Drugs

Homology Clinical Hold on Gene Therapy Lifted

FDA has removed a clinical hold on Homology Medicines and its pheNIX gene therapy clinical trial evaluating HMI-102 in adults with phenylketonuria (PKU). Homology says it is planning to inform clinical trial sites about changes to the protocol, which include a new steroid-sparing immunosuppression regimen that incorporates a T-cell inhibitor and a shorter course of steroids.

The company previously disclosed that the clinical hold on the pheNIX trial was related to elevated liver function tests observed in the trial, which it says were all resolved with no hospitalizations. Homology says its response to FDA included changes to the protocol intended to enhance risk-mitigation measures and a new immunosuppression regimen with the T-cell inhibitor tacrolimus, which will be used in combination with a reduced duration of prophylactic steroids. The same approach is being used in Homology’s ongoing pheEDIT gene editing trial for PKU and juMPStart gene therapy trial for Hunter syndrome. “Similar regimens have been shown to dampen the immune response to AAVs in the clinical setting,” it says.

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