Drugs

Regenxbio Gets ‘Complete Response’ for Gene Therapy

FDA has issued Regenxbio a complete response letter (CRL) on its BLA seeking accelerated approval for RGX-121 (clemidsogene lanparvovec), a one-time gene therapy for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. In the CRL, FDA cited several concerns, including uncertainty in defining the neuronopathic patient population, questions about the comparability of natural history external controls, and whether the proposed CSF HS D2S6 biomarker is a reliable surrogate endpoint to predict clinical benefit, according to the company. The agency suggested multiple potential paths forward, including additional studies, longer-term follow-up, and inclusion of untreated control arms — options that are particularly challenging in an ultra-rare disease population like MPS II, it says.

“This decision is devastating for the families of boys living with this progressive, life-threatening disease,” company CEO Curran Simpson is quoted in a release as saying. He added that the company remains confident in the long-term potential of RGX-121 and plans to work with FDA to clarify the neuronopathic patient population and provide additional clinical data. Regenxbio intends to request a Type A meeting to discuss next steps and aim for a BLA resubmission as quickly as possible.

The RGX-121 program has been in development for more than a decade, according to the company. The therapy is designed to deliver the iduronate-2-sulfatase (IDS) gene) to the central nervous system (CNS), potentially providing a permanent source of the enzyme and enabling long-term correction of affected cells. Across the CAMPSIITE I/II/III trials, RGX-121 has been well-tolerated and supported by biomarker, functional, and safety data, including outcomes through 12 months, it says.

MPS II is a rare X-linked disease caused by a deficiency in IDS, leading to accumulation of glycosaminoglycans in tissues and progressive CNS dysfunction, according to the company. Severe forms of the disease result in developmental delays apparent by 18–24 months and early death in adolescence without effective intervention.

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