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Novartis Ligelizumab (QGE031) Receives FDA Breakthrough Therapy Designation for Patients with Chronic Idiopathic Urticaria (CIU)

East Hanover, January 14, 2021 — Novartis today announced that the US Food and Drug Administration (FDA) has granted ligelizumab (QGE031) Breakthrough Therapy designation for the treatment of chronic idiopathic urticaria (CIU), also known as chronic spontaneous urticaria (CSU), in patients who have an inadequate response to H1 antihistamine treatment.

CIU is an unpredictable and severe disease of the skin, affecting 0.5-1% of the global population at any time2. It is characterized by the development of itchy, painful wheals (hives), swelling (angioedema), or both, lasting for at least 6 weeks and occurring with no known cause3. CIU can be challenging or frustrating for patients due to the severity and unpredictable nature4. It most commonly persists for 1-5 years, but in some cases, even longer2.

“Chronic idiopathic urticaria can be a frustrating and debilitating condition for patients because the symptoms can disrupt daily life,” said Victor Bultó, President, Novartis Pharmaceuticals Corporation. “The granting of Breakthrough Therapy designation is welcome news for those who suffer from the disease because there are not many treatment options available to them. We look forward to working closely with the FDA to bring a potential new treatment option to those that need it most.”

According to FDA guidelines, treatments that receive Breakthrough Therapy designation must target a serious or life-threatening disease and demonstrate a potential substantial improvement over existing therapies on one or more significant clinical endpoints5.

About ligelizumab (QGE031)
Ligelizumab is a next-generation monoclonal anti-immunoglobulin E (IgE) antibody. Ligelizumab is thought to work by blocking the IgE/FcεRI pathway, a key driver of the inflammatory process in CIU6,7. In a Phase IIb dose-finding trial, more patients experienced complete resolution of wheals (hives) with ligelizumab compared with Xolair (omalizumab)8. No safety concerns were found with ligelizumab compared with omalizumab or placebo8. Ligelizumab compared with omalizumab is currently being investigated in ongoing Phase III clinical trial programs including PEARL 1 and PEARL 2 (NCT03580369 and NCT03580356)9,10. The clinical trials have recruited more than 2,000 patients globally across 48 countries and results are expected in the second half of 20219,10.

About Novartis in CIU
Novartis is curious about the science beneath the skin and dedicated to reimagining the care of patients with diseases that can severely limit quality of life such as CIU, psoriasis, acne and atopic dermatitis. Advancing ligelizumab further strengthens the immuno-dermatology pipeline of Novartis. In the US, Novartis and Genentech, a member of the Roche Group, work together to develop and co-promote Xolair. Outside the US, Novartis markets Xolair and records all sales and related costs. Xolair is indicated as an add-on therapy for the treatment of CIU. Novartis is also testing remibrutinib (LOU064), a Bruton’s tyrosine kinase (BTK) inhibitor that is being tested in Phase II clinical studies for CIU11.

This media update contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “may,” “could,” “would,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this media update, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this media update will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this media update as of this date and does not undertake any obligation to update any forward-looking statements contained in this media update as a result of new information, future events or otherwise.

About Novartis
Located in East Hanover, NJ Novartis Pharmaceuticals Corporation – an affiliate of Novartis – is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis employs more than 15,000 people in the United States. For more information, please visit


  1. 1.US Food and Drug Administration (FDA). CDER Breakthrough Therapy designation approvals as of December 31, 2019. Accessed December 2020.
    2.Maurer M, Weller K, Bindslev-Jensen C, et al. Unmet clinical needs in chronic spontaneous urticaria: a GA2LEN task force report. Allergy. 2011;66(3):317-330.
    3.Vestergaard C, Deleuran M. Chronic spontaneous urticaria: latest developments in aetiology, diagnosis and therapy. Ther Adv Chronic Dis. 2015;6(6):304-313.
    4.Ferrer, M., Bartra, J., Giménez‐Arnau, A., Jauregui, I., Labrador‐Horrillo, M., Ortiz de Frutos, J., Silvestre, J. F., Sastre, J., Velasco, M., Valero, A., Clinical & Experimental Allergy, 2015 ( 45) 731– 743.
    5.US Food and Drug Administration (FDA). Frequently asked questions: breakthrough therapies. Accessed December 2020.
    6.Puxeddu I, Pratesi F, Ribatti D, Migliorini P. Mediators of inflammation and angiogenesis in chronic spontaneous urticaria: are they potential biomarkers of the disease? Mediators Inflamm. 2017;4123694.
    7.Gasser P, Tarchevskaya SS, Guntern P, et al. The mechanistic and functional profile of the therapeutic anti-IgE antibody ligelizumab differs from omalizumab. Nat Commun. 2020;11(1):165.
    8.Maurer M, Giménez-Arnau AM, Sussman G, et al. Ligelizumab for chronic spontaneous urticaria. N Engl J Med. 2019;381(14):1321-1332.
    9.US National Library of Medicine (NIH). A Phase III study of the efficacy and safety of ligelizumab in the treatment of CSU in adolescents and adults inadequately controlled with H1-histamines. Posted July 9, 2018. Updated December 8, 2020. Accessed December 2020. ID: NCT03580369.
    10.US National Library of Medicine (NIH). A Phase III study of the efficacy and safety of ligelizumab in the treatment of CSU in adolescents and adults inadequately controlled with H1-histamines. Posted July 9, 2018. Updated December 9, 2020. Accessed December 2020. ID: NCT03580356.
    11.US National Library of Medicine (NIH). NCT03926611. Dose-finding Study to Evaluate Efficacy and Safety of LOU064 in Patients With CSU Inadequately Controlled by H1-antihistamines. Posted April 24, 2019. Updated December 9, 2020.  Accessed January 2021. ID: NCT03926611.

Source: Novartis

Posted: January 2021

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