Momenta Pharmaceuticals Announces FDA Rare Pediatric Disease Designation for Nipocalimab in HDFN

Clinical Trials & Research

CAMBRIDGE, Mass., July 28, 2020 (GLOBE NEWSWIRE) – Momenta Pharmaceuticals, Inc. (NASDAQ: MNTA) today announced that its novel drug candidate, nipocalimab, has received rare pediatric disease designation from the U.S. Food and Drug Administration (FDA) for the prevention of hemolytic disease of the fetus and newborn (HDFN). Additionally, FDA granted nipocalimab orphan drug designation in HDFN. HDFN is a serious blood disorder in a fetus or newborn that occurs when red blood cell incompatibility exists between the blood types of a mother and fetus in utero.

“In HDFN, a mismatch in parent’s blood group antigens causes the mother’s immune system to recognize the fetus’ red blood cells as foreign.  This results in the development of pathogenic antibodies that transfer across the placenta during pregnancy and attack the baby’s red blood cells causing fetal anemia which can be fatal,” said Santiago Arroyo, M.D., Ph.D., Senior Vice President of Development and Chief Medical Officer of Momenta Pharmaceuticals. “The standard treatment for HDFN involves intrauterine blood transfusions which are invasive and can be associated with significant complications, including in some cases fetal mortality. Receiving this designation emphasizes the need for a non-invasive, safe, and effective treatment option. Our trial in this indication is progressing and we are committed to working with the FDA to bring this option to families who experience this devastating disease as soon as possible.”

Rare pediatric disease designation is granted by the FDA to drug candidates addressing serious or life-threatening diseases or conditions that affect fewer than 200,000 children in the U.S. Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product. For more information about the rare pediatric disease program, please visit the FDA website at

About Hemolytic Disease of the Fetus and Newborn (HDFN)

Hemolytic disease of the fetus and newborn (HDFN) is a rare alloimmune disorder affecting pregnant women and their families. It can vary from mild to severe and can result in pre-term birth, neonatal morbidity, fetal anemia requiring intervention, or fetal or neonatal loss. HDFN typically becomes more severe with subsequent pregnancies, with severe HDFN impacting between 3,000 to 8,000 pregnancies per year. HDFN is caused by the placental transfer of alloantibodies from the mother’s circulatory system to the fetus during pregnancy. This transfer enables maternal antibodies to attack the red blood cells of the fetus, resulting in fetal anemia and morbidity or mortality. Currently, pregnant women with severe cases are treated with intrauterine blood transfusions, an invasive surgical procedure that can cause significant complications (including up to 20% fetal mortality in the most severe disease and significant morbidity in newborns).

Nipocalimab Overview

Using proprietary antibody engineering technology, Momenta has developed nipocalimab, a high affinity, fully human, aglycosylated, effectorless IgG1 anti-FcRn monoclonal antibody. In alloimmune and autoimmune diseases of the fetus and newborn such as HDFN, FcRn allows the mother’s pathogenic IgG to be transported across the placenta to the fetus causing disease in the fetus or after delivery in the newborn.  FcRn also maintains high circulating concentrations of pathogenic IgG antibodies in the mother through a process called IgG recycling which increases the extent of pathogenic antibody passage to the fetus.

Nipocalimab’s high affinity targeting of FcRn is uniquely designed to both prevent the transfer of IgG from mother to fetus as well as to lower maternal high levels of pathogenic antibody available to cross the placenta. Both effects aim to prevent fetal exposure to pathogenic antibodies and reduce the risk and severity of fetal disease and the resulting morbidities. In addition, due to its high affinity binding, nipocalimab transfer into fetal circulation is insignificant, which could minimize exposure of the fetus. Nipocalimab is the first and only FcRn inhibitor being investigated in alloimmune and autoimmune diseases of the fetus and newborn.

Currently the UNITY Trial, the Company’s global multi-center Phase 2 clinical study of nipocalimab in hemolytic disease of the fetus and newborn (HDFN), is actively enrolling in Europe and five sites are participating in the U.S. The CLARITY Study is actively enrolling HDFN patients who are unable to participate in the UNITY trial. Additional information on the UNITY trial can be found at:

Nipocalimab is also being evaluated in two ongoing clinical trials: the ENERGY Study and the VIVACITY-MG trial. The ENERGY Study is the Company’s adaptive Phase 2/3 clinical study of nipocalimab in warm autoimmune hemolytic anemia (wAIHA). The Company is activating clinical sites globally and expects to reinitiate patient enrollment in the fourth quarter 2020. Nipocalimab has been granted Fast Track and Orphan Drug designation by the FDA in this indication.

The Company recently announced positive top line interim data from the VIVACITY-MG study, investigating nipocalimab in generalized myasthenia gravis (gMG). Over the 8-week study, nipocalimab exhibited no serious adverse events, was well tolerated and significantly improved activities of daily living (ADL) scores in MG patients. The full data is expected in the fourth quarter of 2020. Additional clinical trial information can be found here and patients and families can find more information at

About Momenta

Momenta Pharmaceuticals is a biotechnology company with a validated innovative scientific platform focused on discovering and developing novel therapeutics to treat rare, immune-mediated diseases and advancing its late stage biosimilar portfolio. The company is headquartered in Cambridge, MA.

To receive additional information about Momenta, please visit the website at, which does not form a part of this press release.

The Company’s logo, trademarks, and service marks are the property of Momenta Pharmaceuticals, Inc. All other trade names, trademarks, or service marks are property of their respective owners.

Forward-Looking Statements

Statements in this press release regarding management’s future expectations, beliefs, intentions, goals, strategies, plans or prospects, are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including but not limited to statements about development timelines and strategies; hypotheses regarding certain actions and effects of our product candidates; market potential of our products and product candidates; design, timing, enrollment, strategy and goals of clinical trials and the availability, timing and announcement of data and results ; the use, efficacy, safety, potency, tolerability, convenience and commercial potential of our product candidates; and incidence of disease and patient populations. Forward-looking statements may be identified by words such as “believe,” “continue,” “plan to”, “potential,” “will,” and other similar words or expressions, or the negative of these words or similar words or expressions. Such forward-looking statements involve known and unknown risks, uncertainties and other important factors, including those referred to under the section “Risk Factors” in the Company’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2020 filed with the Securities and Exchange Commission, as well as other documents that may be filed by the Company from time to time with the Securities and Exchange Commission. As a result of such risks, uncertainties and factors, the Company’s actual results may differ materially from any future results, performance or achievements discussed in or implied by the forward-looking statements contained herein. The Company is providing the information in this press release as of this date and assumes no obligations to update the information included in this press release or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Source: Momenta Pharmaceuticals, Inc.

Posted: July 2020

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