Novartis receives complete response letter from U.S. FDA for inclisiran
- The U.S. Food and Drug Administration (FDA) has not raised any concerns related to the efficacy or safety of inclisiran. The complete response letter is due to unresolved facility inspection-related conditions
- No onsite inspection was conducted of the single third-party facility in question. If a facility inspection is needed, FDA will define an approach once safe travel may resume based on public health need and other factors
- Novartis will work with FDA and the third-party manufacturing facility in Europe to complete the inclisiran review, to bring this potential first-in-class siRNA to patients in the U.S. as quickly as possible
Basel, December 18, 2020 — The U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the new drug application (NDA) for inclisiran, a potential treatment for hyperlipidemia in adults who have elevated low-density lipoprotein cholesterol (LDL-C) while being on a maximum tolerated dose of a statin therapy. The FDA stated that the agency cannot approve the NDA by the Prescription Drug User Fee Act (PDUFA) action date of December 23, 2020, due to unresolved facility inspection-related conditions. The conditions will be conveyed to the European manufacturing facility within 10 business days. The third-party facility is responsible for drug product manufacturing. Satisfactory resolution of the unresolved facility inspection-related conditions is required before the Novartis NDA may be approved. No onsite inspection was conducted. If it is determined that a facility inspection is needed to approve the application, the FDA will define an approach for scheduling once safe travel may resume based on public health need and other factors.
“Novartis is confident in the quality of the regulatory submission for inclisiran, which includes a robust body of evidence related to efficacy and safety. We look forward to meeting with the FDA and our third-party manufacturing partner to discuss the feedback received and next steps,” said John Tsai, Head Global Drug Development and Chief Medical Officer, Novartis. “We are committed to bringing this potential first-in-class small interfering RNA cholesterol-lowering treatment to patients as soon as possible.”
The European Commission (EC) recently granted Novartis marketing authorization for Leqvio® (inclisiran) in Europe on December 11, 2020.
Inclisiran (KJX839) is the first and only small interfering RNA (siRNA) therapy to reduce low-density lipoprotein cholesterol (LDL-C) levels via an RNA interference (RNAi) mechanism of action and could help improve outcomes for patients with atherosclerotic cardiovascular disease (ASCVD), a deadly form of cardiovascular disease1-3. With two doses a year and effective and sustained LDL-C reduction, inclisiran works as a complement to statins. Inclisiran works differently from other therapies by preventing the production of the target protein in the liver, increasing hepatic uptake of LDL-C and clearing it from the bloodstream3. Inclisiran is dosed initially, again at 3 months, and then once every 6 months. In three clinical trials, patients taking inclisiran maintained LDL-C reduction throughout each 6-month dosing interval1,2. Administered in-office as a subcutaneous injection, inclisiran is expected to integrate seamlessly into a patient’s healthcare routine1,2.
In the Phase III trials, inclisiran was well-tolerated1,2. The most common adverse events reported (≥3% of patients treated with inclisiran and occurring more frequently than placebo) were injection site reaction, arthralgia, urinary tract infection, diarrhea, bronchitis, pain in extremity and dyspnea1,2. Among those, injection site reactions were the most frequent ones. Those were generally mild and none were severe or persistent1,2.
Novartis has obtained global rights to develop, manufacture and commercialize inclisiran under a license and collaboration agreement with Alnylam Pharmaceuticals, a leader in RNAi therapeutics.
About Novartis in Cardiovascular-Renal-Metabolism
Bending the curve of life requires addressing some of society’s biggest public health concerns. Novartis has an established and expanding presence in diseases covering the heart, kidney and metabolic system. In addition to essential treatment Entresto® (sacubitril/valsartan), Novartis has a growing pipeline of potentially first-in-class molecules addressing cardiovascular, metabolic and renal diseases.
This press release 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 “will,” “may,” “as quickly as possible,” “potential,” “confident,” “look forward,” “committed,” “potential,” “as soon as possible,” “could,” “expected,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for inclisiran and the other investigational and approved products described in this press release, 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 inclisiran will be approved for sale in the United States, or if approved, at any particular time. Neither can there be any guarantee that inclisiran or the other investigational or approved products described in this press release 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 inclisiran and such other products could be affected by, among other things, regulatory actions or delays or government regulation generally; the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; 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 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.
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 products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.
Novartis is on Twitter. Sign up to follow @Novartis at https://twitter.com/novartisnews
For Novartis multimedia content, please visit https://www.novartis.com/news/media-library
For questions about the site or required registration, please contact firstname.lastname@example.org
- Ray KK, Wright RS, Kallend D, et al. Two phase 3 trials of inclisiran in patients with elevated LDL cholesterol. N Engl J Med. 2020;382:1507-1519. doi:10.1056/NEJMoa1912387.
- Raal FJ, Kallend D, Ray KK, et al. Inclisiran for the treatment of heterozygous familial hypercholesterolemia. N Engl J Med. 2020;382(16):1520-1530. doi:10.1056/NEJMoa1913805.
- Stoekenbroek RM, Kallend D, Wijngaard PL, et al. Inclisiran for the treatment of cardiovascular disease: the ORION clinical development program. Future Cardiol. 2018;14(6):433–442.
# # #
|Novartis Media Relations|
| Anja von Treskow|
Novartis External Communications
+41 61 324 2279 (direct)
| Phil McNamara|
Global Head, Cardio-Renal-Metabolism Communications
+41 79 510 8756 (mobile)
| Eric Althoff|
Head, US Corp & Country External Comms
Global Media & Corp Communications
+1 646 438 4335
| Jamie Bennett|
Director, US Media Relations
+1 862 217 3976
|Novartis Investor Relations|
Central investor relations line: +41 61 324 7944
|Samir Shah||+41 61 324 7944||Sloan Simpson||+1 862 778 5052|
|Thomas Hungerbuehler||+41 61 324 8425|
|Isabella Zinck||+41 61 324 7188|
One Liberty Plaza - 165 Broadway
NY 10006 New York
GlobeNewswire is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.
Subscribe to releases from GlobeNewswire
Subscribe to all the latest releases from GlobeNewswire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from GlobeNewswire
Participation Notification by Blackrock Inc.27.1.2021 08:30:00 CET | Press release
Participation notification by Blackrock Inc. Brussels, 27 January 2021, 08:30 CET - According to Belgian transparency legislation (Law of May 2, 2007), BlackRock Inc. (55 East 52nd Street, New York, NY, 10055, U.S.A.) recently sent to Solvay the following transparency notifications indicating that it crossed the threshold of 3%. Here is a summary of the moves: Date on which the threshold was crossedVoting rights after the transactionEquivalent financial instruments after the transactionTotalJanuary 21, 20212.94%0.09%3.03%January 22, 20213.03%0.09%3.12% The latest notification, dated January 25, 2021, contains the following information: Reason for the notification: acquisition or disposal of voting securities or voting rightsNotified by: BlackRock Inc. (55 East 52nd Street, New York, NY, 10055, U.S.A.)Date on which the threshold is crossed: January 22, 2021Threshold of direct voting rights crossed: 3% upwardsDenominator: 105,876,416Additional information: The disclosure obligation arose
Statkraft og Aker Offshore Wind utforsker prosjektmuligheter i Norge27.1.2021 08:01:03 CET | Pressemelding
(Oslo, 27. januar, 2021) Statkraft, Europas største fornybarprodusent, Aker Offshore Wind og Aker Horizons har signert en samarbeidsavtale for å utforske prosjektmuligheter for havvind i Norge. Selskapene vil forene sine sterke og komplementære ferdigheter og ekspertise for sammen å utforske mulighetene for å utvikle, drifte og levere kraft fra kommersielle fullskala havvindparker i Norge. Norske myndigheter annonserte i fjor at de ville åpne for søknader om utvikling av havvind på to områder i Nordsjøen, nærmere bestemt Utsira Nord og Sørlige Nordsjø II. Samarbeidspartnerne vil utforske muligheten for bunnfast havvind på Sørlige Nordsjø II (SN2), hvor selskapene vil etablere en felles prosjektgruppe, 50/50 delt mellom Aker Offshore Wind og Statkraft for felles utarbeidelse og innlevering av melding, samt videre utvikling av prosjektet. Statkrafts ambisjon er å bli et verdensledende selskap innen fornybar energi og har som ledd i dette satset betydelig på utbygging av sol- og vindkraft
Statkraft and Aker Offshore Wind explore project opportunities in Norway27.1.2021 08:01:03 CET | Press release
(Oslo, Norway – January 27, 2021) Statkraft, Europe’s largest renewable energy producer, Aker Offshore Wind and Aker Horizons have signed a cooperation agreement to explore offshore wind project opportunities in Norway. The companies will combine their strong, complementary skills and expertise to work together to explore the possibility to develop, operate and deliver power from commercial scale offshore wind facilities in Norway. Last year, the Norwegian government announced it will open for applications for licenses for the development of wind power in two areas in the North Sea, namely “Utsira Nord” and “Sørlige Nordsjø II”. The cooperation will explore the opportunity for bottom-fixed offshore wind at Sørlige Nordsjø II (“SN2”), for which the companies will establish an integrated project team, 50/50 split between Aker Offshore Wind and Statkraft, for joint preparation and submission of an acreage application and further development activities. Statkraft’s ambition is to be a worl
Ökad omsättning och lönsamhet för 2020 under ett utmanande fjärde kvartal27.1.2021 08:00:00 CET | Pressemelding
Januari - december Nettoomsättningen ökade med +0,8 procent till 310,1 (307,6) mkr (+3 procent i lokala valutor)Rörelseresultatet uppgick till 28,6 (6,2) mkr Rörelsemarginalen uppgick till 9,1 (2,0) procentResultat efter skatt uppgick till 16,3 (4,9) mkrResultat per aktie före/efter utspädning uppgick till 0,88 (0,28) krKassaflöde från den löpande verksamheten uppgick till 52,0 (17,7) mkrLikvida medel uppgick vid periodens utgång till 101,2 (63,9) mkr Fjärde kvartalet Nettoomsättningen minskade med -20,6 procent till 66,7 (84,0) mkr, (-14,5 procent i lokala valutor)Rörelseresultatet uppgick till -4,0 (3,7) mkr Rörelsemarginalen uppgick till -6,0 (4,4) procentResultat efter skatt uppgick till -7,0 (1,3) mkrResultat per aktie uppgick till -0,38 (0,07) krKassaflöde från den löpande verksamheten uppgick till 47,2 (10,5) mkr VD's Kommentar 2020, som vi nu lämnat bakom oss, har utan tvekan varit ett av de mest omvälvande åren under min tid som VD för MSAB. Pandemin har definierat helt nya oc
Increased sales and profitability in 2020 during a challenging fourth quarter27.1.2021 08:00:00 CET | Press release
January - December Net sales increased +0.8 percent to SEK 310.1 (307.6) million (+3 percent in local currencies).EBIT was SEK 28.6 (6.2) million.The EBIT margin was 9.1 (2.0) percent.Profit after tax amounted to SEK 16.3 (4.9) million.Earnings per share before/after dilution amounted to SEK 0.88 (0.28).Cash flow from operating activities amounted to SEK 52.0 (17.7) million.Cash and cash equivalents at the end of the period amounted to SEK 101.2 (63.9) million. Fourth quarter Net sales declined -20.6 percent to SEK 66.7 (84.0) million (-14.5 percent in local currencies).EBIT was SEK -4.0 (3.7) million.The EBIT margin was -6.0 (4.4) percent.Profit after tax amounted to SEK -7.0 (1.3) million.Earnings per share amounted to SEK -0.38 (0.07).Cash flow from operating activities amounted to SEK 47.2 (10.5) million. Comments from the CEO Looking at 2020 in the rear-view mirror, I can say that it has without doubt been one of my stormiest years as CEO for MSAB. The pandemic has defined an enti
Immunicum AB (publ) planerar att expandera bolagets anläggning för forskning och processutveckling i Leiden, Nederländerna27.1.2021 08:00:00 CET | Pressemelding
27 januari 2021 Immunicum AB (publ) planerar att expandera bolagets anläggning för forskning och processutveckling i Leiden, Nederländerna Immunicum AB (publ; IMMU.ST) meddelar idag att de aktiviteter inom forskning och processutveckling som bolaget driver i egen regi kommer att flytta till en ny anläggning i Leiden Bio Science Park i Nederländerna. Bolaget har ingått avtal om ett långtidshyreskontrakt med entreprenören Kadans Science Partner avseende cirka 1 100 m2 laboratorie- och kontorsyta i Plus Ultra Leiden, en ny byggnad som är under konstruktion. Vidare har Immunicum option att hyra ytterligare 1 100 m2 för att ytterligare stödja bolagets framtida tillväxt., Den nya anläggningen, som förväntas vara i drift år 2022, kommer att fungera som Immunicums nav för forskning och utveckling. ”Denna toppmoderna anläggning i en av Europas hetaste platser för forskning inom life science är en integrerad del av vår bolagsstrategi som gör det möjligt för oss att snabbt och kostnadseffektivt f
Immunicum AB (publ) Plans to Expand its Research and Process Development Facilities in Leiden, the Netherlands27.1.2021 08:00:00 CET | Press release
Press Release 27 January 2021 Immunicum AB (publ) Plans to Expand its Research and Process Development Facilities in Leiden, the Netherlands Immunicum AB (publ; IMMU.ST) announced today that its in-house research and process development activities will move into a new facility at the Leiden Bio Science Park, the Netherlands. The Company has signed a long-term lease with developer Kadans Science Partner for approximately 1,100 square meters of laboratory and office space in the Plus Ultra Leiden, a new building currently under development. Furthermore, Immunicum has the option to rent an additional 1,100 square meters to further support its future growth. The new location, that is expected to be fully commissioned in 2022, will serve as Immunicum’s core research and development hub. “This state-of-the-art facility in one of Europe’s most vibrant life sciences research communities is an integral part of our corporate strategy as it will enable us to rapidly and cost-effectively continue