Biologics

Biomarin Reports Leukemia in Patient on Gene Therapy

BioMarin Pharmaceutical says it reported an adverse event to FDA about a patient participating in the company’s hemophilia A Phase 3 gene therapy trial of valoctocogene roxaparvovec (BMN 270) being diagnosed with B-cell acute lymphoblastic leukemia. “Based on BioMarin’s assessment of the case to date, including initial genetic testing of the leukemic cells, BioMarin believes at this time that this cancer is unrelated to BMN 270,” the company says. “The overall rate of all cancers observed in all BMN 270 trial patients (two in approximately 400 patient years of observation) appears consistent with expected rates of cancer in persons with hemophilia.”

The company says it has not been issued any clinicals holds on any trial at this time. “Currently, all trials of BMN 270 are ongoing, including additional enrollments, with no modifications, and BioMarin does not expect to make any modifications in the future due to this event,” it says. “The company’s independent data safety monitoring board agreed that this case did not require any modification to BioMarin’s ongoing trials at this time.

In 8/2020, FDA issued a complete response letter on a BLA for the therapy that requested two years of data from the Phase 3 study to provide evidence of a durable effect using annualized bleeding rate as the primary endpoint. BioMarin says it will file a resubmission by the end of the month. Last month, the European Commission granted conditional marketing authorization for the treatment. “Typically, BLA resubmissions are followed by a six-month review procedure,” BioMarin says. “However, the company anticipates three additional months of review may be necessary based on the number of data read-outs that will emerge during the procedure.”

Additionally, the company said in May that based on “recent feedback received from the FDA related to BioMarin’s plans for the upcoming BLA, the agency has requested additional information and analyses of data to be included in the BLA prior to submission.” While the agency has not requested additional pre-clinical or clinical studies, according to the company, data on three years after dosing will become available during the expected BLA review.

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