SPRING HOUSE, Pa. March 26, 2024 – Johnson & Johnson introduced right now that the U.S. Meals and Drug Administration (FDA) has granted Quick Observe designation (FTD) for nipocalimab to cut back the chance of FNAIT in alloimmunizeda pregnant adults throughout their present being pregnant. FNAIT is a uncommon and extreme situation that happens when the immune system of a pregnant particular person mistakenly assaults platelets in a creating fetus. This immune response can result in impaired clotting capacity and bleeding, posing a big danger to the fetus or new child.1 Nipocalimab, an investigational monoclonal antibody concentrating on FcRn, is the one investigational remedy presently reported to be in scientific improvement to handle the wants of alloimmunized pregnant people susceptible to FNAIT.
The FDA’s Quick Observe program is designed to expedite improvement and evaluation timelines of medication that show the potential to deal with critical situations and tackle unmet medical wants for critical or life-threatening situations. Quick Observe designation helps shut communication between the FDA and sponsor with the intention of delivering new therapeutics to sufferers extra rapidly.
“Receiving Quick Observe designation for nipocalimab in FNAIT underscores the urgency to handle the unmet want for protected, efficient, and focused therapies to stop FNAIT, a situation that might carry extreme well being penalties and even be deadly for the fetus or new child,” mentioned Katie Abouzahr, M.D., Vice President, Autoantibody and Maternal Fetal Immunology Illness Space Chief, Johnson & Johnson. “We’re dedicated to making use of our a long time of immunology management to pioneer progressive approaches to rework therapy for sufferers and their households affected by FNAIT and different alloantibody-driven illnesses of being pregnant.”
Johnson & Johnson is conducting analysis and improvement for nipocalimab, an FcRn blocker, to handle the numerous unmet want in decreasing the chance of FNAIT. Nipocalimab is believed to work by blocking the switch of immunoglobulin G (IgG) alloantibodies from pregnant people to their infants by the placenta whereas not suppressing the broader immune methods of the pregnant particular person or creating fetus.2 Johnson & Johnson can also be continuing with two Part 3 trials targeted on FNAIT. Nipocalimab was granted orphan drug designation by the U.S. FDA for FNAIT in December 2023.
Nipocalimab is moreover being studied in hemolytic illness of the fetus and new child (HDFN), one other alloimmune illness of being pregnant with an identical illness mechanism, sometimes called the pink blood cell counterpart to FNAIT.3 After Part 2 security and efficacy outcomes from the UNITY trial, Johnson & Johnson is moreover continuing with Part 3 trials targeted on HDFN.4
a. Alloimmunized: an immune response to overseas antigens upon publicity to genetically totally different cells or tissues
About Fetal Neonatal Alloimmune Thrombocytopenia (FNAIT)
Fetal neonatal alloimmune thrombocytopenia (FNAIT) is a uncommon and probably life-threatening alloimmune situation wherein a pregnant particular person’s immune system develops antibodies in opposition to fetal or new child platelet antigens, resulting in thrombocytopenia (low platelet counts within the fetus or new child).1
FNAIT can lead to extreme bleeding problems for a fetus or new child and is characterised by organ bleeding within the gastrointestinal tract, lungs, or eyes, and should lead to lifelong incapacity or dying.1 If a extreme bleed happens within the mind, termed intracranial hemorrhage (ICH), dying or life-long neurologic results might happen.1 ICH happens in as much as 26 % of untreated pregnancies with FNAIT.5
There are not any permitted focused therapies for FNAIT administration. FNAIT is just not routinely screened for throughout being pregnant and firstborn kids with FNAIT are sometimes solely recognized postnatally.1
About HDFN
Hemolytic illness of the fetus and new child (HDFN) is a uncommon illness (and in its extreme kind, extremely uncommon) that arises in pregnancies with maternal-fetal incompatibility in sure pink blood cell sorts.6 Alloantibodies produced by the maternal immune system in opposition to fetal pink blood cells cross the placenta throughout being pregnant and assault fetal pink blood cells inflicting fetal anemia or persist after start within the neonate to trigger neonatal hyperbilirubinemia and anemia.2 The signs of HDFN can vary from gentle jaundice, to neurotoxic hyperbilirubinemia within the new child, to life-threatening fetal anemia requiring invasive intervention.7 The potential for in utero onset at an more and more earlier GA with rising danger of extreme outcomes might happen with every incompatible being pregnant on account of pregnancy-related alloimmunization.8 Presently there are not any non-surgical interventions permitted for pregnancies at excessive danger for extreme HDFN.3 Pregnancies affected by extreme HDFN might necessitate repeated intrauterine transfusions (IUTs), that are invasive, technically advanced surgical procedures carried out by specialists at specialised medical facilities, and these procedures are related to an elevated fee of fetal mortality and untimely start.9,10,11 Probably the most tough to deal with circumstances of HDFN are early onset extreme HDFN (EOS-HDFN) that develops at ≤24 weeks gestational age (GA) and leads to important fetal/neonatal morbidity and mortality. In keeping with the American Journal of Obstetrics and Gynecology, within the U.S., it’s estimated that as much as 80 of each 100,000 pregnancies are affected by HDFN annually.12
About Nipocalimab
Nipocalimab is an investigational, high-affinity, absolutely human, aglycosylated, effectorless, monoclonal antibody that goals to selectively block FcRn to cut back ranges of circulating immunoglobulin G (IgG) antibodies, together with autoantibodies and alloantibodies that underlie a number of situations.13 Nipocalimab is the one FcRn blocker being studied throughout three key segments within the autoantibody area: Uncommon Autoantibody illnesses (e.g., generalized myasthenia gravis in adults and youngsters, persistent inflammatory demyelinating polyneuropathy, heat autoimmune hemolytic anemia, and idiopathic inflammatory myopathies); Maternal Fetal illnesses mediated by maternal alloantibodies (e.g., hemolytic illness of the fetus and new child and fetal and neonatal alloimmune thrombocytopenia); and Prevalent Rheumatology (e.g., rheumatoid arthritis, Sjögren’s illness, and systemic lupus erythematosus).14,15,16,17,18,19,20,21,22 Blockade of FcRn has the potential to cut back total IgG together with pathogenic alloantibody ranges whereas preserving immune operate with out inflicting broad immunosuppression. Blockade of IgG binding to FcRn within the placenta can also be believed to stop transplacental switch of maternal alloantibodies to the fetus.8,23
About Johnson & Johnson
At Johnson & Johnson, we imagine well being is the whole lot. Our energy in healthcare innovation empowers us to construct a world the place advanced illnesses are prevented, handled, and cured, the place therapies are smarter and fewer invasive, and options are private. By way of our experience in Revolutionary Medication and MedTech, we’re uniquely positioned to innovate throughout the total spectrum of healthcare options right now to ship the breakthroughs of tomorrow, and profoundly impression well being for humanity.
Cautions Regarding Ahead-Trying Statements
This press launch accommodates “forward-looking statements” as outlined within the Non-public Securities Litigation Reform Act of 1995 concerning product improvement and the potential advantages and therapy impression of nipocalimab. The reader is cautioned to not depend on these forward-looking statements. These statements are based mostly on present expectations of future occasions. If underlying assumptions show inaccurate or identified or unknown dangers or uncertainties materialize, precise outcomes might differ materially from the expectations and projections of Janssen Analysis & Improvement, LLC, Janssen Biotech, Inc. and/or Johnson & Johnson. Dangers and uncertainties embody, however are usually not restricted to: challenges and uncertainties inherent in product analysis and improvement, together with the uncertainty of scientific success and of acquiring regulatory approvals; uncertainty of economic success; manufacturing difficulties and delays; competitors, together with technological advances, new merchandise and patents attained by opponents; challenges to patents; product efficacy or security considerations leading to product remembers or regulatory motion; adjustments in habits and spending patterns of purchasers of well being care services and products; adjustments to relevant legal guidelines and rules, together with international well being care reforms; and tendencies towards well being care price containment. An additional listing and descriptions of those dangers, uncertainties and different components will be present in Johnson & Johnson’s Annual Report on Type 10-Ok for the fiscal yr ended December 31, 2023, together with within the sections captioned “Cautionary Notice Concerning Ahead-Trying Statements” and “Merchandise 1A. Threat Components,” and in Johnson & Johnson’s subsequent Quarterly Reviews on Type 10-Q and different filings with the Securities and Trade Fee. Copies of those filings can be found on-line at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. None of Janssen Analysis & Improvement, LLC, Janssen Biotech, Inc. nor Johnson & Johnson undertakes to replace any forward-looking assertion because of new info or future occasions or developments.
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2 Ling LE, Hillson JL, Tiessen RG, et al. M281, an anti-FcRn antibody: pharmacodynamics, pharmacokinetics, and security throughout the total vary of igG discount in a first-in-human research. Clin Pharmacol Ther. 2019;105(4):1031-1039.
3 Orphanet. Fetal and Neonatal Alloimmune Thrombocytopenia. https://www.orpha.internet/en/illness/element/853. Final accessed on February 28, 2024.
4 Clinicaltrials.gov. A Research to Consider the Security, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Pregnant Girls at Excessive Threat for Early Onset Extreme Hemolytic Illness of the Fetus and New child (HDFN). Final accessed: November 18, 2022. https://clinicaltrials.gov/ct2/present/NCT03842189.
5 Constantinescu S, Zamfirescu V, Vladareanu PR. Fetal and neonatal alloimmune thrombocytopenia. Maedica (Bucur). 2012 Dec;7(4):372-6. PMID: 23482913; PMCID: PMC3593293.
6 Hemolytic illness of the new child. Medline Plus. Final accessed: November 18, 2022.
7 Ree IMC, Smits-Wintjens VEHJ, van der Bom JG, et al. Neonatal administration and final result in alloimmune hemolytic illness, Knowledgeable Assessment of Hematology, 10:7, 607-616, DOI: 10.1080/17474086.2017.1331124. Final accessed: June 2023.
8 Lobato G, Soncini CS. Relationship between obstetric historical past and Rh(D) alloimmunization severity. Arch Gynecol Obstet. 2008 Mar;277(3):245-8. DOI: 10.1007/s00404-007-0446-x. Final accessed: June 2023.
9 Texas Youngsters’s Hospital. Intrauterine Transfusion. Out there at: https://girls.texaschildrens.org/program/texas-childrens-fetal-center/procedures-offered/intrauterine-transfusion. Final accessed: June 2023.
10 de Winter DP, Kaminski A, et al. Hemolytic illness of the fetus and new child: systematic literature evaluation of the antenatal panorama. BMC Being pregnant and Childbirth. 2023;23(12). DOI: https://doi.org/10.1186/s12884-022-05329-z. Final accessed: June 2023.
11 Lindenburg IT, van Kamp IL, van Zwet EW, Middeldorp JM, Klumper FJ, Oepkes D. Elevated perinatal loss after intrauterine transfusion for alloimmune anaemia earlier than 20 weeks of gestation. BJOG. 2013 Jun;120(7):847-52. doi: 10.1111/1471-0528.12063.
12 Delaney M, Matthews DC. Hemolytic illness of the fetus and new child: managing the mom, fetus, and new child. Hematology Am Soc Hematol Educ Program. (2015) 2015(1):146-151. DOI: https://doi.org/10.1182/asheducation-2015.1.146. Final accessed: June 2023.
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23 Roy S, Nanovskaya T, Patrikeeva S, et al. M281, an anti-FcRn antibody, inhibits IgG switch in a human ex vivo placental perfusion mannequin. Am J Obstet Gynecol. 2019;220(5):498 e491-498 e499.
Supply: Johnson & Johnson
Posted: March 2024
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