Biologics

FDA Orders Stricter Safety Labeling for Skysona

FDA has mandated significant labeling changes for Bluebird Bio’s Skysona (elivaldogene autotemcel), a gene therapy approved for treating early, active cerebral adrenoleukodystrophy (CALD), after seeing an increase in reports of life-threatening hematologic malignancies among patients who received the treatment.

Skysona, a lentiviral autologous hematopoietic stem cell (HSC)-based gene therapy, was initially approved in 2022 for boys aged 4 to 17 with CALD. At the time of approval, the therapy’s risk profile included a 4% incidence (3 of 67 patients) of myelodysplastic syndrome (MDS), according to an FDA safety update. However, new data from clinical trials now indicate 10 of 67 patients (15%) have developed serious blood cancers, including MDS and acute myeloid leukemia — a more than threefold increase in incidence.

The FDA's updated review revealed that the time to cancer diagnosis following treatment ranged from 14 months to 10 years, and in many cases, malignancies occurred before Skysona had time to potentially deliver therapeutic benefit. Nine of the ten affected patients required allogeneic hematopoietic stem cell transplants (allo-HSCT) —some with chemotherapy—and one patient died from treatment-related complications.

As a result, the FDA has required revisions to Skysona’s prescribing information, including:

Boxed Warning: Updated to reflect increased risk of hematologic malignancy.
Indications and Usage: Now restricted to patients without an available HLA-matched allo-HSC donor.
Warnings and Precautions and Adverse Reactions – Clinical Trials Experience: Expanded to include recent safety data.

In effect, FDA says Skysona should now only be used as a last resort in CALD patients who lack other treatment options. Skysona was granted accelerated approval, and its continued availability depends on confirmation of clinical benefit through ongoing studies. A 15-year postmarketing safety study is currently underway to monitor long-term outcomes and the risk of secondary cancers, including periodic assessments for clonal expansion — an early indicator of malignancy.

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