US FDA approves Sanofi's bleeding disorder therapy
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.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Buzz Feed
4 hours ago
- Buzz Feed
According To Reviewers, These 28 Skincare Products "Work Like Magic"...So, Yeah, You'll Want To Try 'Em
A convenient set of dual-textured toner pads made with lactic and salicylic acid to gently exfoliate skin and refine the appearance of those T-zone pores you swear can be seen from a mile away. A fragrance-free CeraVe eye cream because you've seen this holy grail product evveerrryyyyyyywhere and wanna know what all the hullabaloo is about. Here's the deal — the non-greasy formula was developed with dermatologists and helps combat puffiness and dark circles with a nourishing combination of ceramides and hyaluronic acid. A bottle of professional-grade callus-removing gel so you can quickly get your feet ready to be seen by removing years' (I repeat, YEARS') worth of hard calluses without endless scraping and scrubbing. Time to schedule a date with your strappiest sandals! A reviewer-loved Juno & Co.'s Clean 10 Cleansing Balm if disposable wipes just can't seem to cut through your most stubborn mascara, foundation, and SPF. All you need is a pea-sized amount of this creamy miracle worker to leave your skin feeling clean and hydrated without any icky residues. A TikTok-beloved Bio-Collagen Real Deep mask that works the night shift so you can wake up to skin that's next-level bouncy and glowy. The mask turns transparent as its absorbed into the skin, and loads of reviewers mention visible results after just one treatment. A Hero Cosmetics post-blemish rescue balm for after the big pop to soothe irritation before a long-lasting scar sets up shop on your face. Simply pat your pimple with this gentle, nonirritating formula to relieve dryness, calm angry redness, and help reduce hyperpigmentation. An Acure brightening facial scrub to de-gunk pores with a combination of French green clay, sea kelp, and Madonna lily that delivers soft, glowy results while still being gentle enough to use several times a week. A soothing piercing solution because you've been watching that bump growing on your new piercing for weeks, and it's time to take action. This aftercare formula is designed to reduce the size of your keloid over time with 2–3 daily applications. Plus, a two-ingredient Briotech spray reviewers have used to soothe inflammation of all kinds, from angry piercings and acne to stubborn rashes and rosacea. It uses hypochlorous acid, an ingredient found naturally in our white blood cells, similar to the much pricier Tower 28 spray. An exfoliating mitt if you love the delightful feeling of disgust that comes with seeing dead skin slough away right before your eyes and knowing that your skin is going to feel SO SMOOTH after. A gentle pore-clearing cleansing oil that's so effective at removing makeup and other residue that reviewers say you can actually see bits of gunk leaving your pores. WILD. It's suitable for all skin types and is formulated with heartleaf extract to help you get that *glowy* glass skin look. Or a mega-popular CeraVe hydrating facial cleanser so you can keep your skin clean and clear with one simple product that won't leave your face feeling stripped and dry afterward. It also doubles as a bodywash if you're struggling with bacne, chestne, or buttne. A powerful 2% BHA salicylic acid exfoliant if harsh scrubs haven't done your skin any favors. This helps to clear skin and unclog pores by gently sloughing away dead skin to reveal the texture-free glow hiding underneath. A blemish-busting Mario Badescu drying lotion to take on pimples while you sleep with an effective blend of salicylic acid, sulfur, calamine, and zinc oxide. It'll feel like Christmas morning when you jump out of bed to check out your gifts. In this case, the gift of smaller pimples and a more clear complexion. Plus, a pack of nose pore patches for making your skincare dreams a reality by expunging gunk and excess oil from troublesome hot spots like your nose or chin. The hardest part will be resisting the urge to touch your post-treatment skin now that it's as smooth as a baby seal. A gentle LilyAna Naturals retinol cream because retinol is a skincare workhorse that can do wonders for the appearance of fine lines and uneven texture. Combine that with a slew of hydrating ingredients like hyaluronic acid, shea butter, organic green tea, and jojoba oil, and you've got a daily moisturizer that feels closer to a magic potion. A bottle of fragrance-free Goami rice toner so you can add an extra dose of hydration to your skincare regimen when you need it without shaking up your entire routine. A Shea Butter Vanilla Cashmere body lotion if you're still on the hunt for a moisturizer that's hydrating, lightweight, and soothing. Allow me to introduce you to this delicious-smelling formula at a price that's just * chef's kiss.* Or a retinol body lotion jam-packed with dermatologist-recommended ingredients like retinol and ferulic acid that promote skin cell turnover to target common complaints like wrinkles and rough, crepey texture. And that's on top of just being a nourishing, non-greasy moisturizer you can use allllllll over. A hydrating snail mucin repairing essence for soothing *all* skin types (reviewers love how gentle it is) with the potential to help heal acne scars, fade dark spots, and smooth fine for under $20!! An oil-free La Roche-Posay daily face moisturizer to seal in hydration with a fragrance-free formula that does more than just moisturize with ceramides and niacinamide to help repair the skin barrier. And you can get it with or without SPF 30?!? Pinch me. A wildly popular Bio-Oil because real-life magic (aka science) has gifted us this powerful blend of moisturizing oils and vitamins to help fade the scars and stretch marks you thought were going to stick around for the foreseeable ever. An anti-bacterial butt acne-clearing lotion made with tea tree oil that'll finally give your booty the same blemish-busting TLC you treat your face to. A cruelty-free intense-therapy lip balm to take even chronically irritated lips from dry and cracked to hydrated and smooth while protecting them from future damage. Plus, a vegan and cruelty-free lip scrub because your favorite lipstick will go on much smoother with a bit of extra skin prep. Scrub away any dry, flaky layers with this nourishing combination of coconut, sugar, avocado oil, and jojoba oil. A vegan exfoliating body scrub for anyone who struggles with persistent KP. This buffs away bumps by combining the benefits of a chemical peel and microdermabrasion into one gentle, at-home treatment. A pack of two dark spot corrector soap bars because they're packed with all the skin-loving good stuff (vitamin C, turmeric, hyaluronic acid, collagen, and MORE) to help even out skin and diminish dark spots wherever they pop up. And a multi-tasking, plant-powered vitamin C serum with over 97,000 5-star ratings that can help minimize the appearance of a long list of skin woes like dark circles, sun spots, redness, and even breakouts.
Yahoo
9 hours ago
- Yahoo
Press Release: Sanofi's rilzabrutinib earns orphan designation in the EU for IgG4-related disease
Sanofi's rilzabrutinib earns orphan designation in the EU for IgG4-related disease Data from the positive Phase 2 study evaluating rilzabrutinib for the treatment of IgG4-related disease presented at EULAR 2025 Additional orphan designation underscores Sanofi commitment to advancing new medicines in immune-mediated rare diseases Paris, August 14, 2025. The European Medicines Agency has granted orphan designation to rilzabrutinib, a reversible covalent Bruton's tyrosine kinase (BTK) inhibitor, for IgG4-related disease (IgG4-RD). EMA grants orphan designation to investigational therapies addressing rare, life-threatening or debilitating medical diseases or conditions that affect no more than 5 in 10,000 persons in the EU. Rilzabrutinib for the treatment of IgG4-related disease was evaluated in a phase 2 study (clinical study identifier: NCT04520451) and results were presented at the European Alliance of Associations for Rheumatology (EULAR) 2025 Congress. In IgG4-RD patients, treatment with rilzabrutinib for 52 weeks led to reduction in disease flare, other disease markers, and glucocorticoid sparing. The safety profile of rilzabrutinib in the study was consistent with previous studies, with no new safety signals observed. In addition to IgG4-related disease, rilzabrutinib has received orphan designations for immune thrombocytopenia (ITP) in the US, the EU, and Japan; and for warm autoimmune hemolytic anemia, IgG4-RD and sickle cell disease in the US. Rilzabrutinib has also been granted fast track designation in the US in ITP and IgG4-RD. Rilzabrutinib is currently under regulatory review in the US, the EU, and China for its potential use in ITP. The target action date for the US FDA regulatory decision for ITP, which was granted fast track designation, is August 29, 2025. Rilzabrutinib is an investigational agent, and its safety and efficacy have not been evaluated by any regulatory authority. About rilzabrutinib Rilzabrutinib is a novel, advanced, oral, reversible covalent BTK inhibitor that has the potential to be an effective new medicine for several rare immune-mediated or inflammatory diseases by working to restore immune balance via multi-immune modulation. BTK, expressed in B cells, macrophages, and other innate immune cells, plays a critical role in multiple immune-mediated disease processes and inflammatory pathways. With the application of the TAILORED COVALENCY® technology, rilzabrutinib can selectively inhibit the BTK target while potentially reducing the risk of off-target side IgG4-RD IgG4-RD is a progressive, relapsing, chronic immune-mediated rare disease, which can manifest in almost every organ and can lead to organ damage and irreversible dysfunction with a sometimes-fatal outcome. People with IgG4-RD experience frequent flare-ups of the condition characterized by periods of exacerbated symptoms. It affects approximately eight out of 100,000 adult patients in the US each year. Due to its rarity and challenges with diagnosis, the global prevalence of IgG4-RD is unknown. About Sanofi Sanofi is an R&D driven, AI-powered biopharma company committed to improving people's lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people's lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Media RelationsSandrine Guendoul | +33 6 25 09 14 25 | Berland | +1 215 432 0234 | Léo Le Bourhis | +33 6 75 06 43 81 | Victor Rouault | +33 6 70 93 71 40 | Timothy Gilbert | +1 516 521 2929 | Ubaldi | +33 6 30 19 66 46 | Investor RelationsThomas Kudsk Larsen |+44 7545 513 693 | Alizé Kaisserian | +33 6 47 04 12 11 | Lauscher | +1 908 612 7239 | Browne | +1 781 249 1766 | Nathalie Pham | +33 7 85 93 30 17 | Elgoutni | +1 617 710 3587 | Thibaud Châtelet | +33 6 80 80 89 90 | Li | +33 6 84 00 90 72 | Sanofi forward-looking statements This 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 and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. 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, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi's ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, 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. Attachment Press ReleaseError while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
Business Wire
17 hours ago
- Business Wire
Amber Specialty Pharmacy Begins Dispensing Newly Approved BRINSUPRI™ (brensocatib) for Non-Cystic Fibrosisbronchiectasis (NCFB)
OMAHA, Neb.--(BUSINESS WIRE)--Amber Specialty Pharmacy, a pioneer in specialty pharmacy for more than 25 years, today announced it is now dispensing BRINSUPRI™ (brensocatib) following the medication's U.S. Food and Drug Administration (FDA) approval as the first treatment specifically indicated to reduce pulmonary exacerbations in adults and adolescents with non-cystic fibrosis bronchiectasis, a chronic neutrophil-mediated airway disease. 'Our nationally recognized clinical program is ready to ensure rapid access, personalized counseling, and comprehensive reimbursement support for every patient who needs it,' said Kristin Williams, president of Amber Specialty Pharmacy. Amber Specialty Pharmacy streamlines the treatment process for both patients and referring providers by offering the following support services: Nationwide dispensing through its URAC and ACHC-accredited network 24/7 clinical support and adherence counseling from specialty-licensed pharmacists End-to-end benefits investigation, prior authorization and financial assistance coordination Healthcare providers can initiate BRINSUPRI referrals by calling (888) 370-1724 or visiting to download electronic referral forms. About Amber Specialty Pharmacy Amber Specialty Pharmacy is a pioneer in the specialty pharmacy industry, with more than 25 years of experience in providing specialized care for patients with chronic and complex medical conditions. With a focus on personalized support and quality clinical care, Amber Specialty Pharmacy serves patients across the United States. The pharmacy is accredited by both the Utilization Review Accreditation Commission (URAC) and the Accreditation Commission for Health Care (ACHC). Amber Specialty Pharmacy is headquartered in Omaha, Nebraska. To learn more, visit
