Etavopivat

FDA Approves Novo Nordisk's Alhemo for Hemophilia A and B with Inhibitors

5 months ago

• The FDA has approved Alhemo (concizumab-mtci) as a once-daily prophylactic treatment for hemophilia A and B with inhibitors in patients 12 years and older. • Alhemo, a subcutaneous injection, is the first of its kind for this patient population, offering an alternative to intravenous infusions. • Clinical trials showed an 86% reduction in treated bleeding episodes with Alhemo compared to no prophylaxis, significantly improving patient outcomes. • Alhemo works by blocking tissue factor pathway inhibitor (TFPI), enhancing thrombin production to facilitate blood clotting in the presence of inhibitors.

Error parsing response

5 months ago

Error occurred while parsing the model response.

Etavopivat Shows Promise in Reducing Vaso-Occlusive Events in Sickle Cell Disease

5 months ago

• Phase II trial results reveal that etavopivat, a PKR activator by Novo, significantly reduces vaso-occlusive events (VOCs) in sickle cell disease patients by nearly 50% compared to placebo. • The study also demonstrated a delay in VOC occurrence and improvements in key blood biomarkers among patients treated with etavopivat. • Etavopivat-treated patients reported reduced fatigue, suggesting a potential improvement in the overall quality of life. • A Phase III trial is ongoing, with endpoints currently under discussion with regulatory authorities to further evaluate the efficacy and safety of etavopivat.

ASH 2024: Sanofi's Rilzabrutinib, Novo Nordisk's Etavopivat, and Beam's CRISPR Therapy Show Promise

5 months ago

• Sanofi's rilzabrutinib demonstrated improved platelet response and reduced bleeding in patients with immune thrombocytopenia (ITP) in a Phase 3 trial. • Novo Nordisk's etavopivat reduced pain crises in sickle cell disease patients by 46% compared to placebo in a Phase 2 study. • Beam Therapeutics' CRISPR therapy, BEAM-101, showed consistent results in increasing fetal hemoglobin production in sickle cell patients.

ASH 2024: Advances in Sickle Cell Disease and Immune Thrombocytopenia Therapies

5 months ago

• Researchers presented promising data on new therapies for sickle cell disease (SCD) and immune thrombocytopenia (ITP) at the ASH Annual Meeting. • Etavopivat significantly reduced vaso-occlusive crises in SCD patients in a Phase II trial, suggesting a novel approach to managing the condition. • Rilzabrutinib demonstrated durable platelet response in refractory ITP patients in a Phase III trial, offering a new option for challenging cases. • A gene therapy trial (BEAM-101) showed robust results in SCD patients, with high fetal hemoglobin production and no vaso-occlusive crises post-treatment.

Etavopivat Shows Promise in Reducing VOCs and Increasing Hemoglobin in Sickle Cell Disease

6 months ago

• Phase 2 HIBISCUS trial data indicates etavopivat may reduce vaso-occlusive crises (VOCs) in sickle cell disease patients. • Etavopivat demonstrated a trend toward reduced VOC incidence over 52 weeks and increased hemoglobin response at 24 weeks compared to placebo. • The investigational oral therapy also showed improvements in fatigue and hemolysis markers in SCD patients. • Phase 3 HIBISCUS trial will further evaluate etavopivat's efficacy and safety in a larger patient population.

Novo Nordisk Presents Promising Data on Sickle Cell Disease and Hemophilia Treatments at ASH 2024

6 months ago

• Novo Nordisk presented Phase 2 results from the HIBISCUS trial, showing etavopivat reduced vaso-occlusive crises in sickle cell disease patients. • Interim Phase 3 data from the FRONTIER4 trial highlighted the safety and efficacy of Mim8 prophylaxis in hemophilia A patients with or without inhibitors. • Phase 3 explorer7 study results demonstrated the efficacy of concizumab in hemophilia A or B patients with inhibitors, regardless of target joints at baseline.

FDA Approves Pyrukynd as First Treatment for Pyruvate Kinase Deficiency

a year ago

• The FDA has approved mitapivat (Pyrukynd) as the first treatment for adults with pyruvate kinase (PK) deficiency, a rare inherited anemia. • Pyrukynd, a small molecule, works by activating PK enzymes to improve red blood cell function, offering a new approach to managing this condition. • Clinical trials demonstrated that Pyrukynd significantly increased hemoglobin levels in non-transfusion-dependent patients and reduced transfusion burden in regularly transfused patients. • Agios Pharmaceuticals, now focused on rare genetic diseases, estimates that many PK deficiency cases remain undiagnosed, highlighting the need for increased awareness and testing.

Drug Development Updates