Drugs

Ionis/AstraZeneca Planning NDA for Eplontersen

Ionis Pharmaceuticals and AstraZeneca plan to submit an NDA for eplontersen later this year for treating patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) based on new study results announced 9/7. ATTRv is a severe, progressive, and life-threatening disease caused by the abnormal formation of the transthyretin (TTR) protein.

An interim analysis of the Phase 3 NEURO-TTRansform study of eplontersen found the therapy demonstrated a statistically significant and clinically meaningful change from baseline for its co-primary and secondary efficacy endpoints compared to a placebo, according to the companies. Eplontersen also demonstrated a favorable safety and tolerability profile, they say.

In the study, eplontersen achieved an 81.2% mean reduction in the co-primary endpoint of serum TRR concentration compared to baseline, demonstrating reduced TTR protein production, Ionis and AstraZeneca say. The therapy also demonstrated a significant treatment effect on the co-primary endpoint of modified Neuropathy Impairment Score +7 with a statistically significant difference in mean change from baseline for those treated with eplontersen versus placebo.

Eplontersen has been granted an orphan drug designation. It is also currently being evaluated in the Phase 3 CARDIO-TTRansform study for amyloid transthyretin cardiomyopathy, a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis.

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