Cystic fibrosis (CF) is a genetic disease that results in lung infections and greatly impairs lung function.1 Mutations in the CF transmembrane conductance regulator (CFTR) gene affect CFTR protein function, rendering it unable to maintain chloride and sodium movement within physiological conditions. The lack of chloride on the cell surface results in less water being…
Sickle cell disease (SCD) is an inherited blood disorder characterized by the presence of abnormal hemoglobin S (HbS). HbS polymerization causes red blood cells (RBCs) to deform into a crescent or sickle shape. Sickle cells are less flexible and deformable than normal RBCs.They adhere to other cells, interrupt vascular blood flow, and trigger vaso-occlusive events,…
Oxbryta® (voxelotor), previously known as GBT440, is an oral daily prescription medicine developed by Global Blood Therapeutics (GBT) that is indicated for the treatment of sickle cell disease (SCD) in patients aged 12 years and older.1 The active pharmaceutical ingredient of Oxbryta is voxelotor, a hemoglobin S (HbS) polymerization inhibitor. Oxbryta was approved under accelerated…
Adakveo® (crizanlizumab-tmca) is a prescription medicine indicated for preventing vaso-occlusive crises (VOCs) in patients aged 16 years and older with sickle cell disease.1,2 Adakveo was developed by Novartis Pharmaceuticals and was approved by the US Food and Drug Administration (FDA) in November 2019.3 Sickle cell disease, which is inherited in autosomal-recessive fashion, is caused by…
Progress in sickle cell disease (SCD) was stifled for almost 2 decades prior to the US Food and Drug Administration (FDA) approval of Endari™ (L-glutamine oral powder) in 2017. It is the second drug to gain FDA approval for patients with SCD and the first new drug to enter the arena in almost 20 years.1…
Hydrea and Droxia are brand names for hydroxyurea, a myelosuppressive agent.1-3 Hydroxyurea is indicated for the treatment of resistant chronic myeloid leukemia and locally advanced squamous cell carcinomas of the head and neck.1 This drug has also been approved for reducing the frequency of pain crises in patients with sickle cell disease (SCD) and their…
Overview Before 2019, no FDA-approved drugs were available for the treatment of neuromyelitis optica spectrum disorder (NMOSD). In 2019, Solaris (eculizumab) was the first drug to be approved; approval of Uplizna (inebilizumab-cdon) and Enspryng (satralizumab-mwge) followed in 2020.1–3 Enspryng is an interleukin 6 (IL-6) receptor antagonist indicated for the treatment of NMOSD in adult patients…
Uplizna® (inebilizumab-cdon) is a cytolytic monoclonal antibody directed againt CD19. It is indicated for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD).1 NMOSD is an autoimmune demyelinating disease of the central nervous system (CNS) characterized by recurrent attacks of optic neuritis and transverse myelitis. These attacks often lead to…
Soliris® (eculizumab) is a terminal complement protein inhibitor developed and commercialized by Alexion Pharmaceuticals that is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 water channel autoantibody (AQP4-IgG)-positive.1,2 In the EU, eculizumab is also indicated for patients with relapses of the disease.1 Eculizumab is a recombinant humanized monoclonal…
Prior to 2019, there were no US Food and Drug Administration (FDA)-approved therapies for the treatment of neuromyelitis optica spectrum disorder (NMOSD). There has been substantial progress since then, with the approval of Soliris® (eculizumab) in 2019 followed by Uplizna® (inebilizumab-cdon) and Enspryng™ (satralizumab-mwge).1-3 There are also 3 monoclonal antibodies in the experimental phase that…
