Drugs

BioMarin Expanded Indication for Voxzogo

FDA has approved a BioMarin Pharmaceutical supplemental NDA for Voxzogo (vosoritide) to increase linear growth in pediatric patients with achondroplasia with open epiphyses (growth plates). The company says the indication was approved under accelerated approval based on an improvement in annualized growth velocity. Previously, the drug was indicated for children who were five years of age and older, and the expanded indication now includes children of all ages.

Approval was based on data from a Phase 2 clinical trial evaluating the safety and efficacy of Voxzogo in children aged five years and under. “Based on the results of this trial, together with evidence from the adequate and well-controlled Phase 3 study in pediatric patients aged five years and older (Study 111-301), safety and effectiveness of Voxzogo have been established in pediatric patients of all ages for the improvement in linear growth in children with achondroplasia with open epiphyses,” BioMarin says. The drug’s overall safety profile in children under five years of age was similar to that seen in older children, it adds.

Achondroplasia is the most common form of skeletal dysplasia leading to disproportionate short stature in humans, according to BioMarin. It is characterized by slowing of endochondral ossification, which causes “disproportionate short stature and disordered architecture in the long bones, spine, face, and base of the skull,” 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