invited to negotiate a grant agreement with the European Commission for a collaborative project aiming to develop the first drug that will be able to prevent severe bleeding in fetuses and newborns due to fetal/neonatal alloimmune thrombocytopenia (FNAIT). The project will establish the scientific, clinical and quality documentation that is required to obtain Marketing Authorization in EU and the US for the new drug. In addition to Prophylix, the project includes 8 consortium partners in Norway, Sweden, Denmark and Germany.
The European Commission grants Prophylix’s FNAIT prophylaxis «Orphan Medicinal Product»status
The European Commission has granted Prophylix Pharma’s development drug Tromplate (aka. NAITgam) Orphan Medicinal Product status. Tromplate is intended for preventing the development of the potential lethal condition fetal/neonatal alloimmune thrombocytopenia (FNAIT) in newborns. Tromplate’s new status as an Orphan Medicinal Productwill give Prophylix access to extended support from EMA during the clinical trial phases. Upon market introduction, the product will be granted ten years of market exclusivity.