Romiplostim

Sanofi has hailed rilzabrutinib's potential as a “multi-indication blockbuster” that could deliver 2 billion euros to 5 billion euros at its sales peak. While Sanofi originally had its eye on tolebrutinib when it purchased its partner Principia Biopharma for $3.7 billion five years ago, another one of the Bruton tyrosine kinase (BTK) inhibitors inherited from the biotech has made it across the FDA finish line first.The drug rilzabrutinib, which is now approved as Wayrilz in immune thrombocytopenia (ITP), has been hailed (PDF) by Sanofi as a potential “multi-indication blockbuster” that could deliver 2 billion euros to 5 billion euros at its sales peak. The FDA’s approval Friday specifically clears Wayrilz for use in adults with persistent or chronic ITP who haven't responded to prior therapy. The regulatory endorsement marks the first approval for a BTK inhibitor to treat ITP in the U.S., where the condition is estimated to affect around 100,000 people, Mike Quigley, Ph.D., Sanofi’s chief scientific officer and global head of research, said in an interview with Fierce.According to a Friday approval announcement, Sanofi believes there are about 25,000 patients who could specifically benefit from the new medication in the U.S. ITP is an autoimmune disorder in which low levels of platelets—the cells that allow blood to clot—can lead patients to bruise easily, start bleeding without cause and bleed more than those without ITP.To holistically treat ITP, a drug must attack the disease on three fronts, Quigley said, singling out autoantibody production by B cells, FC gamma receptor biology implicated in the body’s immune responses and inflammation. Sanofi believes that BTK proteins form a “key node that sort of permeates all three components of that pathophysiology,” Quigley explained. By targeting BTK with Wayrilz, the French pharma believes it can address ITP across all three of those axes.In contrast, most standard treatments for ITP focus solely on raising patients’ platelet counts, targeting a symptom rather than the underlying cause of the disease, Quigley pointed out.Many patients with the clotting disease receive steroids or thrombopoietin receptor agonists, such as Amgen’s Nplate or Novartis’ Promacta. Roche’s Rituxan is also used off-label to treat the condition in the U.S., though the antibody comes with some trade-offs when deployed against ITP. At the end of the day, ITP is “an unpredictable disease” that is “difficult to treat,” Quigley said.“By our accounts, more than 60% of patients worry about sudden changes in platelet counts,” Quigley said, describing a sense of "helplessness that these patients feel in the context of really unregulatable disease pathophysiology.”In turn, Sanofi is confident that Wayrilz, with its novel mechanism of action, will have a major role to play in subsequent ITP treatment, he said.The FDA green light was informed by Sanofi’s late-stage Luna 3 trial, in which Wayrilz helped raise and sustain platelet levels in 23% of ITP patients at 25 weeks, compared to none in the study’s control arm.The drug also helped patients achieve first platelet responses quicker than placebo, and those responses were longer-lasting, too. The time to first platelet response in the Wayrilz arm of the study landed at 36 days and was "not reached" in the control cohort, according to Sanofi's approval announcement.  Wayrilz also helped patients chart improvements in areas like physical fatigue and bleeding, as measured by the Immune Thrombocytopenic Purpura-Patient Assessment Questionnaire and the ITP Bleeding Scale.The drug’s safety profile was largely on par with the results observed in the trial’s placebo cohort, though patients on Wayrilz were more likely to experience side effects like low-grade diarrhea, nausea, headache and abdominal pain.Sanofi did not disclose pricing plans for the drug in its press release, but it noted that patients will be able to seek financial assistance, navigate insurance coverage and receive other materials for Wayrilz through its HemAssist patient support program. Rilzabrutinib was not the main draw for Sanofi when it moved to acquire the drug’s original developer, Principia, back in 2020. At the time, the company was all in on tolebrutinib—another BTK inhibitor that Sanofi had been developing in partnership with Principia as a potential treatment for multiple sclerosis (MS).But, after reviewing mixed MS data from the asset in 2024, Sanofi in January nixed further development of tolebrutinib in that indication. Sanofi has said it still plans to pursue approval of the drug in another setting.Meanwhile, Sanofi figures rilzabrutinib still has several more arrows in its quiver. Last February, Sanofi elected to move rilzabrutinib into a phase 3 study in chronic hives after the BTK inhibitor posted a win in a midstage chronic spontaneous urticaria study.The French drugmaker is also testing the treatment in asthma, focal segmental glomerulosclerosis, warm autoimmune hemolytic anemia and IgG4-related disease, where rilzabrutinib recently picked up an EU orphan designation.  Outside the U.S., Wayrilz already boasts an ITP approval in the United Arab Emirates, where it was cleared for the treatment of the bleeding disorder in June. Regulators in China and the EU are reviewing the drug for approval in ITP, as well. 

SOUTH SAN FRANCISCO, Calif., Aug. 20, 2025 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced the appointment of James M. Daly to its Board of Directors effective August 19, 2025. Mr. Daly brings over 30 years of global biopharmaceutical leadership experience with particular expertise in commercialization to the Company’s Board of Directors. “Jim has longstanding expertise leading commercial launches of innovative therapies, coupled with extensive Board experience guiding later-stage, global biopharma companies,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “We are pleased to welcome him to our Board and look forward to his contributions and oversight as our company advances toward becoming a fully integrated commercial organization.” Most recently, Mr. Daly served as the Chief Commercial Officer at Incyte Corporation where he led the launch of Jakafi® (ruxolitinib) and oversaw development of the company’s global commercial capabilities. Previously, he spent 10 years at Amgen where he held multiple senior leadership positions, including Senior Vice President of North America Commercial Operations, and launched several successful medicines including Neulasta®, Xgeva®, Nplate® and Vectibix®. He began his career at GlaxoSmithKline, serving in escalating positions of seniority over his 16-year tenure with the company. He earned a B.S. in Pharmacy and an M.B.A. from the University at Buffalo, SUNY. Mr. Daly currently serves on the Boards of Madrigal Pharmaceuticals, argenx SE, and Acadia Pharmaceuticals. About Cytokinetics Cytokinetics is a specialty cardiovascular biopharmaceutical company, building on its over 25 years of pioneering scientific innovations in muscle biology to advance a pipeline of potential new medicines for patients suffering from diseases of cardiac muscle dysfunction. Cytokinetics is readying for potential regulatory approvals and commercialization of aficamten, a cardiac myosin inhibitor following positive results from SEQUOIA-HCM, the pivotal Phase 3 clinical trial in patients with obstructive hypertrophic cardiomyopathy (HCM). Aficamten is also being evaluated in additional clinical trials enrolling patients with obstructive and non-obstructive HCM. Cytokinetics is also developing omecamtiv mecarbil, a cardiac myosin activator, in patients with heart failure with severely reduced ejection fraction (HFrEF), ulacamten, a cardiac myosin inhibitor with a mechanism of action distinct from aficamten, for the potential treatment of heart failure with preserved ejection fraction (HFpEF) and CK-089, a fast skeletal muscle troponin activator with potential therapeutic application to a specific type of muscular dystrophy and other conditions of impaired skeletal muscle function. For additional information about Cytokinetics, visit www.cytokinetics.com and follow us on X, LinkedIn, Facebook and YouTube. Forward-Looking Statements This press release contains forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995 (the “Act”). Cytokinetics disclaims any intent or obligation to update these forward-looking statements and claims the protection of the Act’s Safe Harbor for forward-looking statements. Examples of such statements include, but are not limited to, statements relating to Cytokinetics’ and its partners’ research and development activities of Cytokinetics’ product candidates. Such statements are based on management’s current expectations, but actual results may differ materially due to various risks and uncertainties, including, but not limited to the risks related to Cytokinetics’ business outlines in Cytokinetics’ filings with the Securities and Exchange Commission. Forward-looking statements are not guarantees of future performance, and Cytokinetics’ actual results of operations, financial condition and liquidity, and the development of the industry in which it operates, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that Cytokinetics makes in this press release speak only as of the date of this press release. Cytokinetics assumes no obligation to update its forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release. CYTOKINETICS® and the CYTOKINETICS and C-shaped logo are registered trademarks of Cytokinetics in the U.S. and certain other countries. Contact:Cytokinetics Diane WeiserSenior Vice President, Corporate Affairs(415) 290-7757