MedPath

Apadamtase alfa

Generic Name
Apadamtase alfa
Drug Type
Biotech
CAS Number
2086325-24-6
Unique Ingredient Identifier
U5SFU33XUX
Background

Apadamtase alfa is under investigation in clinical trial NCT03922308 (Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)).

Clinical Trials
Related News

Takeda's Adzynma Becomes First UK-Approved Treatment for Rare Blood Clotting Disorder cTTP

• The UK's Medicines and Healthcare products Regulatory Agency (MHRA) has approved Takeda's Adzynma (rADAMTS13) as the first treatment for congenital thrombotic thrombocytopenic purpura (cTTP) in the UK for patients of all ages. • Adzynma is an enzyme replacement therapy that provides a recombinant form of ADAMTS13, addressing the underlying enzyme deficiency that causes this ultra-rare inherited blood clotting disorder. • In clinical trials, no patients experienced acute thrombotic thrombocytopenic purpura events while receiving Adzynma as preventative treatment, demonstrating significant efficacy compared to plasma-based therapies.

Global Thrombocytopenia Clinical Trials Market Sees Significant Growth in 2025

• The global thrombocytopenia clinical trials landscape is expanding rapidly with over 25 pharmaceutical companies developing 25+ treatment therapies, according to recent market analysis. • Key industry players including GSK, Novartis, Amgen, and Sanofi are leading clinical development efforts, with several promising candidates in late-stage trials showing efficacy in reducing thrombocytopenia events. • Recent advances include Takeda's TAK-755 demonstrating 60% reduction in thrombocytopenia events compared to standard of care, and HUTCHMED completing enrollment for its pivotal Phase III ESLIM-01 trial of sovleplenib for immune thrombocytopenia.

Rusfertide Achieves Primary Endpoint in Phase 3 VERIFY Trial for Polycythemia Vera

• Rusfertide significantly reduced the need for phlebotomies in polycythemia vera (PV) patients, with 77% achieving clinical response compared to 33% on placebo. • The VERIFY trial met all key secondary endpoints, including hematocrit control and improved patient-reported outcomes, demonstrating comprehensive efficacy. • The hepcidin mimetic was well-tolerated, with no new safety concerns, suggesting a favorable risk-benefit profile for PV patients. • Protagonist and Takeda plan to submit detailed results for presentation at upcoming medical conferences and to regulatory agencies for potential approval.

Thrombocytopenia Treatment Market Shows Promising Growth with Novel Therapies and Clinical Trials

• The thrombocytopenia market is experiencing significant growth, driven by the introduction of novel therapies and ongoing clinical trials. • Key players like Sanofi, Takeda, and Amgen are actively involved in developing and marketing treatments for various types of thrombocytopenia. • Emerging therapies such as Rilzabrutinib and Nipocalimab show promise in addressing unmet needs in immune and alloimmune thrombocytopenia. • Clinical trials are exploring new treatments for chemotherapy-induced thrombocytopenia and myelofibrosis-related thrombocytopenia.

Takeda's Growth & Launch Portfolio Drives H1 2024 Revenue Increase; Full-Year Guidance Revised

• Takeda reported a 5% revenue increase in H1 2024, driven by an 18.7% growth in its Growth & Launch product portfolio, offsetting declines in VYVANSE sales. • The company raised its full-year guidance due to strong H1 performance and updated foreign exchange assumptions, while core operating profit margin reached 30.2%. • Takeda is actively investing in late-stage pipeline programs, including Phase 3 trials for TAK-861 (narcolepsy) and mezagitamab (IgA nephropathy). • Despite robust performance, Takeda anticipates challenges in H2 due to accelerated generic erosion of VYVANSE and increased R&D spending.

Takeda's Growth Products Drive Strong First Half, Phase 3 Trial of TAK-861 Initiated

• Takeda reports a 13.4% revenue increase in the first half of FY2024, driven by Growth & Launch Products, including ENTYVIO, and geographical expansion with ADZYNMA and FRUZAQLA. • The company has initiated a Phase 3 trial for TAK-861 in narcolepsy type 1, showcasing advancements in its late-stage pipeline and commitment to developing life-transforming treatments. • Takeda upgrades its full-year forecasts, reflecting stronger-than-anticipated performance and revised foreign exchange assumptions, while planning to increase R&D investment in the second half. • Management Guidance for FY2024 has been upgraded, with Core Revenue expected to be flat to slightly increasing and Core Operating Profit decline in the mid-single-digit percentages.

FDA Approves Takeda's Adzynma as First Treatment for Ultra-Rare Blood Clotting Disorder cTTP

• The FDA has approved Takeda's Adzynma (ADAMTS13, recombinant-krhn) as the first therapeutic option for congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder affecting fewer than 1,000 people in the US. • Clinical trials demonstrated Adzynma's efficacy with no patients experiencing acute TTP events while on prophylactic treatment, compared to one event in the plasma-based therapy group, offering a significant advancement over current plasma-based treatments. • Adzynma works by replacing the deficient ADAMTS13 enzyme that regulates blood clotting, providing both prophylactic and on-demand treatment options for adult and pediatric patients with cTTP, a condition that has a >90% mortality rate when left untreated.
© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy