PUBLISHER: DelveInsight | PRODUCT CODE: 1605426
PUBLISHER: DelveInsight | PRODUCT CODE: 1605426
DelveInsight's "Immune Thrombocytopenic Purpura (ITP) - Market Insights, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of ITP, historical and forecasted epidemiology as well as ITP market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.
The ITP market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM ITP market size from 2020 to 2034. The report also covers current ITP treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.
ITP Overview
Immune thrombocytopenic purpura (ITP) is an acquired immune-mediated disorder characterized by isolated thrombocytopenia and the absence of other conditions or agents known to induce thrombocytopenia. The incidence is 100 cases per 1 million persons annually, and approximately 50% of cases occur in children. Immune thrombocytopenic purpura in children often resolves spontaneously but is more insidious and chronic in adults. The risk of bleeding correlates to the severity of thrombocytopenia. Patients may present without symptoms, minimal bleeding, or serious haemorrhage (e.g., mucosal, intracranial, gastrointestinal, genitourinary). Older patients, patients on antiplatelet therapy, and patients with comorbid conditions may have severe bleeding manifestations.
ITP can be acute or chronic. Acute ITP is the most common form of the disease and usually affects children between 2 and 6 years old. In these patients, symptoms can develop after a viral infection and disappear after a few weeks to 6 months. Chronic ITP affects persons of any age, most often women, and lasts for at least 6 months. This form of ITP can recur, so continuous medical monitoring is required.
ITP Diagnosis
A careful history, physical examination, CBC, and review of the blood smear make a presumptive diagnosis of ITP. Response to initial treatment with corticosteroids, intravenous immunoglobulin (IVIg), or anti-RhD supports the diagnosis and confirms the immune nature of thrombocytopenia. However, additional investigation is necessary to exclude secondary ITP and to provide additional information to assist with patient management.
Occasionally, a low platelet count may be detected incidentally by blood tests such as a CBC ordered for other purposes, and the individual is without apparent symptoms (asymptomatic). Inspection of the blood smear under the microscope will verify if the platelets are truly reduced in number and not simply clumped (stuck together, so they are too big to be counted by automated machines as platelets) and that the platelets are not uniformly small or exceeding large (giant platelets approximating the size of red blood cells). The red and white blood cells are normal in number and appear normal to the eye. This helps exclude consideration of leukemia and/or aplastic anemia, among other causes of thrombocytopenia. The presence of unusual cells in the blood or additional abnormalities in the blood counts might indicate the need for a bone marrow biopsy to exclude other causes of impaired platelet production and/or consideration of secondary ITP.
ITP Treatment
Treatment for thrombocytopenia depends on its cause and severity. The main goal of treatment is to prevent death and disability caused by bleeding.
If the condition is mild, the patient may not need treatment. A normal platelet count is unnecessary to prevent bleeding, even with severe cuts or accidents.
Thrombocytopenia often improves when its underlying cause is treated. People who inherit the condition do not usually need treatment. If a reaction to a medicine is causing a low platelet count, the doctor may prescribe another medicine. Most people recover after the initial medicine has been stopped. For heparin-induced thrombocytopenia (HIT), stopping the heparin is not enough. Often, the patients need another medicine to prevent blood clotting. If the immune system is causing a low platelet count, the doctor may prescribe medicines to suppress the immune system.
One treatment approach is to direct treatment to the etiology of thrombocytopenia (e.g., discontinuation of the drug that caused thrombocytopenia, treatment of the underlying infection, immunoglobulin G (IgG) replacement, chemotherapy directed at CLL). Unfortunately, only in a minority of cases is the etiology of thrombocytopenia clear and the cause found. In addition, in some cases, "curing" the underlying medical cause of the ITP may not change the platelet count. In the case of severe bleeding, if the etiology of thrombocytopenia is unknown but not thought to be immunologic, platelet transfusion can provide an immediate platelet increase. In contrast, if the underlying cause is immune, the effect from platelet transfusion may be minimal and, at best, very short-lived. It should be reserved for life-threatening bleeding (ideally transfused following intravenous immunoglobulin to "protect" the platelets).
The ITP epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases of ITP, age-specific diagnosed prevalent cases of ITP, and gender-specific diagnosed prevalent cases of ITP in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), United Kingdom, and Japan from 2020 to 2034.
The drug chapter segment of the ITP report encloses a detailed analysis of the marketed and the late, mid, and early stage (Phase III, Phase II, Phase I/II, and Phase I) pipeline drugs. The marketed drugs segment encloses drugs such as TAVALISSE/TAVLESSE (fostamatinib disodium hexahydrate), DOPTELET (avatrombopag), NPLATE/ROMIPLATE (romiplostim, AMG-531), VYVGART (efgartigimod alfa-fcab), and others. The current emerging key players and their respective drug candidates include Rilzabrutinib (Sanofi), Ianalumab (Novartis), Mezagitamab (Takeda), and others. The drug chapter also helps understand the ITP clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, and the latest news and press releases.
Marketed Drugs
TAVALISSE/TAVLESSE (fostamatinib disodium hexahydrate): Rigel Pharmaceuticals, Kissei Pharmaceutical and Grifols
TAVALISSE is an orally bioavailable investigational agent developed by Rigel Pharmaceuticals and approved for treating patients suffering from persistent/chronic adult idiopathic thrombocytopenic purpura. The therapeutic candidate inhibits FcR-triggered, Syk-dependent cytoskeletal rearrangement during phagocytosis. As stated by Rigel Pharmaceuticals, fostamatinib has a unique mechanism of action, blocking IgG receptor signaling in both macrophages and B-cells via SYK kinase.
TAVALISSE was approved by the US FDA in April 2018 for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to previous treatment. It gained European approval in January 2020, launching in Germany and the UK by July 2020. In Japan, the MHLW approved TAVALISSE in December 2022, with its market launch in April 2023.
DOPTELET (avatrombopag): Sobi (Dova Pharmaceuticals) and Asahi Kasei Pharma
DOPTELET is an orally administered thrombopoietin receptor agonist that mimics the biological effects of thrombopoietin in stimulating the development and maturation of megakaryocytes, resulting in increased platelet count. For the treatment of ITP, DOPTELET has been approved in the US, Europe, etc., and is currently the subject of a Phase III clinical study in Japan by Sobi. DOPTELET received FDA approval in June 2019 and EMA approval in January 2021 for treating chronic ITP in adults unresponsive to other therapies (e.g., corticosteroids and immunoglobulins). In the Q2 2024 report, Sobi announced plans for regulatory submission in Japan and pediatric submissions in the US and EU in the second half of 2024.
Emerging Drugs
Rilzabrutinib (PRN-1008): Sanofi/Principia Biopharma
Rilzabrutinib is an oral, reversible, covalent BTK inhibitor that has the potential to be a first- or best-in-class treatment of several immune-mediated diseases. BTK, expressed in B cells, mast cells, and other cells from the innate immune system, plays a critical role in inflammatory pathways and multiple immune-mediated disease processes. With the application of Sanofi's TAILORED COVALENCY technology, rilzabrutinib can selectively inhibit the BTK target.Currently trhe drug is being investigated in Phase III trial.
In April 2024, Sanofi announced positive results from the LUNA 3 Phase III study demonstrating that rilzabrutinib 400 mg twice daily orally achieved the primary endpoint of durable platelet response in adult patients with persistent or chronic ITP. As per the news published in April 2024, Sanofi anticipates regulatory submissions of rilzabrutinib in the US and EU by year-end.
VAY736 (Ianalumab): Novartis
Ianalumab is a novel, fully human immunoglobulin G1 monoclonal antibody that targets the BAFF receptor and has a unique dual mechanism of action: direct antibody-dependent cellular cytotoxicity-mediated B-cell depletion and inhibition of B-cell differentiation, proliferation, and survival via blockade of BAFF-R-mediated signaling. Currently, ianalumab is in Phase III of the clinical trial for 1st and 2nd line treatment of ITP.
In Novartis' Q2 2024 presentation, the company projected the upcoming results of two important trials: NCT05653349 (VAYHIT1) for first-line therapy, expected in 2026, and NCT05653219 (VAYHIT2) for second-line therapy, anticipated in 2025.
Drug Class Insights
The emergence of new therapeutic classes in ITP is showing strong potential, including BTK inhibitors, BAFF-R inhibitors, anti-CD38 antibodies, CXCR5 antagonists, and BAFF/APRIL antagonists. Currently, Takeda's Mezagitamab is the only anti-CD38 monoclonal antibody in clinical trials for ITP, with Phase II studies underway (NCT04278924).
Spleen tyrosine kinase (SYK) inhibitor
TAVALISSE/TAVLESSE (fostamatinib) is a highly selective SYK inhibitor with significant immunomodulatory potential, targeting Fc and B-cell receptor signaling pathways. It competes with various existing therapies and emerging drug candidates for ITP treatment. Notably, post-hoc data analysis suggests that TAVALISSE achieves higher response rates when used as a second-line therapy. In October 2022, Grifols' TAVLESSE (fostamatinib) received a NICE recommendation for treating refractory chronic immune thrombocytopenia. Currently, TAVALISSE is the sole FDA-approved SYK inhibitor, while Genosco/Oscotec's SKI-O-703 (cevidoplenib) is under investigation in Phase II trials.
Neonatal Fc receptor inhibitor
VYVGART (efgartigimod alfa) shows promise in treating various IgG-mediated autoimmune diseases, and patients in Japan now have access to this new treatment option for ITP. In March 2024, the FDA approved VYVGART for adults with primary immune thrombocytopenia, based on findings from the global Phase III ADVANCE-IV trial, published in the September 2023 issue of The Lancet. The trial met its primary endpoint, revealing that a greater percentage of chronic ITP patients treated with VYVGART achieved a sustained platelet count response compared to those on placebo. Moreover, VYVGART was well-tolerated during the 24-week study, with its safety and tolerability profile aligning with results from previous clinical trials.
Corticosteroids are the primary first-line treatment for ITP, often combined with Intravenous Immunoglobulin (IVIg) or anti-Rh(D) to boost platelet counts in urgent cases. However, durable remissions are rare, and steroid-related side effects usually limit their use to about four weeks, leading many patients to progress to persistent or chronic ITP, requiring alternative therapies. Long-term management of chronic ITP typically involves cycling through various treatments, including splenectomy, Thrombopoietin-Receptor Agonists (TPO-RAs), and immunosuppressants like rituximab. The varied mechanisms and inconsistent response rates of these therapies complicate comparisons, and there is no clear consensus on treatment sequencing. Despite these options, many patients experience prolonged thrombocytopenia, increasing their risk of spontaneous or trauma-induced bleeding. TAVLESSE offers a novel mechanism of action, adding a valuable option to the current ITP treatment landscape. Other FDA-approved therapies in the US that promote platelet production through TPO receptor binding include PROMACTA, NPLATE, and DOPTELET. VYVGART is approved for ITP in Japan, and YIMMUGO (BT-595) is available in Germany from 2022 for chronic ITP in adults.
Anti-RhD immunoglobulin has been successfully used in the setting of both acute and chronic ITP. Anti-RhD comprises immunoglobulin G (IgG) prepared from the plasma of repeatedly immunized human RhD-negative donors. Anti-RhD IgG itself contains >90% polyclonal immunoglobulin G anti-RhD.
Thrombopoietin receptor agonists (TPO-RAs) are new promising drugs in ITP treatment. Thrombopoietin is the primary factor responsible for platelet production. Hence, TPO-RAs are able not only to promote platelet production from existing megakaryocytes but can also enhance the proliferation of megakaryocytes in the bone marrow. This has been investigated in both in vitro studies and clinical trials. Amgen's ROMIPLATE and Novartis' REVOLADE are TPO-RAs approved by the European Commission for ITP patients. Both agents increase the platelet level in ITP patients and healthy volunteers.
New therapies featuring innovative mechanisms of action-such as FcRn inhibitor, BTK inhibitor, SYK inhibitor, and CD38 targeting-offer more personalized management strategies for refractory ITP. Rilzabrutinib, a BTK inhibitor, addresses both Fc? receptor-mediated functions and autoantibody production, while Ianalumab (VAY736), an anti-BAFF-R monoclonal antibody, and Mezagitamab, an anti-CD38 monoclonal antibody, are in late and mid-stage trials, respectively. Additionally, Cevidoplenib (SKI-O-703), a SYK inhibitor, shows promising efficacy in refractory cases. Together, these therapies represent significant advancements in addressing unmet needs in ITP, offering patients more tailored and effective treatment options.
The ITP market is positioned for substantial growth, driven by the introduction of novel therapies, including biologics, small molecules, and targeted treatments. Factors such as increased awareness, enhanced diagnostic capabilities, and a broader range of treatment options are propelling market expansion.
Key Updates
This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2020-2034. The landscape of ITP treatment has experienced a profound transformation with the uptake of novel drugs. Rilzabrutinib (PRN-1008), an innovative BTK inhibitor developed by Sanofi, anticipated to enter the market in 2025. Rilzabrutinib is anticipated to take medium fast uptake in the ITP.
ITP Pipeline Development Activities
The report provides insights into therapeutic candidates in Phase III, Phase II, Phase I/II, and Phase I. It also analyzes key players involved in developing targeted therapeutics.
Pipeline Development Activities
The report covers information on designation, collaborations, acquisitions and mergers, licensing, and patent details for ITP emerging therapy. For Example, In March 2024, the FDA granted cevidoplenib (SKI-O-703 an ODD for the treatment of patients with ITP. The designation was given after the results of a Phase II clinical study that demonstrated the efficacy of cevidoplenib in patients with chronic ITP.
KOL Views
To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on ITP's evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including oncologists, radiation oncologists, surgical oncologists, and others.
Delveinsight's analysts connected with 30+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Centers such as - Massachusetts General Hospital & Harvard Medical School, Graduate School in Hospital Pharmacy, Queen Mary University of London, Massachusetts General Hospital, Harvard Medical School, Boston, etc., were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or ITP market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.
Qualitative Analysis
We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Analyst views. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.
Market Access and Reimbursement
? TAVALISSE/TAVLESSE (fostamatinib)
Rigel Pharma's, Kissei Pharmaceutical's, and Grifols' TAVALISSE/TAVLESSE is the only oral Spleen Tyrosine Kinase (SYK) inhibitor for the treatment of adult patients with chronic ITP who have had an insufficient response to a previous treatment. TAVALISSE was approved in the US and EU in 2018 and 2020, respectively. It was also approved in Japan for marketing authorization in 2022 and was launched in 2023.
Rigel offers services and support for TAVALISSE. Rigel OneCare provides dedicated support for patients and practices. Rigel OneCare will help patients get started with TAVALISSE eligibility and limitations:
Eligible patients may receive TAVALISSE at a USD 15 co-pay for each prescription if they pay through commercial insurance.
? DOPTELET (avatrombopag)
DOPTELET is a small molecule thrombopoietin receptor agonist that increases platelet production. DOPTELET is a prescription medicine used to treat low blood platelet counts in adults with chronic ITP when other treatments have not worked well. DOPTELET is not used to make platelet counts normal in adults with chronic liver disease or chronic ITP. The company has stated that the price of avatrombopag is GBP 640.00 or GBP 960.00 per 5-day treatment course for the 40,000 to below 50,000 and below 40,000 platelets per µL of blood groups, respectively.
DOPTELET is available for 97% of commercial and 75% of Medicare-insured patients nationwide.
Dova Pharmaceutical's DOPTELET USD 0 Co-pay Program assists patients in getting DOPTELET at 0 cost. Eligible commercially insured patients may qualify for the DOPTELET USD 0 Co-pay Program. Eligible patients pay USD 0 for each DOPTELET prescription-annual maximum benefit up to USD 15,000.
Eligibility
Terms and condition