Blood Disorders

Hematologists discussed the still limited treatment options for sickle cell disease at ASH, weighing the risks and benefits of disease-modifying versus curative therapies after the withdrawal of Pfizer’s Oxbryta (voxelotor).

Hematologists’ concerns about hepatoxicity with hemophilia A gene therapies may limit use, but Phase III data for Pfizer’s candidate at ASH show a lower rate of ALT increases than seen with BioMarin’s Roctavian.

Beam presented data from three more patients than during its last update for BEAM-101, showing stem cell engraftment and efficacy consistent with the first four patients.

Patients treated with Novo’s PKR activator had a nearly 50% reduction in vaso-occlusive events relative to placebo in Phase II, VOC occurrence was delayed and blood biomarkers improved.

TRiCares announced first implantation of the Topaz transfemoral tricuspid heart valve replacement system as part of the company’s EU pivotal study. If all goes to plan, the device will compete with Edwards’ Evoque system. The announcement follows the company’s $50m series D funding raise in July.

The second instalment of In Vivo’s three-part series delves into the therapeutic categories that will propel forecast pharmaceutical sales growth for 2025, focusing on blood malignancies, skin conditions and generalized cardiovascular disease.

The US FDA has cleared a novel software application to enhance the analysis of bone marrow smears. Using the AI-powered tool, hematopathologists may be able to better diagnose various blood and marrow diseases.

The US FDA is advising the public that certain plastic syringes from China could fail, leak, or break. If necessary, the agency may prevent Chinese syringes from entering the US.

As the US FDA works to finalize new regulation of lab-developed tests, it must consider more than two thousand comments that have poured into the agency since the proposed rule was published in October. The comment period closes Monday.

This week, the US FDA labelled several recalls class I, approved an at-home diagnostic for chlamydia and gonorrhea, and announced the expansion of its TAP pilot program. Guardant Health also announced it would appeal a verdict in a patent suit against the company and AdvaMed voiced its continued support for pending breakthrough device legislation.