Latest news with #Qfitlia
Time Business News
24-06-2025
- Health
- Time Business News
RNAi Crucial Aspects and Innovations in the RNAi Technology Market
RNA interference (RNAi) is biological process where RNA molecules inherit gene expression or translation by neutralizing targeted mRNA molecules. RNAi is crucial in regulating genes and virus protection in eukaryotic organisms. A growing prevalence of genetic disorders, rising need for therapies that target cancer, growing emphasis on neurological disorders, growing RNA interference usage in viral and infectious disease treatments, increased research and development and innovation in delivery technologies regarding RNAi technology market. Developmental regulators occur naturally as hormones or proteins which foster cellular differentiation, development, proliferation, and healing. These cells are essential for immune system control, tissue repair, and development. Key Growth Drivers and Opportunities Genetic Disorder Cases Spike: As genetic disorders become more prevalent, there is increasing demand for enhanced and specialized treatments. One of the most promising methods in this area is RNA interference (RNAi) technology. With given factors the RNAi technology will possibly be surged, generating investments, research and development and end-up with more extensive use for genetic disorder treatment. Advancement in Delivery Methods: Delivery method advancements include nanotechnology-based delivery systems and non-transgenic delivery technologies, and are estimated to mitigate delivery issues of RNAi therapeutics. Ongoing research is focusing on studying the mechanisms OD siRNA delivery and catalytic RNA vehicle development to make RNAi based cancer gene therapy having more impact. Challenges The Off-target implications which result when RNAi treatments accidently alter genes other than the intended target, restrict growth in markets. Off target effects or phenotypic results are caused by an RNAi reagent's elimination of unintended genes rather that the gene of interest. Accidental gene suppression is often referred to as an off-target effect. It develops when the RNAi molecule attaches itself to something that is not intended. Innovation and Expansion F FDA Approves Qfitlia (fitusiran), the First siRNA (RNAi Therapeutic) for the Treatment of Hemophilia A or B In March 2025, Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, highlighted the significance of the U.S. Food and Drug Administration's (FDA) approval of Qfitlia (fitusiran), the sixth Alnylam-discovered RNAi therapeutic approved in the U.S., and the first and only therapeutic to lower antithrombin (AT), a protein that inhibits blood clotting, with the goal of promoting thrombin generation to rebalance hemostasis and prevent bleeds. Alnylam's vutrisiran shows positive Phase 3 results in ATTR cardiomyopathy In June 2024, RNAi drug vutrisiran was announced by Alnylam that reduces mortality and recurrent cardiovascular events by up to 33% in patients with transthyretin amyloidosis–related cardiomyopathy, clearing the way for an upcoming NDA submission. RNAi Therapies emerge for Hereditary Transthyretin Amyloidosis (ATTR) In April 2025, The Yuzu Matcha Martini has become a new ready-to-drink bottle that was introduced by world-renowned bartender Hidetsugu Ueno in collaboration with On the Rocks Cocktails and The House of Suntory. The bottle is matched with the Japanese Boulevardier, a traditional whiskey cocktail with an Eastern touch, so it's not simply a matcha martini to add to the family's bar. Four RNAi drugs show >80% Reduction in Lipoprotein (a) In August 2024, Early-phase trials of four RNAi-based therapeutics have managed to lower plasma Lp(a) by more than 80%, and Phase 3 studies are now being conducted to assess their impact on cardiovascular outcomes. Combination mRNA+RNAi Nanoparticle Therapy for Complex Diseases In December 2024, Codedeliry of mRNA and siRNA via nanoparticles can simultaneously boost positive gene expression and mute detrimental genes, revealed to research from the University of Ottawa. This opens up fresh therapies for a range of ailments, notably cancer and heart disorders. Inventive Sparks, Expanding Markets Companies' actives in Rnai technology market includes Alnylam Pharmaceuticals, Inc., ARROWHEAD PHARMACEUTICALS, INC., Silence Therapeutics, Thermo Fisher Scientific Inc., Revvity Discovery Limited, Company, and among others. Companies for RNAi technology aim on developing safe, precise, and efficient gene-silencing therapies for broad range of rare and genetic diseases. Their goals involve getting regulatory clearances for additional indications, refining delivery methods, minimizing off-target effects, and expanding the application of RNA interference beyond liver-targeted therapies. About Author: Prophecy is a specialized market research, analytics, marketing and business strategy, and solutions company that offer strategic and tactical support to clients for making well-informed business decisions and to identify and achieve high value opportunities in the target business area. Also, we help our client to address business challenges and provide best possible solutions to overcome them and transform their business. TIME BUSINESS NEWS
Yahoo
28-03-2025
- Health
- Yahoo
US FDA approves Sanofi's bleeding disorder therapy
By Sriparna Roy and Padmanabhan Ananthan (Reuters) -The U.S. Food and Drug Administration approved French drugmaker Sanofi's hemophilia therapy, paving the way for a new type of treatment for patients with the rare blood clotting disorder, the regulator said on Friday. US-listed shares of Paris-based Sanofi were up 1.7%. Sanofi's subcutaneous, or under-the-skin, therapy helps prevent bleeding and lowers antithrombin, a protein that delays blood clotting, for hemophilia patients aged 12 years and older. The therapy Qfitlia is a first-in-class for people with hemophilia A or B, with or without inhibitors, given once every two months, compared to other treatments such as Pfizer's weekly injection Hympavzi and Novo Nordisk's daily Alhemo. "If you can take a drug every other month, that's also an incredible simplification and improvement in your quality of life," said Margaret Ragni, professor at the University of Pittsburgh. The hemophilia market has been dominated by factor replacement therapies for decades. However, recently, gene therapies such as CSL's Hemgenix and BioMarin Pharmaceutical's Roctavian have been approved as an alternative in the United States. People with hemophilia have a fault in a gene that regulates the production of proteins called clotting factors, causing spontaneous and severe bleeding following injuries or surgery. Hemophilia A is caused by a lack of blood clotting factor VIII, while those with hemophilia B lack factor IX. As many as 33,000 males are estimated to be living with hemophilia in the United States, according to the U.S. Centers for Disease Control and Prevention. In trials, Qfitlia reduced annualized bleeding rates by 90%, compared to the control arms, showing a meaningful improvement in bleeding episodes over on-demand treatments.
Yahoo
28-03-2025
- Health
- Yahoo
Press Release: Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors
Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors Qfitlia (fitusiran), the first antithrombin-lowering therapy in hemophilia, offers consistent protection with as few as six injections a year via a prefilled pen or vial and syringe Unique mechanism helps reduce the frequency of bleeding episodes for people with hemophilia Paris, March 28, 2025. The US Food and Drug Administration (FDA) has approved Qfitlia (fitusiran), the first antithrombin-lowering (AT) therapy for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B with or without factor VIII or IX inhibitors. The approval is based on data from the ATLAS phase 3 studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR) across hemophilia patients with or without inhibitors. President and CEO, National Bleeding Disorders Foundation 'Current treatment options can make people with hemophilia feel they need to choose between effective bleed control and convenient dosing schedules, leading to trade-offs when it comes to disease management. Qfitlia takes a novel approach to providing protection for people living with hemophilia while reducing the frequency of dosing for patients and their families.' By lowering AT, a protein that inhibits blood clotting, Qfitlia helps increase thrombin generation to restore hemostasis in people with hemophilia. Qfitlia uses small-interfering RNA technology, which enables low treatment frequency, subcutaneous dosing, and low volume injections. Executive Vice President, Head of Specialty Care, Sanofi'This approval highlights our commitment to advancing innovation and improving care for the rare blood disorders community. Qfitlia has the potential to meaningfully change the hemophilia landscape through effective bleed protection, infrequent dosing, and simplified administration. Our robust portfolio of hemophilia treatment options continues to grow as we focus on offering protection with reduced treatment burden that best fits an individual's needs.' Director, Hemostasis and Thrombosis Center at Children's Hospital, Los Angeles'Qfitlia delivers the fewest doses of any prophylactic therapy in hemophilia, and its unique mechanism allows it to be used to treat all types of hemophilia, including with inhibitors and hemophilia B, where unmet medical needs remain. By targeting antithrombin, which can be reliably measured with an FDA-cleared blood assay, Qfitlia is proven to help rebalance hemostasis and improve bleed rates and protection.' In the ATLAS clinical development program, Qfitlia demonstrated low bleed rates across subgroups with as few as six injections a year. Key results include: Significant bleed reduction by 71% in ABR for patients without inhibitors treated with Qfitlia prophylaxis compared to clotting factor concentrate on-demand (estimated mean: ABR 9.0 vs. 31.4, respectively; p<0.0001) and by 73% in ABR compared to bypassing agent on-demand for patients with inhibitors (estimated mean: ABR 5.1 vs. 19.1, respectively; p=0.0006) Median observed ABR during the open-label extension study was 3.8 (IQR: 0.0–11.2) in patients without inhibitors and 1.9 (IQR: 0.0–5.6) in patients with inhibitors Median observed annualized spontaneous bleeding rate during the open-label extension study was 1.9 (interquartile range (IQR): 0.0-7.5) in patients without inhibitors and 1.9 (IQR: 0.0-3.7) in patients with inhibitors Nearly half of patients in the open-label extension study experienced one or fewer bleeds (31% 0 bleeds and 47% 0-1 bleeds) There is also the potential for significant adverse reactions, including thrombotic events, acute and recurrent gallbladder disease, and hepatotoxicity. The most common adverse reactions (incidence >10%) are viral infection, nasopharyngitis, and bacterial infection. In conjunction with the Qfitlia approval, the FDA also cleared the Siemens Healthineers' INNOVANCE® Antithrombin assay as a companion diagnostic for Qfitlia to measure AT levels. Through the Qfitlia Testing Program with Labcorp, the FDA-cleared companion diagnostic will be available to patients prescribed Qfitlia to measure AT levels at no cost. Qfitlia can offer the fewest doses of all prophylactic therapies, and it will have a comparable price to other prophylactic hemophilia treatments. HemAssist is launching alongside Qfitlia to provide comprehensive patient support services, including insurance and financial assistance as well as educational resources. This program is for patients prescribed Qfitlia or other hemophilia treatments from Sanofi's portfolio. The FDA granted Qfitlia Orphan Drug Designation for hemophilia A and B, Fast Track Designation for hemophilia A and B with and without factor VIII or IX inhibitors, and Breakthrough Therapy Designation for hemophilia B with factor IX inhibitors. A regulatory submission for Qfitlia for the treatment of hemophilia A or B in adults and adolescents with or without inhibitors is under review in Brazil. A regulatory decision is expected in China in the second half of 2025. About hemophiliaHemophilia A and B are rare, congenital, lifelong, bleeding disorders in which the ability of a person's blood to clot is impaired, leading to excessive bleeds and spontaneous bleeds into joints that can result in joint damage and chronic pain, and significantly impact quality of life. Hemophilia A and B are caused by a deficiency of factor VIII and IX, respectively, resulting in insufficient thrombin generation and ineffective clot formation, which is further complicated in people who develop inhibitors to their factor treatment. About the ATLAS clinical development programThe efficacy and safety of Qfitlia is being investigated in the ATLAS clinical development program. The program includes completed phase 3 studies ATLAS-INH (NCT03417102), ATLAS-A/B (NCT03417245), and ATLAS-PPX (NCT03549871). There are three ongoing phase 3 studies ATLAS-NEO (NCT05662319), ATLAS-PEDS (NCT03974113), and ATLAS OLE (NCT03754790). The ongoing ATLAS-OLE study is a single-arm, phase 3, open-label study evaluating the safety and efficacy of Qfitlia with a revised AT-based dosing regimen (AT-DR), which was designed to maintain an AT target range of 15%-35% in patients who have completed a prior phase 3 ATLAS clinical trial. This study includes lower doses and less-frequent dosing than earlier studies of Qfitlia. The efficacy of Qfitlia AT-DR treatment was assessed by comparing the AT-DR treatment data from ATLAS-OLE to the control data from studies ATLAS-INH and ATLAS-A/B. The analyses follow the intent to treat principle. About QfitliaQfitlia (fitusiran) is a first-in-class AT lowering therapy approved by the US FDA for prophylactic treatment of adults and pediatric patients (aged 12 and older) living with hemophilia A and B with or without factor VIII or IX inhibitors, and is administered via subcutaneous injection with a convenient, prefilled pen for the 50 mg dose. Qfitlia prevents bleeds and helps rebalance hemostasis by lowering AT, a protein that inhibits blood clotting, to promote thrombin generation. Qfitlia is a small interference RNA therapeutic that utilizes Alnylam Pharmaceutical Inc.'s ESC-GalNAc conjugate technology. About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people's lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our is listed on EURONEXT: SAN and NASDAQ: SNY Media RelationsSandrine Guendoul | +33 6 25 09 14 25 | Evan Berland | +1 215 432 0234 | Nicolas Obrist | +33 6 77 21 27 55 | Victor Rouault | +33 6 70 93 71 40 | Gilbert | +1 516 521 2929 | Investor RelationsThomas Kudsk Larsen |+44 7545 513 693 | Alizé Kaisserian | +33 6 47 04 12 11 | Lauscher | +1 908 612 7239 | Keita Browne | +1 781 249 1766 | Pham | +33 7 85 93 30 17 | Elgoutni | +1 617 710 3587 | Thibaud Châtelet | +33 6 80 80 89 90 | Sanofi forward-looking statementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates regarding the marketing and other potential of the product, or regarding potential future revenues from the product. Forward-looking statements are generally identified by the words 'expects', 'anticipates', 'believes', 'intends', 'estimates', 'plans', and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, unexpected regulatory actions or delays, or government regulation generally, that could affect the availability or commercial potential of the product, the fact that product may not be commercially successful, the uncertainties inherent in research and development, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general, risks associated with intellectual property and any related future litigation and the ultimate outcome of such litigation, and volatile economic and market conditions, and the impact that global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under 'Risk Factors' and 'Cautionary Statement Regarding Forward-Looking Statements' in Sanofi's annual report on Form 20-F for the year ended December 31, 2024. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. All trademarks mentioned in this press release are the property of the Sanofi group apart from INNOVANCE. Attachment Press Release
Yahoo
28-03-2025
- Health
- Yahoo
US FDA approves Sanofi's bleeding disorder therapy
(Reuters) -The U.S. Food and Drug Administration approved French drugmaker Sanofi's hemophilia therapy, paving the way for a new type of treatment for patients with the rare blood clotting disorder, the regulator said on Friday. Sanofi's subcutaneous, or under-the-skin, therapy helps prevent bleeding and lowers antithrombin, a protein that delays blood clotting, for hemophilia patients aged 12 years and older. It is a first-in-class, antithrombin-lowering therapy for people with hemophilia A or B, with or without inhibitors. The hemophilia market has been dominated by factor replacement therapies for decades. However, recently, gene therapies such as CSL's Hemgenix and BioMarin Pharmaceutical's Roctavian have been approved as an alternative in the United States. Sanofi's therapy Qfitlia is to be used once every two months, compared to other available treatments such as Pfizer's once-a-week injection Hympavzi and Novo Nordisk's once-daily Alhemo. "Today's approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options," said Tanya Wroblewski, deputy director of the Division of Non-Malignant Hematology in the FDA's Center for Drug Evaluation and Research.
Bloomberg
28-03-2025
- Health
- Bloomberg
Sanofi's Easier to Use Hemophilia Drug Approved by US FDA
Sanofi 's treatment for hemophilia that can be administered as infrequently as once every other month, was approved by the US Food and Drug Administration, offering patients greater choice as the market for therapies grows. The drug fitusiran, which will go by the brand name Qfitlia, is the first new treatment for both types of hemophilia — A and B. It can also be given to both patients who have antibodies that prevent replacement therapies from working, as well as those who don't have those antibodies.
