Biologics

Protalix Gets ‘Complete Response’ on Fabry Therapy

FDA has issued Protalix BioTherapeutics a Complete Response Letter (CRL) for its BLA seeking accelerated approval for pegunigalsidase alfa (PRX–102) for treating adult patients with Fabry disease. The letter did not cite any safety or efficacy concerns regarding the therapy, according to the company.

The letter also noted that an inspection of Protalix’s manufacturing facility in Carmiel, Israel, including the agency’s subsequent assessment of any related findings, is required before it can approve the BLA. “Due to travel restrictions, the FDA was unable to conduct the required inspection during the review cycle,” it says. “With respect to the third-party facility in Europe at which fill and finish processes are performed for PRX-102, due to Covid–19, the FDA reviewed records under Section 704(a)(4) of the Federal Food, Drug, and Cosmetic Act in lieu of a pre-licensing inspection. In the CRL, the FDA stated that it will communicate remaining issues to the facility in order to seek prompt resolution of any pending items.”

Additionally, FDA indicated that because Genzyme recently converted Fabrazyme’s accelerated approval to full approval, this must be “addressed in the context of any potential resubmission seeking accelerated approval of PRX 102,” the company says. This likely means the agency is raising the bar on “unmet clinical need” in light of Fabrazyme’s full approval. “Protalix intends to work collaboratively with the agency to identify the most expeditious pathway to approval, including accelerated approval,” Protalix says.

Protalix describes pegunigalsidase alfa as an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant α-Galactosidase-A enzyme.

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