Rilzabrutinib

Dozens of new drugs made the grade in 2025, and a clutch of newly commercial biopharma companies are now underway with their first launches. Photo by Sarah Silbiger/Getty Images How did staff layoffs, inspection backlogs and unprecedented leadership turnover affect the ability of the FDA to approve new drugs in 2025?While it would be presumptuous to base conclusions solely on the number of drugs the FDA approved in 2025, it is noteworthy that FDA nods of new treatments fell short of previous years, though a surge of green lights in December helped make the final numbers more respectable.There were 46 novel drug approvals in 2025, compared to 55 in 2023 and 50 in 2024. Meanwhile, the FDA’s Center for Biologics Evaluation and Research endorsed 18 new biological treatments in 2025, compared to 25 in 2023 and 18 in 2024.The surge in December included seven novel approvals, which was the most in any month of 2025. There also were many more novel approvals (30) in the second half of 2025 than in the first half (16), indicating that the U.S. regulator functioned more efficiently as it gained stability through the year.In March 2025, the Department of Health and Human Services announced a plan to cut 10,000 jobs, including 3,500 at the FDA. In the following months, scores of companies revealed delays for their product approvals that were initiated by the agency.A study from RBC Capital Markets revealed there were more marketing applications rejected in the third quarter of last year than in the previous six periods. Additionally, the FDA delayed 11% of the reviews of those applications in the third quarter compared to an average delay rate of 4% from the previous six quarters.Decline in potential blockbusters In addition to the decline in approvals in 2025, there was a drop in the number of new meds that are expected to generate blockbuster sales.In 2024, there were nearly 20 new products approved that were expected to deliver sales of at least $1 billion by 2030, according to Evaluate Pharma. On this year’s list, only eight newly approved medicines carry those expectations.In the last two weeks of December, Cytokinetics secured a nod for cardiomyopathy treatment Myqorzo and GSK scored with asthma medicine Exdensur. Myqorzo has been projected by Evaluate Pharma to generate sales of $2.8 billion by 2030, with Exdensur’s estimate at $1.2 billion.Of the new drugs that were approved in 2025, the one expected to generate the highest sales by 2030 is AstraZeneca and Daiichi Sankyo’s Datroway, which is the first TROP2-directed antibody-drug conjugate to treat breast cancer. Five months after it was approved, the companies earned an FDA clearance to treat patients with non-small cell lung cancer (NSCLC) who have EGFR mutations.The new drug projected to generate the second-most revenue in 2030 is Insmed’s Brinsupri, which is the first DPP1 inhibitor to reach the market. It also is the first treatment for non-cystic fibrosis bronchiectasis and is projected to reach sales of $3.1 billion in 2030.Other products approved in 2025 that are expected to be blockbusters by 2030 are Vertex’s non-opioid pain reliever Journavx, Novartis’ Rhapsido for chronic spontaneous urticaria, Johnson & Johnson’s Imaavy for generalized myasthenia gravis and GSK’s Penmenvy, a meningococcal vaccine.There were many more high-profile drugs approved in 2024, including Madrigal Pharmaceuticals' Rezdiffra for metabolic dysfunction-associated steatohepatitis; Merck’s Winrevair for pulmonary arterial hypertension; Bristol Myers Squibb’s Cobenfy for schizophrenia; J&J’s Lazcluze for NSCLC; BridgeBio’s Attruby for cardiomyopathy; Vertex’s Alyftrek for cystic fibrosis; and a pair from Eli Lilly—Alzheimer’s disease treatment Kisunla and atopic dermatitis therapy Ebglyss.   Treatment types in 2025 The FDA signed off on 14 oncology treatments last year compared to 15 in 2024. Of the 14, five are for NSCLC, including two for non-squamous NSCLC. The splurge of NSCLC nods came after the FDA blessed three new treatments in the indication in 2024. In addition to AstraZeneca and Daiichi Sankyo’s high-profile approval for breast cancer drug Datroway, Eli Lilly also scored a nod for its breast cancer treatment Inluriyo.The FDA gave its thumbs-up to two blood cancer treatments: Kura Oncology and Kyowa Kirin’s Komzifti for acute myeloid leukemia and Regeneron’s Lynozyfic for multiple myeloma. It’s a continued slowdown from 2023, when the FDA signed off on seven new blood cancer therapies.Of the FDA’s 46 novel drug approvals in 2025, 26 (57%) were designated as rare disease (orphan) treatments. Drugs that are for diseases with fewer than 200,000 patients in the U.S. are slotted into the orphan category. It was a higher rate of rare disease treatments than in the previous two years, when just over half of the new approved drugs were for orphan diseases.Three of the orphan approvals were for the same disorder, hereditary angioedema (HAE), and came in a span of 10 weeks. The splurge began with a nod for CSL’s Andembry. The FDA also endorsed Ionis Pharmaceuticals’ Dawnzera, which is the first RNA-targeted treatment for HAE. The other approval came for KalVista Pharmaceuticals' plasma kallikrein inhibitor Ekterly, which differs from the other injected treatments in that it is taken orally and is used on demand to quell acute attacks of HAE.After a record number of new cell and gene therapy (CGT) approvals in 2024 with nine, there was a slowdown in CGT nods in 2025 with five. It was the fewest since 2022, when the FDA also issued five CGT approvals. With the exception of Novartis’ nod for spinal muscular atrophy treatment Itvisma, the approvals were scored by small companies Abeona, Precigen, Neurotech and Fondazione Telethon, an Italian firm that became the first nonprofit to get a CGT across the FDA finish line. European companies excel in 2025 European companies won many more approvals for new products than their counterparts in the U.S. In fact, no U.S. company earned more than one FDA nod in 2025. Meanwhile, five European companies scored multiple FDA approvals last year.GSK and Novartis achieved three approvals each. GSK earned its nods for meningococcal vaccine Penmenvy, urinary tract and gonorrhea antibiotic Blujepa and severe asthma treatment Exdensur. Novartis won approvals for nephropathy drug Vanrafia, urticaria medicine Rhapsido and spinal muscular atrophy gene therapy Itvisma.The companies with two FDA endorsements were Sanofi, which secured endorsements for thrombocytopenia therapy Wayrilz and hemophilia drug Qfitlia; Boehringer Ingelheim, which won over the regulator with idiopathic pulmonary fibrosis drug Jascayd and lung cancer treatment Hernexeos; and Bayer, which scored with menopause treatment Lynkuet and lung cancer therapy Hyrnuo.It was a lean year for U.S. Big Pharma as AbbVie, Eli Lilly, Johnson & Johnson, Merck and Regeneron were the only top 20 companies that gained approvals for new drugs.Note: The numbers for our annual roundup typically vary slightly from the FDA's official list, because our report includes approvals for biologic therapeutics and vaccines but excludes diagnostic imaging agents, which are represented on the FDA's list.  

Sanofi’s Wayrilz approved in the EU as the first BTK inhibitor to treat immune thrombocytopenia Novel treatment targets BTK through multi-immune modulation to help address the underlying causes of ITP Approval based on the LUNA 3 phase 3 study that demonstrated rapid and durable platelet response and improvements in other ITP symptoms ITP is a rare disease of complex immune dysregulation leading to lower platelet counts, bleeding, and reduced quality of life December 23, 2025 – The European Commission has approved Wayrilz (rilzabrutinib), a novel, oral, reversible, Bruton’s tyrosine kinase (BTK) inhibitor, as a new treatment for immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. This follows the positive opinion by the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP). Wayrilz can help address the underlying causes of ITP through multi-immune modulation, targeting different pathways across the immune system. “ITP is caused by complex immune system dysregulation leading to low platelet counts, bleeding and other often overlooked symptoms that can affect both physical and mental health, significantly impacting quality of life,” said Waleed Ghanima, MD, Head of Research and a Consultant Hematologist at Østfold Hospital, Norway. “The traditional approach to disease management focuses on restoring platelet counts and reducing bleeding risk, but patients may still experience other symptoms. Wayrilz offers a new approach, targeting the underlying cause of ITP through multi-immune modulation to help address the multi-faceted burden of this disease.” “The approval of Wayrilz in the EU for the treatment of ITP underscores Sanofi’s commitment to leveraging our knowledge of the immune system to develop innovative treatments that make a meaningful impact on people living with rare and inflammatory diseases,” said Brian Foard, Executive Vice President, Head of Specialty Care at Sanofi. “Wayrilz has a differentiated mechanism of action, enabling multi-immune modulation to address the underlying pathology of ITP, allowing patients to benefit from an advanced treatment to help manage their disease.” The EU approval of Wayrilz is based on the pivotal LUNA 3 phase 3 study (clinical study identifier: NCT04562766), in which Wayrilz met the primary and secondary endpoints, demonstrating a positive impact on sustained platelet counts as well as other ITP symptoms. The LUNA 3 phase 3 study, presented at the 66th American Society of Hematology Annual Meeting and Exposition, and also published in Blood, evaluated the efficacy and safety of Wayrilz compared to placebo in adults (n=202) with persistent or chronic ITP. Patients who achieved platelet count response at 12 weeks were eligible to continue the full 24-week double-blind period (64% of patients in the Wayrilz arm and 32% of patients in the placebo arm). Patients receiving Wayrilz experienced the following compared to patients receiving placebo: Statistically significant durable platelet response at week 25 (23% of patients in Wayrilz arm vs. 0% in placebo arm; p<0.0001) Faster time to first platelet response (36 days in Wayrilz arm vs. not reached in placebo arm; p<0.0001) Longer duration of platelet response (least square mean of 7 weeks in Wayrilz arm vs. 0.7 weeks in placebo arm) Patients receiving Wayrilz reported an overall 10.6-point improvement in the overall quality of life domain compared to a 2.3-point increase in the placebo arm, based on The Immune Thrombocytopenia Patient Assessment Questionnaire, a clinical tool designed to measure ITP symptoms and impacts. The results of this analysis are descriptive and were not powered for statistical significance. The most common adverse reactions (incidence ≥10%) are diarrhea, nausea, headache, abdominal pain, and COVID-19. Wayrilz has already been approved in the US and the United Arab Emirates (UAE), and it is currently under regulatory review for ITP in Japan and China. It received fast track and orphan drug designations (ODD) in the US for ITP, with similar orphan designations in the EU and Japan. In other indications under investigation, the US Food and Drug Administration (FDA) granted Wayrilz ODD for three additional rare diseases, including warm autoimmune hemolytic anemia (wAIHA), IgG4-related disease (IgG4-RD), and sickle cell disease (SCD). Wayrilz also received FDA fast track designation and EU orphan designation in IgG4-RD. LUNA 3 (clinical study identifier: NCT04562766) was a randomized, multicenter, phase 3 study evaluating the efficacy and safety of Wayrilz vs. placebo in adult and adolescent patients with persistent or chronic ITP. Patients received either oral Wayrilz 400 mg twice a day or placebo through a 12- to 24-week double-blind treatment period, followed by a 28-week open-label treatment period, and then a four-week safety follow-up or long-term extension period. The adolescent part of the study is ongoing. The primary endpoint for the EU is the proportion of adult participants able to achieve platelet counts at or above 50,000/μL for at least eight out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy. Secondary endpoints included time to platelet response (platelet count ≥50 x 109/L or between 30 x 109/L and 9/L and at least doubled from baseline in absence of rescue therapy), number of weeks maintaining a specific platelet response (i.e., doubled or within range), rescue therapy use, physical fatigue score, and bleeding score as assessed by change from baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) assessment at Week 25. Wayrilz (rilzabrutinib) is the first BTK inhibitor for ITP that helps address the root cause of disease through 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 Sanofi’s TAILORED COVALENCY® technology, Wayrilz can selectively inhibit the BTK target. This innovative therapy is now approved for ITP in the US, in the EU, and in the UAE. Regulatory review for use in ITP is currently ongoing in China and Japan. Wayrilz is being studied across a variety of rare diseases, including wAIHA, IgG4-RD, and SCD. These additional indications are currently under investigation and have not been approved by regulatory authorities. ITP is a disease of complex immune dysregulation that causes low platelet counts (<100,000/μL), resulting in a variety of bleeding symptoms and high risk of thromboembolism risk. Beyond bruising and bleeding, which can include potentially life-threatening episodes like intracranial hemorrhage, people living with ITP may experience reduced quality of life, including physical fatigue and cognitive impairment. 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. The content above comes from the network. if any infringement, please contact us to modify.