Hydroxyurea

CMS and manufacturers of sickle cell disease gene therapies, Lyfgenia and Casgevy, enter agreements for the Cell and Gene Therapy Access Model, aiming to improve patient access through outcomes-based agreements.

Sanofi's rilzabrutinib shows promise in treating immune thrombocytopenia, while Novo Nordisk's etavopivat reduces sickle cell pain crises. Beam Therapeutics' CRISPR therapy for sickle cell yields consistent results, though fertility preservation remains a concern.

Five studies at the 66th ASH Annual Meeting present new treatments for sickle cell disease (SCD) and immune thrombocytopenia (ITP), including a first-in-class oral medication for ITP, experimental drugs for SCD reducing pain episodes and improving red blood cell health, hydroxyurea benefits for HbSC variant, and a base-edited gene therapy showing robust results in early trials.

Beti-cel (ZYNTEGLO™) treatment for beta-thalassemia shows durable transfusion independence and normal hemoglobin levels up to 10 years post-treatment, with a favorable safety profile. 81% of participants have over 5 years of follow-up, and 90.2% achieved transfusion independence. Beti-cel is a one-time gene therapy approved for adult and pediatric patients requiring regular red blood cell transfusions.

Incyte to present new oncology data at 2024 ASH Annual Meeting, including late-breaking results from the Phase 3 inMIND study on tafasitamab in follicular lymphoma. The company will also host a virtual analyst and investor event on December 12, 2024, to discuss key data presentations.

Incyte presents 20 oncology data presentations at 2024 ASH Annual Meeting, including Phase 3 inMIND study results on tafasitamab in follicular lymphoma. Hosts virtual analyst and investor event on December 12, 2024.

Combination of ruxolitinib and low-dose Peg-IFN-α2a in newly diagnosed PV patients showed increased cell counts, decreased JAK2 V617F VAF, and acceptable toxicity. 52% and 56% ORR at 12 and 24 months, respectively. 52% and 68% overall MR at 12 and 24 months. Median JAK2 V617F VAF decreased from 47% to 7% after 2 years. BMHR in 20% and 16% at 12 and 24 months. Normal spleen in all patients after 24 months. Study supports long-term clinical effect of this therapy.

Intas Pharmaceutical received SEC approval for Phase-III Ruxolitinib cream trial, subject to protocol revision including study centers, 50% government hospital sites, and participant age ≥ 18 years. The study, an equivalence trial, requires submission of study need and design documents for review.

Two new gene editing medications for sickle cell disease, including the first FDA-approved CRISPR-based drug, face barriers like high costs and complex delivery processes. Pfizer withdrew its sickle cell drug Oxbryta due to safety concerns, and patient uptake for new gene therapies has been slow. Despite these challenges, the sickle cell treatment market is expected to grow, with over 40 companies developing more than 50 treatments. Cellarity is developing an oral drug to induce fetal hemoglobin, aiming to provide a more accessible treatment option.

The FDA granted orphan drug status to Kind Pharmaceuticals' AND017 for treating sickle cell disease, offering incentives like tax credits and market exclusivity. AND017, which inhibits HIF-PH to boost red blood cell production, showed promising safety and efficacy in preclinical studies and Phase 1 trials, and is also being developed for anemia in other conditions.