Hematological Disorders

Majority of youth in Michigan and New York with sickle cell anemia did not have filled hydroxyurea prescriptions.

Hydroxyurea used by 24.6 percent with sickle cell disease, while l-glutamine, voxelotor, crizanlizumab were used by 2.0, 1.9, and 1.4 percent

The table provided is a review of notable updates that occurred in February 2023 for investigational products in development.

February 2023 Notable Drug Approvals

Valoctocogene roxaparvovec is an investigational adeno-associated virus (AAV) gene therapy.

The new autoinjector is designed with push-on-skin activation to be used in both in-office and at-home settings of care.

One infusion of etranacogene dezaparvovec noninferior and superior to factor IX prophylaxis

The mean annualized treated bleeding rate decreased by 84.5 percent from baseline to 104 weeks

Altuviiio temporarily replaces the missing coagulation factor VIII needed for effective hemostasis.

Elranatamab is a B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody.

Stimufend is a leukocyte growth factor.

The higher risk of hyponatremia among women vs men persisted regardless of diuretic type.

Crovalimab achieved disease control in patients with paroxysmal nocturnal hemoglobinuria who have not been previously treated with complement inhibitors.

The approval was supported extrapolation of efficacy data from the phase 3 HELP study, along with safety and pharmacodynamic data from the phase 3 SPRING study.

Daprodustat is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor.

The table provided is a review of notable updates that occurred in January 2023 for investigational products in development.

Draft recommendations around the new policy center on ‘individual risk-based questions to reduce the risk of transfusion-transmitted HIV’

Enjaymo is an immunoglobulin G subclass 4 monoclonal antibody.

The sNDA is supported by data from the phase 2 PIONEER trial that evaluated the efficacy and safety of avapritinib in patients with indolent systemic mastocytosis.

Researchers sought to determine whether corticosteroids would be an effective treatment for patients with chemotherapy-induced thrombocytopenia.