Novartis Aktie
WKN: 907122 / ISIN: US66987V1098
11.12.2023 01:29:44
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Press Release: Novartis presents new 48-week -2-
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; 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 press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
About Novartis
Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach more than 250 million people worldwide.
Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on https://www.linkedin.com/company/novartis/ LinkedIn, https://www.facebook.com/novartis/ Facebook, https://twitter.com/Novartis X/Twitter and https://instagram.com/novartis?igshid=MzRlODBiNWFlZA==__;!!N3hqHg43uw!pjp8z253J5NjaOYrW65UbAAlHeHRdQ-w0m4ezZxEQEl0ptafXN2M99VRIk39pf49PAc8NbK93Pxp3uaSBQkAf8oEnzWXG8Sk$ Instagram.
References
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Oral Iptacopan Monotherapy Demonstrates Sustained Long-Term Efficacy and
Safety in Anti-C5-Treated Patients (pts) with Paroxysmal Nocturnal
Hemoglobinuria (PNH) and Persistent Anemia: Final 48-Week Results from
the Multicenter, Phase III APPLY-PNH Trial. Presented at: 65th American
Society of Hematology Annual Meeting & Exposition (ASH); December 9-12,
2023; San Diego, CA
2. Risitano AM, R th A, Kulasekararaj A, et al. Oral Iptacopan Monotherapy
Has Superior Efficacy to Anti-C5 Therapy in Patients with Paroxysmal
Nocturnal Hemoglobinuria and Residual Anemia: Results From the Phase III
APPLY-PNH Study. Presented at: 49th Annual Meeting of the European
Society for Blood and Marrow Transplantation (EBMT); April 23-36, 2023;
Paris, France.
3. Risitano AM, Han B, Ueda Y, et al. Oral Complement Factor B Inhibitor
Iptacopan Monotherapy Improves Hemoglobin to Normal/Near-Normal Levels in
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Meeting of the European Society for Blood and Marrow Transplantation
(EBMT); April 23-36, 2023; Paris, France.
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factor B inhibitor, to eculizumab in patients with paroxysmal nocturnal
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5. Jang H, Wong L, Ko BS, et al. Iptacopan monotherapy in patients with
paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label
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6. Novartis receives FDA approval for Fabhalta(R) (iptacopan), offering
superior hemoglobin improvement in the absence of transfusions as the
first oral monotherapy for adults with PNH. Novartis. Accessed December
6, 2023.
https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-fabhalta-iptacopan-offering-superior-hemoglobin-improvement-absence-transfusions-first-oral-monotherapy-adults-pnh
7. Cançado RD, Araújo A da S, Sandes AF, et al. Consensus
statement for diagnosis and treatment of paroxysmal nocturnal
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survey. Ann Hematol. 2022;101(2):251-263. doi:10.1007/s00277-021-04715-5
9. Debureaux PE, Kulasekararaj AG, Cacace F, et al. Categorizing
hematological response to eculizumab in paroxysmal nocturnal
hemoglobinuria: a multicenter real-life study. Bone Marrow Transplant.
2021;56(10):2600-2602. doi:10.1038/s41409-021-01372-0
10. Debureaux PE, Cacace F, Silva BGP, et al. Hematological Response to
Eculizumab in Paroxysmal Nocturnal Hemoglobinuria: Application of a Novel
Classification to Identify Unmet Clinical Needs and Future Clinical
Goals. Blood. 2019;134(Supplement_1):3517-3517.
doi:10.1182/blood-2019-125917
11. Novartis Pharmaceuticals. A Randomized, Multicenter, Active-Comparator
Controlled, Open-Label Trial to Evaluate Efficacy and Safety of Oral,
Twice Daily LNP023 in Adult Patients With PNH and Residual Anemia,
Despite Treatment With an Intravenous Anti-C5 Antibody.
clinicaltrials.gov; 2022. Accessed September 21, 2022.
https://clinicaltrials.gov/ct2/show/NCT04558918
12. Hill A, DeZern AE, Kinoshita T, Brodsky RA. Paroxysmal nocturnal
haemoglobinuria. Nat Rev Dis Primer. 2017;3(1):17028.
doi:10.1038/nrdp.2017.28
13. Schrezenmeier H, Röth A, Araten DJ, et al. Baseline clinical
characteristics and disease burden in patients with paroxysmal nocturnal
hemoglobinuria (PNH): updated analysis from the International PNH
Registry. Ann Hematol. 2020;99(7):1505-1514.
doi:10.1007/s00277-020-04052-z
14. Schubart A, Anderson K, Mainolfi N, et al. Small-molecule factor B
inhibitor for the treatment of complement-mediated diseases. Proc Natl
Acad Sci. 2019;116(16):7926-7931. doi:10.1073/pnas.1820892116
15. McKinley CE, Richards SJ, Munir T, et al. Extravascular Hemolysis Due to
C3-Loading in Patients with PNH Treated with Eculizumab: Defining the
Clinical Syndrome. Blood. 2017;130(Supplement 1):3471.
doi:10.1182/blood.V130.Suppl_1.3471.3471
16. Barratt J, Rovin B, Zhang H, et al. POS-546 EFFICACY AND SAFETY OF
IPTACOPAN IN IgA NEPHROPATHY: RESULTS OF A RANDOMIZED DOUBLE-BLIND
PLACEBO-CONTROLLED PHASE 2 STUDY AT 6 MONTHS. Kidney Int Rep.
2022;7(2):S236. doi:10.1016/j.ekir.2022.01.577
17. Rizk DV, Rovin BH, Zhang H, et al. Targeting the Alternative Complement
Pathway With Iptacopan to Treat IgA Nephropathy: Design and Rationale of
the APPLAUSE-IgAN Study. Kidney Int Rep. 2023;8(5):968-979.
doi:10.1016/j.ekir.2023.01.041
18. Novartis Pharmaceuticals. A Multicenter, Randomized, Double-Blind,
Parallel Group, Placebo-Controlled Study to Evaluate the Efficacy and
Safety of Iptacopan (LNP023) in Complement 3 Glomerulopathy.
clinicaltrials.gov; 2022. Accessed September 20, 2022.
https://clinicaltrials.gov/ct2/show/NCT04817618
19. Novartis Pharmaceuticals. A Multi-Center, Randomized, Double-Blind,
Placebo-Controlled, Parallel Group, Phase III Study to Evaluate the
Efficacy and Safety of LNP023 in Primary IgA Nephropathy Patients.
clinicaltrials.gov; 2022. Accessed September 21, 2022.
https://clinicaltrials.gov/ct2/show/NCT04578834
20. Novartis announces European Medicines Agency (EMA) has granted orphan
drug designation for iptacopan (LNP023) in IgA nephropathy (IgAN).
Novartis. Accessed September 22, 2022.
https://www.novartis.com/news/media-releases/novartis-announces-european-medicines-agency-ema-has-granted-orphan-drug-designation-iptacopan-lnp023-iga-nephropathy-igan
21. Novartis received European Medicines Agency (EMA) PRIME designation for
iptacopan (LNP) in C3 glomerulopathy (C3G). Novartis. Accessed September
22, 2022.
https://www.novartis.com/news/media-releases/novartis-received-european-medicines-agency-ema-prime-designation-iptacopan-lnp-c3-glomerulopathy-c3g
22. Novartis investigational oral therapy iptacopan (LNP023) receives FDA
Breakthrough Therapy Designation for PNH and Rare Pediatric Disease
Designation for C3G. Novartis. Accessed September 22,
2022. https://www.novartis.com/news/media-releases/novartis-investigation
al-oral-therapy-iptacopan-lnp023-receives-fda-breakthrough-therapy-design
ation-pnh-and-rare-pediatric-disease-designation-c3g
23. Novartis. Data on File.
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