Latest news with #hemophilia
Yahoo
01-08-2025
- Business
- Yahoo
NVO: Novo Nordisk Stock Climbs After Major FDA Approval
Aug 1 - Novo Nordisk (NYSE:NVO) gained nearly 3% in Friday morning trading after the company secured expanded U.S. approval for its Alhemo injection. The therapy can now help prevent or reduce bleeding episodes in adults and children 12 and older with hemophilia A or B. Warning! GuruFocus has detected 1 Warning Sign with NVO. Previously, Alhemo was only authorized for patients with hemophilia A or B who developed inhibitors. The new decision from the U.S. Food and Drug Administration followed a late?stage clinical trial that demonstrated the drug's safety and ability to lower bleeding risk in a broader patient group. Analysts say this approval could significantly expand Alhemo's market potential. The hemophilia space is becoming increasingly competitive with gene therapies and new clotting factor treatments, but Novo Nordisk's broader label should reinforce its rare disease portfolio. The company expects Alhemo to contribute to revenue growth in the second half of 2025 as more patients gain access. Investors will watch whether this momentum continues into upcoming quarters as the therapy reaches a larger base of hemophilia patients. This article first appeared on GuruFocus. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
05-07-2025
- Business
- Yahoo
Pfizer Advances HYMPAVZI After Successful Hemophilia Trial Results
Pfizer Inc. (NYSE:PFE) ranks among the best set-it-and-forget-it stocks to buy. On June 26, Pfizer Inc. (NYSE:PFE) declared that the Phase 3 BASIS research results showed that its medication HYMPAVZI (marstacimab) was superior to other inhibitors in treating hemophilia A or B patients. Pixabay/Public Domain Comparing HYMPAVZI to on-demand treatment, the trial revealed that the former decreased the annualized bleeding rate by 93%. A pre-filled pen was used to administer HYMPAVZI once weekly via subcutaneous injection, requiring little preparation. During the study, no thromboembolic events or deaths were reported, indicating that the medication was generally well tolerated. Pfizer Inc. (NYSE:PFE) intends to present the data to regulatory bodies in order to start the regulatory filing process for HYMPAVZI in hemophilia patients with inhibitors. The medication has already been approved by regulators in the United States and Europe for use in hemophilia patients without inhibitors. Pfizer Inc. (NYSE:PFE) is a leading global pharmaceutical company that develops, produces, and sells biopharmaceutical medicines worldwide. While we acknowledge the potential of PFE as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. Read More: and Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Globe and Mail
04-07-2025
- Business
- Globe and Mail
Will Novo Nordisk's Rare Disease Bets Reduce GLP-1 Reliance?
Novo Nordisk NVO has experienced remarkable growth in recent years, largely fueled by the commercial success of its blockbuster semaglutide (GLP-1 RA)-based therapies, Wegovy for obesity and Ozempic for type II diabetes (T2D). While maintaining its leadership in metabolic diseases, the company is also making significant efforts to diversify its portfolio, particularly in rare blood disorders. NVO's key marketed hemophilia therapies include NovoSeven and Esperoct, which generate incremental revenues for the company. NovoSeven is approved in the United States and the EU for several bleeding disorders, including hemophilia A or B with inhibitors, Factor VII deficiency, and Glanzmann's thrombasthenia. Esperoct is approved for treating Hemophilia A in both adults and children. Recently, Novo Nordisk's Alhemo was approved in the EU as a once-daily subcutaneous prophylactic treatment for patients aged 12 years or older living with haemophilia A or B with inhibitors. However, the drug is not currently approved in the United States. NVO is also currently evaluating Mim8 in a late-stage program (FRONTIER) as a prophylaxis treatment for people with hemophilia A with or without inhibitors in patients aged 12 and older. Data readouts from the program have shown superior efficacy of the candidate, along with a favorable safety profile. Based on the findings, Novo Nordisk anticipates submitting Mim8 for regulatory review in 2025. The company is also investigating semaglutide's potential in metabolic dysfunction–associated steatohepatitis (MASH) resolution, expecting to file for regulatory approvals in the United States and the EU in 2025. Subject to approval, these candidates will further contribute to the top line. Other Pharma Giants Diversifying Beyond Flagship Drugs Novo Nordisk's arch-rival, Eli Lilly LLY, generates most of its revenues from the sale of its tirzepatide medicines, Mounjaro for T2D, and Zepbound for obesity. However, Lilly also boasts a wide range of products that serve other therapeutic areas, such as neuroscience, oncology and immunology — all high-growth areas and representing significant commercial potential. Some key growth products include the oncology drug Verzenio and immunology drug Taltz, which accounted for 9% and 6% of total first-quarter sales, respectively. Over the past couple of years, LLY's new drugs, including Omvoh and Ebglyss in immunology, Jaypirca in oncology and Kisunla in neuroscience, have all been contributing to its top-line growth. Lilly expects the potential launch of new medicines like imlunestrant for metastatic breast cancer to contribute to growth in 2025. Another large-cap drugmaker, Merck MRK, is also making significant efforts in broadening its therapeutic reach beyond core revenue drivers. MRK's flagship oncology drug Keytruda, which accounts for nearly half of its total revenues, is expanding its late-stage pipeline to reduce dependence on the product. We believe new products like Capvaxive (pneumococcal vaccine) and Winrevair (PAH drug) have the potential to generate significant revenues over the long term. Merck recently secured the FDA's approval for long-acting RSV antibody Enflonsia for newborns, which is expected to launch later this month. NVO's Stock Price, Valuation, Estimates Year to date, Novo Nordisk shares have lost 19.6% compared with the industry 's 0.3% decline. The company has also underperformed the sector and the S&P 500 during the same time frame, as seen in the chart below. NVO Stock Underperforms the Industry, Sector & the S&P 500 Novo Nordisk is trading at a premium to the industry, as seen in the chart below. Going by the price/earnings ratio, the company's shares currently trade at 16.31 forward earnings, which is higher than 15.09 for the industry. However, the stock is trading much below its five-year mean of 29.25. NVO Stock Valuation Image Source: Zacks Investment Research Earnings estimates for 2025 have improved from $3.89 to $3.93 per share over the past 60 days. During the same time frame, Novo Nordisk's 2026 earnings per share estimates have decreased from $4.76 to $4.58. NVO Estimate Movement The stock's return on equity on a trailing 12-month basis is 80.95%, which is higher than 33.55% for the large drugmaker industry, as seen in the chart below. NVO Return on Equity Novo Nordisk currently carries a Zacks Rank #3 (Hold). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. 7 Best Stocks for the Next 30 Days Just released: Experts distill 7 elite stocks from the current list of 220 Zacks Rank #1 Strong Buys. They deem these tickers "Most Likely for Early Price Pops." Since 1988, the full list has beaten the market more than 2X over with an average gain of +23.5% per year. So be sure to give these hand picked 7 your immediate attention. See them now >> Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Novo Nordisk A/S (NVO): Free Stock Analysis Report Merck & Co., Inc. (MRK): Free Stock Analysis Report Eli Lilly and Company (LLY): Free Stock Analysis Report
Yahoo
26-06-2025
- Health
- Yahoo
Pfizer Announces Positive Topline Phase 3 Results for HYMPAVZI™ in Hemophilia A or B with Inhibitors
Study demonstrates superiority, showing both statistically significant and clinically meaningful reduction in annualized bleeding rate with a generally well-tolerated safety profile compared to on-demand treatment in patients 12 years and older HYMPAVZI was administered with a straightforward, once-weekly subcutaneous injection that required minimal preparation NEW YORK, June 26, 2025--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) today announced positive topline results from the Phase 3 BASIS study (NCT03938792) evaluating HYMPAVZI™ (marstacimab) for adults and adolescents living with hemophilia A or B with inhibitors. The study met the primary endpoint and key secondary bleeding endpoints demonstrating the superiority of once-weekly subcutaneous HYMPAVZI in improving key bleeding outcomes compared to on-demand treatment in a patient population where less burdensome treatment approaches are needed.1,2,3,4 Inhibitors, or antibodies, which neutralize factor replacement therapies and render them ineffective, may develop in people living with hemophilia.5 Inhibitors can be diagnosed with a blood test.6 Of the more than 800,000 people in the world living with hemophilia A or hemophilia B, approximately 20% of people with hemophilia A and 3% of people with hemophilia B are unable to continue taking factor replacement therapies because they develop inhibitors to FVIII (Factor VIII) and FIX (Factor IX) and these therapies no longer prevent or stop bleeding episodes.6,7 "Patients with inhibitors tend to face frequent complications, and navigating the treatment landscape can introduce complexities and increase disease burden,"1,2,3,4,8 said Davide Matino, M.D., BASIS Principal Investigator, Associate Professor of Medicine, McMaster University. "The strong bleed reduction with HYMPAVZI compared to on-demand treatment in the Phase 3 BASIS study, coupled with its weekly administration method, offers exciting potential for these patients who are in critical need of treatment options." The BASIS trial demonstrated that prophylactic treatment with HYMPAVZI resulted in a statistically significant and clinically relevant reduction in annualized bleeding rate (ABR) of treated bleeds in people living with severe hemophilia A or hemophilia B with inhibitors. Forty-eight people living with hemophilia were treated with HYMPAVZI during a 12-month period versus an on-demand intravenous regimen with bypassing agents, administered as part of usual care in the six-month lead-in period. HYMPAVZI was superior to on-demand treatment with a 93% reduction in ABR over 12 months (ABR 1.39 vs ABR on-demand 19.78; p < 0.0001). Superiority of HYMPAVZI was also demonstrated across all bleeding-related secondary endpoints—spontaneous bleeds, joint bleeds, target joint bleeds, and total bleeds. HYMPAVZI was generally well-tolerated, consistent with the non-inhibitor cohort of the BASIS study and Phase 1/2 results. No deaths or thromboembolic events were reported. "These encouraging results demonstrate HYMPAVZI's potential to help people living with hemophilia A or B with inhibitors, meeting an important need for patients with antibodies that neutralize most factor-based prophylactic options used to manage bleeding episodes," said Michael Vincent, M.D., Ph.D., Chief Inflammation & Immunology Officer, Pfizer. "HYMPAVZI represents Pfizer's latest contribution in more than 40 years of working to advance hemophilia care, as a generally well-tolerated treatment option that could offer bleed protection with a straightforward, once-weekly subcutaneous administration in a pre-filled pen for patients with inhibitors, if approved in this patient population." Analyses of the full Phase 3 dataset from the inhibitor cohort of the BASIS study are ongoing, and additional data will be presented at upcoming medical meetings. Pfizer plans to discuss these data with regulatory authorities, with the goal of initiating regulatory filings for HYMPAVZI for the treatment of patients living with hemophilia with inhibitors. Discovered by Pfizer scientists, HYMPAVZI has a mechanism of action that is differentiated from FVIII and FIX replacement treatments. Instead of replacing missing or insufficient clotting factors, HYMPAVZI is intentionally designed to target tissue factor pathway inhibitor (TFPI), one of the body's natural mechanisms that inhibits the initiation of blood clotting. By targeting the Kunitz 2 domain of TFPI, HYMPAVZI may help re-establish balance between bleeding and blood clot formation with the goal of offering a combination of bleed protection, good tolerability, and straightforward administration. About the BASIS study The pivotal BASIS study is a global Phase 3, open-label, multicenter study to evaluate the efficacy and safety of HYMPAVZI in adolescent and adult participants ages 12 to <75 years with severe hemophilia A (defined as FVIII <1%) or moderately severe to severe hemophilia B (defined as FIX activity ≤2%) with or without inhibitors. This cohort included 48 people living with hemophilia with inhibitors who were treated with HYMPAVZI during a 12-month active treatment period (ATP) versus an on-demand intravenous regimen with bypassing agents, administered as part of usual care in a six-month observational period. During the ATP, participants received prophylaxis (a 300 mg subcutaneous loading dose of HYMPAVZI, followed by 150 mg subcutaneously once weekly) with potential for dose escalation to 300 mg once weekly. An additional three patients in the inhibitor cohort were on routine prophylactic treatment prior to the study and not included in the primary efficacy analysis. The primary endpoint measures the treated ABR during the 12-month ATP with HYMPAVZI compared to treated ABR on prior on-demand replacement therapy. For further information, visit About HYMPAVZI HYMPAVZI has received regulatory approvals in the U.S. and in Europe for eligible patients living with hemophilia A without factor VIII inhibitors, or hemophilia B without factor IX inhibitors. HYMPAVZI was the first anti-TFPI approved in the U.S. and EU for the treatment of hemophilia A or B and the first hemophilia medicine approved in the U.S. and EU to be administered via a pre-filled, auto-injector pen. For eligible people living with hemophilia B, it is the first once-weekly subcutaneous prophylactic treatment. HYMPAVZI can offer a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each individual administration. Pfizer is also conducting BASIS KIDS, an open-label study investigating the safety and efficacy of HYMPAVZI in children 1 to <18 years of age with severe hemophilia A or moderately severe to severe hemophilia B with or without inhibitors. About Hemophilia Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (FVIII in hemophilia A, FIX in hemophilia B), which prevents normal blood clotting. Hemophilia is diagnosed in early childhood and impacts more than 800,000 people worldwide.7 The inability of the blood to clot properly can increase the risk of painful bleeding inside the joints, which can cause joint scarring and damage. People living with hemophilia can suffer permanent joint damage following repeated bleeding episodes.9,10 For decades, the most common treatment approach for hemophilia A and B has been factor replacement therapy, which replaces the missing clotting factors. Factor replacement therapies increase the amount of clotting factor in the body to levels that improve clotting, resulting in less bleeding. The burden of intravenous infusions is believed to be a barrier to treatment adherence for some people living with hemophilia due in part to inconvenience, time constraints, and poor venous access.11 Approximately 20% of people with hemophilia A and 3% of people with hemophilia B are unable to continue taking factor replacement therapies because they develop inhibitors to FVIII and FIX.6 These patients often have higher treatment burden, including potential complications from bleeding such as hospitalization and death, as well as higher treatment-related costs.1,2,3,4,12 HYMPAVZI (marstacimab) U.S. Important Safety Information Important: Before you start using HYMPAVZI, it is very important to talk to your healthcare provider about using factor VIII and factor IX products (products that help blood clot but work in a different way than HYMPAVZI). You may need to use factor VIII or factor IX medicines to treat episodes of breakthrough bleeding during treatment with HYMPAVZI. Carefully follow your healthcare provider's instructions regarding when to use factor VIII or factor IX medicines and the prescribed dose during your treatment with HYMPAVZI. Before using HYMPAVZI, tell your healthcare provider about all of your medical conditions, including if you: have a planned surgery. Your healthcare provider may stop treatment with HYMPAVZI before your surgery. Talk to your healthcare provider about when to stop using HYMPAVZI and when to start it again if you have a planned surgery. have a severe short-term (acute) illness such as an infection or injury. are pregnant or plan to become pregnant. HYMPAVZI may harm your unborn who are able to become pregnant: Your healthcare provider will do a pregnancy test before you start your treatment with HYMPAVZI. You should use effective birth control (contraception) during treatment with HYMPAVZI and for at least 2 months after the last dose of HYMPAVZI. Tell your healthcare provider right away if you become pregnant or think that you may be pregnant during treatment with HYMPAVZI. are breastfeeding or plan to breastfeed. It is not known if HYMPAVZI passes into your breast milk. Tell your healthcare provider about all the medicines you take, including prescription medicines, over-the-counter medicines, vitamins, and herbal supplements. What are the possible side effects of HYMPAVZI? HYMPAVZI may cause serious side effects, including: blood clots (thromboembolic events). HYMPAVZI may increase the risk for your blood to clot. Blood clots may form in blood vessels in your arm, leg, lung, or head and can be life-threatening. Get medical help right away if you develop any of these signs or symptoms of blood clots: swelling or pain in arms or legs redness or discoloration in your arms or legs shortness of breath pain in chest or upper back fast heart rate cough up blood feel faint headache numbness in your face eye pain or swelling trouble seeing allergic reactions. Allergic reactions, including rash and itching have happened in people treated with HYMPAVZI. Stop using HYMPAVZI and get medical help right away if you develop any of the following symptoms of a severe allergic reaction: swelling of your face, lips, mouth, or tongue trouble breathing wheezing dizziness or fainting fast heartbeat or pounding in your chest sweating The most common side effects of HYMPAVZI are injection site reactions, headache, and itching. These are not all the possible side effects of HYMPAVZI. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088. The full Prescribing Information can be found here. About Pfizer: Breakthroughs That Change Patients' Lives At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For 175 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at In addition, to learn more, please visit us on and follow us on X at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Disclosure notice The information contained in this release is as of June 26, 2025. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments. This release contains forward-looking information about HYMPAVZI™ (marstacimab), an anti-tissue factor pathway inhibitor, including its potential benefits and plans to discuss the Phase 3 BASIS data with regulatory authorities with the goal of initiating regulatory filings for HYMPAVZI for the treatment of patients living with hemophilia with inhibitors, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of HYMPAVZI; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any applications may be filed with regulatory authorities in particular jurisdictions for HYMPAVZI for any potential indication; whether and when any such applications that may be pending or filed for HYMPAVZI may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether HYMPAVZI will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of HYMPAVZI; risks and uncertainties related to issued or future executive orders or other new, or changes in, laws or regulations; uncertainties regarding the impact of COVID-19 on our business, operations and financial results; and competitive developments. A further description of risks and uncertainties can be found in Pfizer's Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned "Risk Factors" and "Forward-Looking Information and Factors That May Affect Future Results", as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at and 1 Shapiro AD, Ragni MV, Borhany M, et al. Natural history study of factor IX deficiency with focus on treatment and complications (B-Natural). Haemophilia. 2021;27(1):49-59. doi: 10.1111/hae.14139.2 Valentino LA, Ewenstein B, Navickis RJ, Wilkes MM. Central venous access devices in haemophilia. Haemophilia. 2004;10(2):134-46. doi: 10.1046/j.1365-2516.2003.00840.x.3 Nugent D, Kalnins W, Querol F, et al. Haemophilia Experiences, Results and Opportunities (HERO) study: Treatment-related characteristics of the population. Haemophilia. 2015;21(1):e26-38. doi: 10.1111/hae.12545.4 Soucie JM, Symons Jt, Evatt B, Brettler D, Huszti H, Linden J. Home-based factor infusion therapy and hospitalization for bleeding complications among males with haemophilia. Haemophilia. 2001;7(2):198-206. doi: 10.1046/j.1365-2516.2001.00484.x.5 Teiu P, Chan A, Matino D. Molecular Mechanisms of Inhibitor Development in Hemophilia. Mediterr J Hematol Infect Dis. 2020 Jan 1;12(1):e2020001. doi: 10.4084/MJHID.2020.001.6 Centers of Disease Control and Prevention. Testing for Inhibitors and Hemophilia. Accessed May 14, 2025. Available at: World Federation of Hemophilia. World Federation of Hemophilia Global Report on the Annual Global Survey 2022. Meeks S, Batsuli G. Hemophilia and inhibitors: Current treatment options and potential new therapeutic approaches. Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):657–662. doi:10.1182/asheducation-2016.1.657.9 Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd Edition; 2020. Haemophilia. 26(S6), 1–158. Franchini M, Mannucci PM. Past, present and future of hemophilia: A narrative review. Orphanet J Rare Dis. 2012;7:24. Weyand AC, Pipe SW. New therapies for hemophilia. Blood. 2019;133(5):389–398. Walsh CE, Soucie JM, Miller CH. Impact of inhibitors on hemophilia a mortality in the United States. Am. J. Hematol. 2015; 90: 400-405. Category: Medicines View source version on Contacts Media Contact:+1 (212) 733-1226PfizerMediaRelations@ Investor Contact:+1 (212) 733-4848IR@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Reuters
26-06-2025
- Health
- Reuters
Pfizer's bleeding disorder therapy meets main goal in patients with certain antibodies
June 26 (Reuters) - Pfizer (PFE.N), opens new tab said on Thursday its hemophilia therapy, Hympavzi, met the main goal of a late-stage study in patients with certain types of antibodies. The data comes months after Pfizer said it will stop global development and sale of its hemophilia gene therapy, Beqvez, citing soft demand from patients and their doctors. A weekly injection, Hympavzi, is approved for patients with hemophilia A without factor VIII antibodies or inhibitors, or hemophilia B without factor IX inhibitors. People with hemophilia have a defect in a gene that regulates the production of proteins called clotting factors, causing spontaneous and severe bleeding following injuries or surgery. Pfizer said patients with inhibitors require less burdensome treatments. The hemophilia market has been dominated by factor replacement therapies for decades. The company said around 20% of people with hemophilia A and 3% of people with hemophilia B are unable to continue factor replacement therapies as they develop antibodies and the therapies fail to prevent or stop bleeding episodes.
