Purpura, Thrombotic Thrombocytopenic

Developed by Sanofi, CABLIVI is the first FDA-approved nanobody therapy for this condition. Increasing awareness, improved diagnostic rates, and rising demand for targeted therapies in hematology drive the market growth. With its niche indication and high treatment costs, CABLIVI holds a strong position in the rare disease therapeutics segment. LAS VEGAS, March 19, 2025 /PRNewswire/ -- DelveInsight's " CABLIVI Market Size, Forecast, and Market Insight Report" highlights the details around CABLIVI, a von Willebrand factor (vWF)-directed antibody fragment. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of CABLIVI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Ablynx NV's CABLIVI (caplacizumab-yhdp) Overview CABLIVI is an antibody fragment that targets von Willebrand factor (vWF) and is used to treat adult patients with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. It works by binding to the A1-domain of vWF, preventing its interaction with platelets, which helps reduce vWF-mediated platelet adhesion and consumption. CABLIVI is approved in both the US and EU for treating aTTP. In 2024, CABLIVI sales reached €249 million, reflecting a 9.7% increase, driven by a rise in the number of patients identified for suitable treatment in the US, as well as new launches in Europe and other regions. The recommended CABLIVI dosing schedule is as follows: Day 1: Administer an 11 mg intravenous bolus injection at least 15 minutes before plasma exchange, followed by an 11 mg subcutaneous injection after the exchange on the same day. During Plasma Exchange Therapy: Administer 11 mg subcutaneously once daily after each plasma exchange session. After Plasma Exchange Therapy: Continue with 11 mg subcutaneous injections once daily for 30 days after the final plasma exchange. If signs of persistent underlying disease, such as low ADAMTS13 activity, are still present, treatment can be extended for up to 28 additional days. CABLIVI treatment should be discontinued if the patient experiences more than two recurrences of aTTP while on the medication. Learn more about CABLIVI projected market size for thrombocytopenia @ CABLIVI Market Potential Thrombocytopenia is a medical condition where the platelet count in the blood drops below the normal level (<150,000/µL). Common causes include heparin-induced thrombocytopenia, chemotherapy-induced thrombocytopenia, thrombocytopenia linked to chronic liver disease, immune thrombocytopenia (ITP), and thrombotic thrombocytopenic purpura (TTP). Among the 7MM, the United States reported the highest number of thrombocytopenia cases, accounting for about 40% of the total cases in 2023, according to DelveInsight. Treatment for thrombocytopenia varies based on its cause and severity, with the primary goal of preventing bleeding-related complications and improving patient outcomes. Approved therapies for thrombocytopenia in the US include MULPLETA, DOPTELET, TAVALISSE, PROMACTA, NPLATE, CABLIVI, GAMMAPLEX, OCTAGAM, RHOPHYLAC, and PRIVIGEN. Additionally, danaparoid, argatroban, and lepirudin are approved for treating thrombosis in patients with heparin-induced thrombocytopenia. For severe thrombocytopenia related to chronic liver disease, treatment options include platelet transfusion, splenic artery embolization, splenectomy, and transjugular intrahepatic portosystemic stent shunt (TIPSS) placement. The US holds approximately 60% of the thrombocytopenia market share, surpassing the combined market size of the EU4, the UK, and Japan. Market growth is expected to be driven by an increasing patient population, the introduction of new therapies, and deeper market penetration within the 7MM. Discover more about the thrombocytopenia market in detail @ Thrombocytopenia Market Report Emerging Competitors of CABLIVI The potential therapies that can mark a significant change in the thrombocytopenia treatment landscape and give tough competition to CABLIVI include Rilzabrutinib (Sanofi), Ianalumab (Novartis), Mezagitamab (Takeda), PF-06835375 (Pfizer), Povetacicept (Vertex), Cevidoplenib (Genosco/Oscotec), and Efgartigimod (ARGX-113) (Argenx), among others. In December 2024, Sanofi shared encouraging Phase III results at ASH for rilzabrutinib, its investigational oral BTK inhibitor, in patients with ITP. In June 2024, Takeda reported late-breaking Phase IIb data on Mezagitamab, highlighting its potential to revolutionize the treatment of Primary ITP. In Novartis' Q2 2024 update, the company forecasted key trial results: VAYHIT1 (NCT05653349) for first-line therapy, expected in 2026, and VAYHIT2 (NCT05653219) for second-line therapy, anticipated in 2025. Novartis also aims to file for ianalumabin approval in both first- and second-line ITP treatment by 2027 or later. To know more about the number of competing drugs in development, visit @ CABLIVI Market Positioning Compared to Other Drugs Key Milestones of CABLIVI In June 2022, at the 27th Annual European HematologyAssociation (EHA) Congress, Sanofi presented long-term safety and efficacy results from the post-HERCULES trial (NCT02878603), a three-year prospective follow-up study of patients with acquired thrombotic thrombocytopenic purpura (aTTP) who completed the Phase 3 HERCULES trial. In February 2019, the FDA approved CABLIVI (caplacizumab-yhdp) injection as the first therapy specifically indicated for treating adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening blood clotting disorder. It is to be used in combination with plasma exchange and immunosuppressive therapy. In September 2018, the European Commission approved CABLIVI (caplacizumab) for marketing, authorizing its use in treating adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. CABLIVI is the first therapy specifically designed for aTTP treatment. In July 2017, Ablynx announced a research collaboration and global exclusive licensing agreement with Sanofi, aiming to develop and commercialize Nanobody-based therapies for treating various immune-mediated inflammatory diseases. Discover how CABLIVI is shaping the thrombocytopenia treatment landscape @ CABLIVI FDA Approval CABLIVI Market Dynamics The primary driver of CABLIVI's market growth is the increasing recognition and diagnosis of aTTP, coupled with improved access to specialized treatments. Growing awareness among healthcare providers and advancements in diagnostic capabilities have facilitated early and accurate diagnosis, thereby boosting the demand for targeted therapies like CABLIVI. The drug's ability to prevent microthrombi formation, shorten the duration of plasma exchange, and reduce the risk of relapses has positioned it as a preferred option in the treatment landscape for aTTP. Moreover, Sanofi's robust commercial infrastructure and strategic marketing efforts have further contributed to market penetration and adoption. Despite its therapeutic benefits, CABLIVI faces challenges related to its high cost and reimbursement complexities. The specialized nature of aTTP treatment, combined with the requirement for hospital-based administration and close monitoring, adds to the overall cost burden on healthcare systems and patients. Additionally, competition from alternative therapies, including plasma exchange and immunosuppressive treatments, may limit its market expansion. Regulatory hurdles and the need for long-term safety data could also impact the drug's market trajectory. The CABLIVI market is expected to grow steadily, driven by ongoing clinical research and potential label expansions into related thrombotic disorders. Increased investments in rare disease treatments and orphan drug incentives are likely to support market growth. Moreover, the rise in strategic collaborations and partnerships to improve patient access and streamline reimbursement processes could enhance market uptake. However, balancing pricing pressures with broader patient access will remain a key factor in sustaining long-term market performance. Dive deeper to get more insight into CABLIVI's strengths & weaknesses relative to competitors @ CABLIVI Market Drug Report Table of Contents Related Reports Acquired Thrombotic Thrombocytopenic Purpura Pipeline Acquired Thrombotic Thrombocytopenic Purpura Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acquired thrombotic thrombocytopenic purpura companies, including Sanofi, TargED Biopharmaceuticals, Takeda, Genentech, Alexion Pharmaceuticals, among others. Thrombocytopenia Market Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key thrombocytopenia companies including Sanofi, Principia Biopharma, Baxalta, Takeda, Argenx, Millennium Pharmaceuticals, Biotest, GC Pharma, Genosco (Subsidiary of Oscotec), Rigel Pharmaceuticals, Kissei Pharmaceutical, Shionogi & Co., Ltd, Amgen, Novartis, Zenyaku Kogyo, among others. Thrombocytopenia Pipeline Thrombocytopenia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key thrombocytopenia companies, including Takeda, Argenx, Keros Therapeutics, Principia Biopharma, Momenta Pharmaceuticals, Veralox Therapeutics, Novartis Pharmaceuticals, Pfizer, HUTCHMED, Principia Biopharma, Genosco, UCB, among others. Immune Thrombocytopenia Market Immune Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key immune thrombocytopenia companies including Rigel Pharmaceuticals, Kissei Pharmaceutical, Sobi (Dova Pharmaceuticals), Amgen, Argenx, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Previously, Takeda worked across 10 to 12 modalities. Now, the company has zeroed in on four modalities: small molecules, biologics, antibody-drug conjugates and allogeneic cell therapies. \n Amid a rapidly evolving macroenvironment, Takeda is changing up its R&D priorities and investment strategy.“There\'s an evolution of our economic model that starts with [the] IRA (Inflation Reduction Act), and will get worse and worse and worse, but I think there\'s so many opportunities for us to counter that with new ways of working,” Takeda\'s R&D head Andy Plump, M.D., Ph.D., told Fierce Biotech at the annual J.P. Morgan Healthcare Conference in San Francisco last week. The conference ended right as the U.S. government selected its second round of drugs up for Medicare price negotiations under the Inflation Reduction Act.After a wide-ranging restructure last year, Takeda now touts its “most robust late-stage pipeline” in the pharma’s history, consisting of six late-stage programs across 14 studies, according to Plump. Three of the candidates—a narcolepsy type 1 candidate, an oral TYK2 inhibitor for psoriasis and an injection for chronic blood cancer polycythemia vera—have phase 3 readouts slated for later this year.Developing a more mature pipeline meant that Takeda had to boost its standards, Plump said. Raising the bar resulted in several programs being left behind, including a midstage CD19 CAR-NK cell therapy, a small molecule known as subasumstat and a peptide agonist being studied as a nausea and vomiting candidate, all of which were discarded last May.Previously, Takeda worked across 10 to 12 modalities, according to Plump. Now, the company has zeroed in on four modalities: small molecules, biologics, antibody-drug conjugates and allogeneic cell therapies. Takeda chose the quartet based on past product wins and future need for patients, plus going into areas that the drugmaker can build out its internal capabilities for, Plump said.“That mindset shift is a really big one for our employees because you had people that were just used to getting essentially whatever they wanted or they needed to push every program in as many potential indications as fast as possible,” Plump said. “Now, we\'ll be more thoughtful and selective in where we do that.”  The new perspective also impacts how the pharma views partnerships. Previously, Takeda inked pacts to expand its pipeline and experiment with different modalities. Now, the company is tightening the reins and will be “more thoughtful” in terms of what it brings in, according to Plump.The drugmaker had previously invested “very heavily” in early-stage, higher-risk programs—of which many would fail, Plump explained. “That\'s not something that we can continue to do—and it\'s not something that we have to do anymore—because we have this late-stage pipeline,” he said.The pharma has a new investment thesis for earlier programs, Plump said, explaining that early programs will be stopped if they don’t rapidly reach an inflection point.The pharma is also putting a heavy focus on option-based deals, Plump said, echoing Takeda CEO Christophe Weber’s sentiments that were shared during the organization’s presentation at JPM.“The benefit of an option deal is that we don\'t have to spend our new dollars because our partners will run the programs,” Plump explained, underscoring the economic efficiency of the deal type. “Then you have the option, if things work, to buy the program and bring it in at a cost that\'s a fraction of what it normally would be.”Plump pointed to the deal Takeda penned with Keros Therapeutics in December, putting down $200 million upfront and offering up to $1.1 billion in biobucks for ex-China rights to Keros’ activin inhibitor elritercept. The candidate is being studied as a treatment for anemia tied to blood cancers, including myelodysplastic syndromes and myelofibrosis. The program, which is set to enter phase 3 testing, could challenge Bristol Myers Squibb’s Reblozyl in blood cancer indications if approved. While five of the six company’s late-stage programs are in rare diseases, Plump also insists that Takeda won’t be siloed as a specialty disease pharma.“We\'ve mistakenly been considered just a rare disease company. It\'s not true,” Takeda’s R&D president said. “We are a company that will go after transformative medicines for patients in both rare and more prevalent disease populations.”But Plump understands the misconception. Historically, the pharma’s late-stage pipeline has focused on candidates for ultrarare conditions, such as Adzynma, a recombinant protein product that snagged FDA approval in 2023 for a very rare congenital blood clotting disorder. Known as thrombotic thrombocytopenic purpura, the disease is estimated to affect fewer than 1,000 Americans.However, Plump says these ultrarare programs aren’t indicative of Takeda’s overarching investment strategy, arguing that the diseases the organization goes after have broad potential.      “These are rare diseases with very substantive patient populations, very high unmet medical needs and revenue projections that are in the billions well,” Plump said.  Today, Takeda has three core indications of focus: oncology, neuroscience and what the company calls GI-squared, which encompasses gastrointestinal and inflammation conditions. 

TGD001 is being developed to offer patients with clotting disorders a faster, safer and more effective treatment than today's standard of care – saving lives and reducing disability Following the Phase 1 safety study, TargED intends to further develop TGD001 in patients with AIS and iTTP (a rare micro-thrombotic disorder) in the second half of 2025 UTRECHT, the Netherlands, Dec. 19, 2024 /PRNewswire/ -- TargED Biopharmaceuticals ("TargED"), a private biotechnology company focused on developing improved treatments for thrombotic diseases, today announces the start of clinical development with the dosing of the first participant with TGD001. TGD001 is a groundbreaking thrombolytic designed to break down clots of all sizes and compositions – from large clots which cause AIS, down to 'micro-clots' which cause conditions such as the rare, life-threatening disorder immune-mediated thrombotic thrombocytopenic purpura (iTTP). The first study participant was successfully dosed yesterday [Dec 18] at a Phase 1 clinic in Germany. The study's design is a randomized, double-blind, placebo-controlled single-ascending dose study to evaluate TGD001's safety, tolerability and pharmacokinetics in healthy volunteers (EU CT ID number: 2024-514931-63-00). First safety read-out from the Phase 1 study is expected by mid-2025. TargED plans to continue clinical development with two proof-of-concept trials in the second half of 2025: a Phase 2a trial in AIS patients and a Phase 1b trial in iTTP patients, for which TargED received EU Orphan Drug Designation in July 2024 from the European Commission. Kristof Vercruysse, Chief Executive Officer and co-founder of TargED, said: "The dosing of the first participant with our lead compound TGD001 marks a watershed in the treatment of thrombotic disorders. Rapid treatment with highly effective thrombolytics that are well tolerated and don't exaggerate the risk of bleeding is essential to reduce the risk of serious disability or even death from thrombotic disorders. Today's thrombolytics show efficacy but their significant drawbacks greatly limit their safety and effectiveness, and result in restricted utility. TGD001 is specifically designed to address these shortcomings. Its unique mode of action allows for blood clots of all sizes and compositions to be targeted and rapidly dissolved without increasing bleeding risk, resulting in clinical benefit for a broad population of patients." Steven de Maat, Chief Scientific Officer and co-founder of TargED, said: "Acute ischemic stroke is one of the biggest causes of preventable death and disability in the world today, but up to 80% of AIS patients cannot be treated with currently available thrombolytics such as intravenous alteplase (tPA). Meanwhile, there is significant room for improvement in the treatment of iTTP. Despite recent advances, 15% of iTTP patients who receive treatment do not survive, and half of those who live still suffer long term health impairment. TGD001 has the potential to greatly improve outcomes in both these indications and, we believe, in other thrombotic indications as well." TGD001 is a first-in-class 'fusion protein' drug with a unique two-step mode of action. It targets clots via an antibody fragment that binds to a non-functional domain of von Willebrand Factor (VWF), a protein present in all types of thrombi. Then, in the immediate vicinity of the thrombus, it activates the endogenous enzyme system that degrades both VWF and fibrin, two key clotting ingredients which form the structural bond in the thrombus. This mode of action enables rapid and effective thrombolysis without systemic activation of the lysis cascade and the resulting bleeding risk. TGD001 is administered as short or bolus injection, making it a suitable therapeutic for the emergency, in-patient and first-response setting. About TargED Biopharmaceuticals TargED Biopharmaceuticals B.V. is a Netherlands based biotechnology company developing first-in-class biological drugs to improve treatment of thrombosis. TargED stands for Targeted Enzyme Delivery. TargED's biological drugs are unique by using antibody fragments ("VhH") to deliver enzymes to sites of thrombosis, enabling 'targeted' thrombolysis. Its lead compound TGD001 is currently under development for the treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP) and acute ischemic stroke (AIS). The objective is to accelerate thrombolysis in all forms of thrombosis, irrespective of the thrombus composition. TargED, a spin-off of the University Medical Center Utrecht, was founded in July 2020. Since then, TargED raised more than €45 million of funding, supporting its growth to become a clinical-stage company. For more information please visit Acute Ischemic Stroke (AIS), which occurs when one or more clots block blood flow to part of the brain, accounts for around 90% of all strokes. Worldwide, around 18 million AIS incidents take place every year, resulting in 5 million deaths and 5 million people permanently disabled. A delay in diagnosis and treatment can lead to loss of brain tissue, causing permanent disability or death. Standard of care thrombolysis must be initiated within 4.5 hours of ischemic stroke, since after this time the risk of tPA-induced bleeding outweighs the potential benefit. Due to their limitations, up to 80% of AIS patients are ineligible for current thrombolytics like tPA. Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening blood disorder characterized by sudden episodes of clot formation in small blood vessels throughout the body. Blood flow to organs such as the kidney, heart, and brain is disrupted, causing functional failure. There are about 7,500 incidents of iTTP a year worldwide and it particularly affects young women, sometimes after pregnancy. Without treatment, up to 90% of cases are fatal. Treatment in specialized centers cuts mortality to 15%; however, of the survivors, half still suffer long-term complications. Current treatment is complex, involving blood plasma exchange, immune suppression and administration of the drug caplacizumab, which stops new clot formation. To date, no thrombolytic – i.e. a drug that clears existing thrombi – is licensed in iTTP due to the perceived inherent bleeding risk of patients with thrombotic microangiopathies and the systemic thrombolytic activity of currently licensed thrombolytics. TGD001 has the potential to resolve iTTP attacks very rapidly, saving organ tissue and preventing disability and death. SOURCE TargED WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED