Biologics

Panel Backs Sickle Cell Gene Therapy Safety

FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee supported Vertex Pharmaceuticals and CRISPR Therapeutics safety assessments on their sickle cell disease (SCD) gene therapy exagamglogene autotemcel (exa-cel). This moves the therapy one step closer to FDA approval which would make it the first CRISPR gene-edited therapy for a genetic disease. The agency has set a 12/8 user fee review action target date on the companies’ submission.

According to a briefing document released in advance of the meeting, reviewers said they were seeking input on “whether the off-target analysis (e.g., in silico and cellular methods)” performed by the companies is adequate to assess risk in the intended SCD patient population in the U.S. The reviewers further noted that it was “not clear if the limited SCD donor cells used for the off-target assessment will adequately inform the potential safety risks of exa-cel.”

The reviewers emphasized the current “unmet need” from current available treatments for SCD, which include hydroxyurea, L-glutamine, voxelotor, and crizanlizumab. The disease is associated with vaso-occlusive crises, “which most commonly are severe painful events, but may also present with acute chest syndrome, priapism, or hepatic/splenic sequestration,” they write. “In the longer term, SCD may lead to life-threatening neurologic, pulmonary, cardiac, and renal complications and a shortened life span (survival).”

In June, the company released efficacy data on 17 patients with SCD who had received exa-cel at the time of the analysis which showed that 94% achieved the primary endpoint of freedom from vaso-occlusive crises (VOCs) for at least 12 consecutive months. “Mean duration of VOC-free was 18.7 months, with a maximum of 36.5 months. 17/17 (100%) achieved the key secondary endpoint of being free from hospitalizations related to VOCs for at least 12 consecutive months…” the company said at the time.

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