TPO receptor

Ad hoc announcement pursuant to Art. 53 LR Net sales grew +15% (cc1, +12% USD) with core operating income1 up +27% (cc, +23% USD) Sales growth driven by continued strong performance from Entresto (+22% cc), Kisqali (+56% cc), Kesimpta (+43% cc), Cosentyx (+18% cc), Leqvio (+72% cc) and Scemblix (+76% cc) Core operating income margin1 reached 42.1%, +400 basis points (cc), mainly driven by higher net sales Operating income grew +44% (cc, +38% USD); net income up +37% (cc, +34% USD)Core EPS1 grew +31% (cc, +27% USD) to USD 2.28Free cash flow1 of USD 3.4 billion (+66% USD) driven by higher net cash flows from operating activities Selected innovation milestones: Pluvicto FDA approval for pre-taxane mCRPC Vanrafia (atrasentan) FDA accelerated approval for IgA nephropathy Fabhalta (iptacopan) FDA, EC and China NMPA approvals for C3G Remibrutinib global submissions for CSU, with priority review voucher in US OAV101 IT Phase III STEER study positive readout in SMA Full-year 2025 guidance2 raised: Sales expected to grow high single digit, core operating income expected to grow low double-digit Basel, April 29, 2025 – Commenting on Q1 2025 results, Vas Narasimhan, CEO of Novartis, said:“Novartis has had a strong start to the year, delivering a +15% cc increase in sales and a +27% cc rise in core operating income in Q1. Our priority brands, including Kisqali, Kesimpta and Leqvio, continue to show strong momentum, which we anticipate will drive our growth through 2030 and beyond. We also achieved significant innovation milestones in the quarter, with new approvals for Pluvicto in the pre-taxane setting, Vanrafia for IgA nephropathy, and Fabhalta for C3G. Additionally, we completed global submissions for remibrutinib in CSU, the first indication for this promising pipeline-in-a-pill. We remain focused on advancing our leading pipeline and confident in achieving our growth outlook.” Key figures Q1 2025Q1 2024% change USD m USD mUSDccNet sales to third parties13 23311 8291215Operating income4 6633 3733844Net income3 6092 6883437EPS (USD)1.831.314042Free cash flow3 3912 03866 Core operating income5 5754 5372327Core net income4 4823 6812226Core EPS (USD)2.281.802731 1. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 31 of the Condensed Interim Financial Report. Unless otherwise noted, all growth rates in this Release refer to same period in prior year. 2. Please see detailed guidance assumptions on page 5. Strategy Our focus Novartis is a “pure-play” innovative medicines company. We have a clear focus on four core therapeutic areas (cardiovascular-renal-metabolic, immunology, neuroscience and oncology), with multiple significant in-market and pipeline assets in each of these areas, that address high disease burden and have substantial growth potential. In addition to two established technology platforms (chemistry and biotherapeutics), three emerging platforms (gene & cell therapy, radioligand therapy and xRNA) are being prioritized for continued investment into new R&D capabilities and manufacturing scale. Geographically, we are focused on growing in our priority geographies – the US, China, Germany and Japan. Our priorities Accelerate growth: Renewed attention to deliver high-value medicines (NMEs) and focus on launch excellence, with a rich pipeline across our core therapeutic areas.Deliver returns: Continuing to embed operational excellence and deliver improved financials. Novartis remains disciplined and shareholder-focused in our approach to capital allocation, with substantial cash generation and a strong capital structure supporting continued flexibility. Strengthen foundations: Unleashing the power of our people, scaling data science and technology and continuing to build trust with society. Financials Net sales were USD 13.2 billion (+12%, +15% cc), with volume contributing 15 percentage points to growth. Generic competition had a negative impact of 2 percentage points and pricing had a positive impact of 2 percentage points, benefiting from revenue deduction adjustments mainly in the US. Operating income was USD 4.7 billion (+38%, +44% cc), mainly driven by higher net sales, partly offset by higher investments behind priority brands and launches. Net income was USD 3.6 billion (+34%, +37% cc), mainly driven by higher operating income, partly offset by higher income taxes. EPS was USD 1.83 (+40%, +42% cc), benefiting from the lower weighted average number of shares outstanding. Core operating income was USD 5.6 billion (+23%, +27% cc), mainly driven by higher net sales, partly offset by higher investments behind priority brands and launches, and R&D investments. Core operating income margin was 42.1% of net sales, increasing 3.7 percentage points (4.0 percentage points cc). Core net income was USD 4.5 billion (+22%, +26% cc), mainly due to higher core operating income. Core EPS was USD 2.28 (+27%, +31% cc), benefiting from the lower weighted average number of shares outstanding. Free cash flow amounted to USD 3.4 billion (+66% USD), compared with USD 2.0 billion in the prior-year quarter, driven by higher net cash flows from operating activities. Q1 priority brands Underpinning our financial results in the quarter is a continued focus on key growth drivers (ranked in order of contribution to Q1 growth) including: Entresto(USD 2 261 million, +22% cc) sustained robust, demand-led growth globally, including in China and Japan with increased penetration in hypertensionKisqali(USD 956 million, +56% cc) sales grew strongly across all regions, including +87% growth in the US with strong momentum from the recently launched early breast cancer indication as well as continued share gains in metastatic breast cancerKesimpta(USD 899 million, +43% cc) sales grew across all regions driven by increased demand and strong accessCosentyx(USD 1 534 million, +18% cc) sales grew mainly in the US, emerging growth markets and Europe, driven by recent launches as well as volume growth in core indicationsLeqvio(USD 257 million, +72% cc) continued steady growth, with a focus on increasing account and patient adoption, and continuing medical educationScemblix(USD 238 million, +76% cc) sales grew across all regions, demonstrating the continued high unmet need in CML and strong momentum from the recently launched early-line indication in the USFabhalta(USD 81 million) sales grew driven by continued launch execution across all markets in PNH as well as the recent launch in IgAN in the USPluvicto(USD 371 million, +21% cc) continued stable performance in the US and grew in Europe in the mCRPC post-taxane setting. With the FDA’s approval for earlier use before chemotherapy, which approximately triples the eligible patient population, the focus is on driving demand in established RLT sites while activating new sites and supporting referring providers to enable patient accessZolgensma(USD 327 million, +13% cc) sales grew as it continues to demonstrate strong performance in the incident populationLutathera(USD 193 million, +15% cc) sales grew mainly in the US, Europe and Japan due to increased demand and earlier line adoption particularly in the US and Japan Net sales of the top 20 brands in the first quarter Q1 2025 % change USD mUSDccEntresto2 2612022Cosentyx1 5341618Kisqali 9565256Kesimpta 8994143Tafinlar + Mekinist 5521619Promacta/Revolade 54658Jakavi 49237Xolair 4561419Ilaris 4191820Tasigna 377-5-2Pluvicto 3712021Zolgensma 3271113Sandostatin Group 317-11-9Leqvio 2577072Scemblix 2387576Lutathera 1931415Lucentis 189-40-38Exforge Group 179-7-1Diovan Group 150712Galvus Group 124-17-11Top 20 brands total10 8371719 R&D update - key developments from the first quarter New approvals Pluvicto(lutetium Lu177 vipivotide tetraxetan)FDA expanded the indication for Pluvicto to include patients with PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) who have been treated with an androgen receptor pathway inhibitor and are considered appropriate to delay chemotherapy, approximately tripling the eligible patient population. Vanrafia(atrasentan)FDA granted an accelerated approval for Vanrafia for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. Vanrafia can be seamlessly added to supportive care in IgAN and used as a foundational therapy.Fabhalta(iptacopan)Fabhalta was approved by the US FDA, European Commission and China’s NMPA for adult patients with C3 glomerulopathy (C3G), making it the first and only treatment approved for this condition in all three markets. Regulatory updates RemibrutinibRegulatory submissions for remibrutinib for the treatment of chronic spontaneous urticaria (CSU) were completed in the US, EU and China. A priority review voucher was used in the US and approval is anticipated in H2 2025, and priority review was granted in China. Scemblix(asciminib)Regulatory submission for Scemblix in adults with newly diagnosed CML was completed in the EU based on 96-week data from the Phase III ASC4FIRST study. Results from ongoing trials and other highlights OAV101 IT(onasemnogene abeparvovec)Novartis announced positive safety and efficacy results from the Phase III program for investigational intrathecal OAV101 IT in a broad population of patients aged two to <18 years with spinal muscular atrophy (SMA). In the Phase III STEER study, treatment with OAV101 IT led to a statistically significant and clinically meaningful 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded vs. 0.51 points in the sham control arm. In addition, in the Phase IIIb STRENGTH study, treatment with OAV101 IT in patients who have discontinued treatment with nusinersen or risdiplam demonstrated stabilization of motor function over 52 weeks of follow-up. OAV101 IT demonstrated a favorable safety profile, consistent in both treatment-naïve and treatment-experienced patients. Data were presented at MDA.RemibrutinibThe New England Journal of Medicine published data from the 24-week double-blind placebo-controlled period of the Phase III REMIX-1 and -2 studies. Remibrutinib showed early symptom improvement and sustained efficacy, with improvements in CSU symptoms compared to placebo observed as early as week 1 and response rate maintained through the double-blind treatment period. Remibrutinib was well tolerated, with overall adverse event rates comparable to placebo. Multiple analyses of the REMIX-1 and -2 trials were presented at medical congresses in Q1. At AAAAI, long-term results showed that patients experienced improved urticaria control within two weeks of starting treatment. Patients who switched from placebo to remibrutinib at week 24 achieved similar improvements through week 52. At AAD, data was presented showing that treatment with remibrutinib had positive effects on sleep and daily activities for CSU patients.IanalumabThe results of Phase II VAYHIT3 study in adult patients with advanced primary ITP, previously treated with at least a corticosteroid and a thrombopoietin receptor agonist, indicate that a short course of ianalumab has clinically meaningful efficacy and is well tolerated in these patients. These results will be presented at a future medical meeting and are expected to support a filing in second-line ITP, based on the Phase III VAYHIT2 study, expected to read out in H2 2025.Selected transactionsNovartis has completed the acquisition of Anthos Therapeutics, a clinical-stage biopharmaceutical company developing abelacimab, a potential first-in-class monoclonal antibody targeting the FXI inhibition pathway in development for the prevention of stroke and systemic embolism in patients with atrial fibrillation. The acquisition adds a Phase III asset and is aligned with the Novartis growth strategy and expertise in the cardiovascular therapeutic area. Capital structure and net debt Retaining a good balance between investment in the business, a strong capital structure, and attractive shareholder returns remains a priority. In Q1 2025, Novartis repurchased a total of 24.8 million shares for USD 2.6 billion on the SIX Swiss Exchange second trading line under the up-to USD 15 billion share buyback announced in July 2023 (with up to USD 2.7 billion still to be executed). In addition, 1.5 million shares (equity value of USD 0.2 billion) were repurchased from employees. In the same period, 10.5 million shares (equity value of USD 0.3 billion) were delivered to employees related to equity-based compensation plans. Novartis aims to offset the dilutive impact from equity-based compensation plans of employees over the remainder of the year. Consequently, the total number of shares outstanding decreased by 15.8 million versus December 31, 2024. These treasury share transactions resulted in an equity decrease of USD 2.5 billion and a net cash outflow of USD 2.7 billion. Net debt increased to USD 22.3 billion at March 31, 2025, compared with USD 16.1 billion at December 31, 2024. The increase was mainly due to the free cash flow of USD 3.4 billion being more than offset by the USD 5.3 billion annual net dividend payment in March (which is the gross dividend of USD 7.8 billion reduced by the USD 2.5 billion Swiss withholding tax that was paid in April 2025, according to its due date), cash outflows for treasury share transactions of USD 2.7 billion and cash outflows for purchases of intangible assets of USD 1.2 billion. As of Q1 2025, the long-term credit rating for the company is Aa3 with Moody’s Ratings and AA- with S&P Global Ratings. 2025 outlook Barring unforeseen events; growth vs. prior year in ccNet salesExpected to grow high single-digitCore operating incomeExpected to grow low double-digit Key assumptions: We assume Tasigna, Promacta and Entresto US generic entry mid-2025 for forecasting purposes Foreign exchange impact If late-April exchange rates prevail for the remainder of 2025, the foreign exchange impact for the year would be 0 percentage points on net sales and negative 2 percentage points on core operating income. The estimated impact of exchange rates on our results is provided monthly on our website. Key figures1 Q1 2025Q1 2024% change USD m USD mUSDccNet sales to third parties13 23311 8291215Operating income4 6633 3733844As a % of sales35.228.5 Net income3 6092 6883437EPS (USD)1.831.314042Net cash flows from operating activities3 6452 26561 Non-IFRS measures Free cash flow 3 3912 03866 Core operating income5 5754 5372327As a % of sales42.138.4 Core net income4 4823 6812226Core EPS (USD)2.281.802731 1. Constant currencies (cc), core results and free cash flow are non-IFRS measures. An explanation of non-IFRS measures can be found on page 31 of the Condensed Interim Financial Report. Unless otherwise noted, all growth rates in this Release refer to same period in prior year. Detailed financial results accompanying this press release are included in the Condensed Interim Financial Report at the link below:https://ml-eu.globenewswire.com/resource/download/f5843473-7c9e-4c13-b263-db739fdcf6d6/ DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995, that can generally be identified by words such as “anticipate,” “can,” “will,” “continue,” “ongoing,” “growth,” “launch,” “expect,” “expand,” “deliver,” “accelerate,” “deliver,” “guidance,” “outlook,” “priority,” “potential,” “momentum,” or similar expressions, or by express or implied discussions regarding potential new products, potential new indications for existing products, potential product launches, or regarding potential future revenues from any such products; or regarding results of ongoing clinical trials; or regarding potential future, pending or announced transactions; regarding potential future sales or earnings; or by discussions of strategy, plans, expectations or intentions, including discussions regarding our continued investment into new R&D capabilities and manufacturing; or regarding our capital structure. Such forward-looking statements are based on the current beliefs and expectations of management 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. You should not place undue reliance on these statements. There can be no guarantee that the 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. Neither can there be any guarantee that the expected benefits or synergies from the transactions described in this press release will be achieved in the expected timeframe, or at all. In particular, our expectations could be affected by, among other things: uncertainties concerning global healthcare cost containment, including ongoing government, payer and general public pricing and reimbursement pressures and requirements for increased pricing transparency; uncertainties regarding the success of key products, commercial priorities and strategy; uncertainties in the research and development of new products, including clinical trial results and additional analysis of existing clinical data; our ability to obtain or maintain proprietary intellectual property protection, including the ultimate extent of the impact on Novartis of the loss of patent protection and exclusivity on key products; uncertainties regarding our ability to realize the strategic benefits, operational efficiencies or opportunities expected from our external business opportunities; uncertainties in the development or adoption of potentially transformational digital technologies, including artificial intelligence, and business models; uncertainties surrounding the implementation of our new IT projects and systems; uncertainties regarding potential significant breaches of information security or disruptions of our information technology systems; uncertainties regarding actual or potential legal proceedings, including regulatory actions or delays or government regulation related to the products and pipeline products described in this press release; safety, quality, data integrity, or manufacturing issues; our performance on and ability to comply with environmental, social and governance measures and requirements; major macroeconomic and geo- and socio-political developments, including the impact of any potential tariffs on our products or the impact of war in certain parts of the world; uncertainties regarding future global exchange rates; uncertainties regarding future demand for our products; and other risks and factors referred to in Novartis AG’s most recently filed Form 20-F and in subsequent reports filed with, or furnished to, 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 as a result of new information, future events or otherwise. All product names appearing in italics are trademarks owned by or licensed to Novartis. 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 nearly 300 million people worldwide. Reimagine medicine with us: Visit us at https://www.novartis.com and connect with us on LinkedIn, Facebook, X and Instagram. Novartis will conduct a conference call with investors to discuss this news release today at 14:00 Central European time and 8:00 Eastern Time. A simultaneous webcast of the call for investors and other interested parties may be accessed by visiting the Novartis website. A replay will be available after the live webcast by visiting https://www.novartis.com/investors/event-calendar. Detailed financial results accompanying this press release are included in the Condensed Interim Financial Report at the link below. Additional information is provided on our business and pipeline of selected compounds in late-stage development. A copy of today's earnings call presentation can be found at https://www.novartis.com/investors/event-calendar. Important datesJuly 17, 2025October 28, 2025November 19-20, 2025Second quarter & half year 2025 resultsThird quarter & nine months 2025 resultsMeet Novartis Management 2025 (London, UK) Novartis Media RelationsE-mail: media.relations@novartis.com Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: investor.relations@novartis.com Please find full media release in English attached and on the following link: Media release (PDF) Further language versions are available through the following links: German version is available through the following link: Medienmitteilung (PDF)

The annual meeting of the American Society of Hematology was held from Dec. 7 to 10 in San Diego and attracted participants from around the world, including hematology specialists as well as clinical practitioners and other health care professionals. The conference featured presentations focusing on the diagnosis, treatment, and prevention of disorders affecting blood, bone marrow, and the immunologic, hemostatic, and vascular systems. In one study, Kristin A. Shimano, M.D., of the University of California, San Francisco, Benioff Children's Hospital, and colleagues found that for pediatric patients with newly diagnosed immune thrombocytopenia (ITP) within three months of diagnosis, eltrombopag leads to a more durable platelet response during weeks six 6 to 12 of treatment compared with standard treatments. The authors evaluated the use of eltrombopag (a thrombopoietin receptor agonist approved for persistent or chronic ITP) during the new-diagnosis phase and compared the responses between patients treated with eltrombopag and standard therapies (intravenous immunoglobulin, prednisone, and anti-D globulin). The researchers found that compared with those treated with standard therapies, a greater proportion of patients treated with eltrombopag had a platelet count of more than 50,000 during weeks 6 through 12 of the study without requiring a rescue therapy. "This has a huge potential to change our approach to the management of newly diagnosed pediatric ITP," Shimano said. "For patients with moderate bleeding or significant impact on quality of life due to their ITP, eltrombopag may be a good option to get the platelets to a more hemostatic level during the time that they have active ITP." Several authors disclosed financial ties to pharmaceutical and biotechnology companies, including Novartis, which manufactures eltrombopag and funded the study. Abstract No. 709 In another study, Rushad Patell, M.D., of Harvard Medical School in Boston, and colleagues found that the use of glucagon-like peptide-1 (GLP-1) receptor agonists leads to a significant reduction in the risk for blood clots among patients with type 2 diabetes. The authors compared patients who newly started a GLP-1 receptor agonist for type 2 diabetes to patients being prescribed another antidiabetic medication to assess rates of blood clots at one year. The researchers observed a 20 percent reduction in venous thromboembolism rates at 12 months. This benefit was seen across patients with different body mass index at the time of starting the study and included a breakdown for the two different types of blood clots (pulmonary embolism and deep vein thrombosis) when evaluated individually. "This was the first time this medication has been shown to impact venous thromboembolism to our knowledge," Patell said. "If these results are confirmed, they suggest that the use of these medications can have benefits beyond what has already been shown and can now include a reduction of blood clots." Two authors disclosed financial ties to the pharmaceutical and biotechnology industries. Abstract No. 701 Jenny Paredes, Ph.D., of the City of Hope National Medical Center in Duarte, California, and colleagues found that increasing dietary fiber intake is beneficial for the intestinal microbiome of allogeneic hematopoietic cell transplantation (allo-HCT) patients and could lead to an increase in overall survival and a reduction in acute graft-versus-host disease (GVHD). The authors assessed a preclinical mouse model of GVHD with defined diet fiber concentrations and also analyzed the dietary patterns of allo-HCT patients. The investigators found that dietary fiber intake in allo-HCT patients (days −7 to −30) positively correlated with higher overall survival, lower acute GVHD cumulative incidence, and lower gastrointestinal-GVHD cumulative incidence (landmark analysis to 24 months). In addition, fiber consumption positively correlated with microbial alpha-diversity, relative abundance of butyrate producers, and production of short chain fatty acids (SCFAs). In the murine model of GVHD post allo-HCT, the researchers determined that a diet rich in fiber (12 percent cellulose) increased survival to GVHD, led to a higher relative abundance of butyrate producers, and modulated gene expression associated with higher epithelial homeostasis and lower T-cell mediated inflammation (single cell sequencing). "Although follow-up studies on the strategies that can be used to increase dietary fiber in allo-HCT patients are in course, we would like to recommend reevaluating the nutritional plans and meals for allo-HCT patients and including multiple options of high fiber foods for every meal and beverage," Paredes said. "Increasing fiber consumption by 10 to 20 grams per day, based on our results, would lead to a more diverse microbiome, an increase in SCFA production, and consequently, to better overall survival (the Recommended Dietary Allowance for dietary fiber by the U.S. Department of Agriculture is 25 to 30 grams per day)." One author disclosed financial ties to the pharmaceutical and biotechnology industries. Abstract No. 259 ASH: Blinatumomab + Chemo Aids Survival in B-Cell Acute Lymphoblastic Leukemia THURSDAY, Dec. 12, 2024 -- For patients with newly diagnosed, standard-risk B-cell acute lymphoblastic leukemia with an average or high risk for relapse, adding blinatumomab to combination chemotherapy is associated with improved disease-free survival, according to a study published online Dec. 7 in the New England Journal of Medicine to coincide with the annual meeting of the American Society of Hematology, held from Dec. 7 to 10 in San Diego. Read Full Text ASH: Reduced-Dose Anticoagulants Not Noninferior for Recurrent VTE FRIDAY, Dec. 6, 2024 -- Noninferiority of reduced- versus full-dose anticoagulants cannot be proven in patients with venous thromboembolism who need extended anticoagulation, according to a study to be presented at the annual meeting of the American Society of Hematology, held from Dec. 7 to 10 in San Diego. Read Full Text ASH: Auto-HCT Not Beneficial for Mantle Cell Lymphoma With Undetectable Residual Disease FRIDAY, Dec. 6, 2024 -- Autologous hematopoietic cell transplant is not beneficial for patients with mantle cell lymphoma in first complete remission with undetectable minimal residual disease, according to a study to be presented at the annual meeting of the American Society of Hematology, held from Dec. 7 to 10 in San Diego. Read Full Text ASH: Complications Common With Controlled Ovarian Hyperstimulation in Sickle Cell Anemia THURSDAY, Nov. 14, 2024 -- Many individuals with sickle cell anemia undergoing controlled ovarian hyperstimulation with oocyte cryopreservation have complications, according to a study scheduled for presentation at the annual meeting of the American Society of Hematology, to be held from Dec. 9 to 12 in San Diego. Read Full Text ASH: GLP-1 RA Use Tied to Lower Rate of Venous Thromboembolism in Diabetes THURSDAY, Nov. 14, 2024 -- For patients with type 2 diabetes mellitus, glucagon-like peptide 1 receptor agonist use is associated with a lower risk for venous thromboembolism, according to a study scheduled for presentation at the annual meeting of the American Society of Hematology, to be held from Dec. 9 to 12 in San Diego. Read Full Text Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

Avatrombopag PDUFA date set for July 24, 2025WALTHAM, Mass., Dec. 12, 2024 (GLOBE NEWSWIRE) -- Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi®), today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for avatrombopag (DOPTELET®) for the treatment of thrombocytopenia in pediatric patients one year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a prior therapy. The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of July 24, 2025. The FDA also accepted Sobi’s New Drug Application (NDA) for avatrombopag for oral suspension. Avatrombopag for oral suspension is a capsule where its granules are sprinkled onto a soft food or liquid to prepare for administration. “These application acceptances for avatrombopag brings us one step closer to much-needed, additional treatment options for adolescents and children with persistent and chronic ITP,” said Jamie Freedman, MD, PhD, Chief Medical Officer, Sobi North America. “Now, there’s an opportunity to address an unmet need in our pediatric population, with the added bonus of an additional formulation, an oral suspension option that may be more suitable for some patients.” The application is based on results from the AVA-PED-301 study, evaluating the efficacy and safety of avatrombopag for the treatment of pediatric patients with ITP of at least 6 months’ duration. The study met the primary endpoint of durable platelet response defined as the proportion of patients achieving at least six out of eight weekly platelet counts ≥50×109/L during the last eight weeks of the 12-week treatment period in the core phase in the absence of rescue medication. The alternative primary efficacy endpoint of two consecutive PC ≥50× 109 /L was also met. ContactsFor details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media, click here. About immune thrombocytopeniaImmune thrombocytopenia (ITP) is an autoimmune disorder characterized by low numbers of platelets that results in an increased risk of bleeding. Symptoms can be highly variable and include bruising, a rash of small dots that represent small hemorrhages, bleeding from the gums or nose, blood in the urine/stools and debilitating fatigue. It is estimated that up to 100 people per million live with ITP. The disorder is considered chronic when symptoms last more than 12 months.i Long-term, primary chronic ITP is associated with increased risk of infections, bleeding episodes requiring hospitalization, hematologic malignancies, and mortality. No cure is available, and patients with chronic ITP usually relapse after various treatments and still require treatment to reduce the risk of clinically significant bleeding. About DOPTELET (avatrombopag)DOPTELET is an orally bioavailable, small molecule thrombopoietin receptor agonist (TPO-RA) that stimulates the proliferation and differentiation of megakaryocytes from bone marrow progenitor cells, resulting in increased production of platelets. DOPTELET does not compete with TPO for binding to the TPO receptor. INDICATION DOPTELET® (avatrombopag) is is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment. IMPORTANT SAFETY INFORMATION Thrombotic/Thromboembolic Complications. DOPTELET is a thrombopoietin (TPO) receptor agonist and TPO receptor agonists have been associated with thrombotic complications in patients with chronic liver disease (0.4%; (1/274) in DOPTELET-treated patients) and thromboembolic complications in patients with chronic immune thrombocytopenia (7%; (9/128) in DOPTELET-treated patients). Portal vein thrombosis has been reported in patients with chronic liver disease, and thromboembolic events (arterial and venous) have been reported in patients with chronic immune thrombocytopenia treated with TPO receptor agonists. Consider the potential increased thrombotic risk when administering DOPTELET to patients with known risk factors for thromboembolism, including genetic prothrombotic conditions and acquired risk factors. DOPTELET should not be administered to patients with chronic liver disease or chronic immune thrombocytopenia in an attempt to normalize platelet counts. Monitor platelet counts, and for signs and symptoms of thromboembolic events and institute treatment promptly. Serious Adverse Reactions Serious adverse reaction that occurred more frequently in patients treated with DOPTELET (9%; 12/128) compared to placebo (5%; 1/22) was headache, occurring in 1.6% (2/128). Adverse Reactions The most common adverse reactions (≥10%) in patients with chronic immune thrombocytopenia were headache, fatigue, contusion, epistaxis, upper respiratory tract infection, arthralgia, gingival bleeding, petechiae, and nasopharyngitis. About Sobi North America As the North American affiliate of international biopharmaceutical company Sobi, the Sobi North America team is committed to Sobi’s vision of being a leader in providing innovative treatments that transform lives for individuals with rare diseases. Our product portfolio includes multiple approved treatments focused on immunology, hematology and specialty care. With U.S. headquarters in the Boston area, Canadian headquarters in the Toronto area, and field sales, medical and market access representatives spanning North America, our growing team has a proven track record of commercial excellence. More information is available at https://www.sobi.com/usa or at www.sobi.com. About Sobi®Sobi is a specialized international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of hematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube.