Hydroxyurea

A new study led by Ted Wun shows stroke incidence in sickle cell disease (SCD) is increasing, contrary to prior declines post-STOP trial. Risk factors include age, frequent hospitalization, high blood pressure, and prior transient ischemic attacks. The study highlights the need for better adherence to screening guidelines and increased use of preventive therapies.

Stroke rates in sickle cell disease patients continue to rise despite preventive measures, with risk doubling every 20 years and increasing 13-fold between ages 20 and 60. Recent data shows highest stroke rates in the 2010s across all age groups, emphasizing the need for regular screenings and preventive steps for high cholesterol, high blood pressure, and other risk factors.

Children ages 12+ with sickle cell disease now eligible for FDA-approved gene therapy Casgevy at Children's Wisconsin, which reduces severe vaso-occlusive crises and transfusion dependence. Casgevy uses CRISPR-modified stem cells to produce fetal hemoglobin, offering fewer side effects than traditional treatments.

Bluebird Bio's $3.1 million gene therapy Lyfgenia for sickle cell disease has seen slow uptake, with only four patients treated since its launch. Payer approval and patient readiness are cited as significant barriers. The high cost and complex reimbursement process highlight systemic issues in the adoption of cell and gene therapies. Despite slow initial uptake, experts anticipate a gradual increase as the healthcare system adapts to transformative treatments.

Akums Drugs and Pharmaceuticals has patented a room temperature stable oral suspension of Hydroxyurea, aimed at managing Sickle Cell Disease, addressing storage and accessibility challenges. The formulation, stable at room temperature, is a significant advance for widespread distribution, particularly in tribal areas with limited cold storage facilities.

Akums Drugs and Pharmaceuticals has secured an Indian patent for a room temperature stable oral suspension of hydroxyurea, aimed at managing sickle cell disease. This formulation, stable at room temperature, addresses the need for widespread distribution, especially in tribal areas with limited cold storage facilities. The patented formulation is more affordable and offers dose flexibility, particularly beneficial for pediatric and adolescent patients.

Akums Drugs and Pharmaceuticals has patented a room-temperature stable Hydroxyurea Oral Suspension for managing Sickle Cell Disease, eliminating the need for refrigeration and offering flexible dosing at a lower cost.

A phase 3 trial (NCT06456346) has begun to assess bomedemstat (MK-3543; IMG-7289) for essential thrombocythemia (ET) patients not previously treated with cytoreductive therapy. The Shorespan-007 trial will compare bomedemstat with standard-of-care hydroxyurea in treatment-naive ET patients, with primary end points including durable clinicohematologic response rate and secondary end points like duration of hematologic remission and event-free survival. Bomedemstat has received FDA orphan drug and fast track designations for ET and myelofibrosis.