Hematological Disorders

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

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.

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.

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.

Lunsumio is a first-in-class CD20xCD3 T-cell engaging bispecific antibody that targets CD20 on the surface of B cells and CD3 on the surface of T cells.

Monoferric will be supplied in 100mg/mL, 500mg/5mL, and 1000mg/10mL single-dose vials for intravenous infusion after dilution.

The Food and Drug Administration has granted Orphan Drug designation to ARU-1801 (Aruvant) for the treatment of sickle cell disease.

A recently published report describes the case of a 49-year-old female patient who experienced aplastic anemia secondary to long-term metronidazole use and highlights the importance of monitoring for metronidazole-induced hematologic dyscrasias.