Drugs

Complete Response on Hypoparathyroidism NDA

FDA has issued Ascendis Pharma a complete response letter on its NDA for TransCon PTH (palopegteriparatide), indicated for treating adults with hypoparathyroidism. The agency letter cited manufacturing control strategy concerns related to the variability of the delivered dose in the drug/device combination product, according to the company. FDA did not cite any concerns about the clinical data submitted as part of the NDA package and no new safety or efficacy studies were requested in the letter, it says. Ascendis plans to request a Type A meeting with the agency to understand the best path forward.

Last month, the company was given a heads-up by the agency when it told Ascendis that deficiencies identified in the company’s NDA prevented it from holding further discussions about labeling and post-marketing requirements/commitments. While not disclosing any specifics, an agency letter said that its position does not currently reflect the final regulatory decision on the company’s application, Ascendis said at the time.

The NDA was filed 9/2022 for the investigational prodrug that is designed to restore parathyroid hormone (PTH [1-34]) to physiological levels over 24 hours in adult patients with hypoparathyroidism. “Notably, 57 out of 59 patients continue in the open-label portion of the Phase 2 trial beyond two years of treatment, and 78 out of 79 patients continue in the open-label portion of the Phase 3 trial of TransCon PTH,” the company says. “In these studies, TransCon PTH has been generally well tolerated, with no discontinuations related to [the] study drug.”

Hypoparathyroidism is a rare endocrine disorder where patients experience insufficient levels of parathyroid hormone, which results in low calcium and elevated phosphate levels in the blood.

Gupta Raises Concerns About Overton’s Independence, Ideology

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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