Drugs

FDA Alert on Skysona Hematologic Malignancies

FDA says it has received serious adverse event reports associated with Bluebird Bio’s Skysona (elivaldogene autotemcel). A just-posted safety alert says that since its approval in April the agency has received reports about hematologic malignancies, including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia. The autologous hematopoietic stem cell-based gene therapy is indicated to slow the progression of neurologic dysfunction in boys aged 4-17 years with early, active cerebral adrenoleukodystrophy.

“FDA is investigating the known risk of hematologic malignancies with serious outcomes, including those such as hospitalization, the requirement for allogeneic hematopoietic stem cell transplantation, and death, and is evaluating the need for further regulatory action,” the alert says. “Given the risk of hematologic malignancy, providers should carefully consider alternative therapies, including allogeneic hematopoietic stem cell transplant for patients who have a suitable, willing, and available human leukocyte antigen (HLA)-matched donor, prior to deciding to treat a child with Skysona.”

The agency notes that the prescribing information includes information (e.g., Boxed Warning) about the serious risk of developing hematologic malignancies following treatment with Skysona. “Patients and clinical trial participants receiving treatment with Skysona should be monitored lifelong for hematologic malignancy,” it says. “Therefore, patients should be closely monitored with complete blood counts at least every three months and through assessments for evidence of clonal expansion or predominance at least twice in the first year after Skysona administration and annually thereafter, and bone marrow evaluations should be considered as clinically indicated.”

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome