PU-H71 receives orphan drug designation for myelofibrosis

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Micrograph showing myelofibrosis
The US Food and Drug Administration (FDA) has granted orphan drug designation to PU-H71 to treat patients with myelofibrosis. The drug specifically targets the epichaperome, a network of high-molecular- weight complexes found in multiple diseases, including cancer and neurologic disorders. These complexes enhance cellular survival, irrespective of tissue of origin or... [Read Article]
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Mircera approved for anemia in pediatric patients with CKD

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Red blood cells
Mircera®, methoxy polyethylene glycol-epoetin beta, was approved by the US Food and Drug Administration (FDA) to treat anemia in pediatric patients who have chronic kidney disease (CKD). The drug is indicated for patients ages 5 to 17 years on hemodialysis who are switching from another erythropoiesis-stimulating agent (ESA) after their hemoglobin levels... [Read Article]
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Emicizumab granted priority review for hemophilia A without inhibitors

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Bleeding human finger
By Crystal (Crystl) from Bloomington, USA - Flickr
The US Food and Drug Administration (FDA) has granted priority review for emicizumab (Hemlibra®) for adults and children with hemophilia A without factor VIII inhibitors. Earlier this year, the agency awarded emicizumab breakthrough therapy designation for the same population. Emicizumab is a bispecific factor... [Read Article]
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FDA approves first biosimilar pegfilgrastim

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Vials and a syringe
The US Food and Drug Association (FDA) has approved pegfilgrastim-jmdb (Fulphila™) as the first biosimilar to Neulasta®. The agents reduce the risk of infection or the duration of febrile neutropenia in patients treated with immunosuppressive chemotherapy for non-myeloid hematologic malignancies. The FDA approved Fulphila based on evidence that included extensive... [Read Article]
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Eltrombopag receives priority review designation for SAA

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Eltrombopag (Promacta)
Photo courtesy of GSK
Eltrombopag (Promacta®) in combination with standard immunosuppressive therapy (IST) has received priority review designation from the US Food and Drug Administration (FDA) for first-line treatment of severe aplastic anemia (SAA). The drug is already approved for SAA in the refractory setting for patients who have had an insufficient... [Read Article]
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