Drugs

Health Canada OKs ALS Drug Delayed at FDA

Health Canada has given Amylyx Pharmaceuticals its first worldwide approval for Albrioza (sodium phenylbutyrate and ursodoxicoltaurine), with conditions, for treating amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease. “Clinical data demonstrated a statistically significant and clinically meaningful benefit in functional outcomes for people with ALS taking Albrioza (also known as AMX0035) compared to people taking placebo, either as a stand-alone therapy or when added to existing treatments for ALS,” the company says.

The approval follows FDA’s extension of its review of Abrioza’s NDA earlier this month to evaluate additional analyses of data from the company’s clinical studies. The submission of this information was considered a major amendment to the NDA. The new user fee review action target date is 9/29.

AMX0035 is described as a proprietary oral fixed-dose combination that is designed to reduce the unfolded protein response (UPR), preventing cell death resulting from the UPR and reduced cell death through apoptosis, the company says. It is also intended to target the endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.

Conditions for approval outlined by Health Canada include “the provision of data from the ongoing Phase 3 PHOENIX trial,” the company says. “Other conditions include additional planned or ongoing studies. Amylyx anticipates topline results from the PHOENIX trial in 2024.”

The therapy’s approval was based on data from CENTAUR, a multicenter Phase 2 clinical trial in 137 participants with ALS encompassing a six-month randomized placebo-controlled phase and an open-label long-term follow-up phase…,” the company says. Data demonstrated that Albrioza-treated patients scored, on average, 2.32 points higher on the Amyotrophic Lateral Sclerosis Functional Rating Scale over 24 weeks compared with patients treated with a placebo, 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