Drugs

FDA Questions Efficacy of Patisiran sNDA

FDA medical reviewers are questioning the efficacy of Alnylam Pharmaceuticals’ patisiran (Onpattro) in treating a larger pool of patients as sought in an sNDA. The filing will be considered on 9/13 by the FDA Cardiovascular and Renal Products Advisory Committee.

In a briefing paper released in advance of the committee meeting, FDA says the sNDA seeks an indication to treat patients with cardiomyopathy induced by transthyretin amyloidosis (ATTR-CM). The paper says that while the APOLLO-B study submitted with the sNDA was well designed and executed and met a key primary endpoint and a key secondary endpoint, with the results being statistically significant, the agency finds that “the effects of patisiran compared to placebo on both endpoints were small, of questionable clinical meaningfulness, and may not be detectable by patients.”

Committee members were asked to respond to these points:

whether they agree with reviewers that there are no fundamental problems with the conduct of the APOLLO-B trial;
whether they agree with reviewers that the patisiran safety profile has been adequately characterized;
whether the results of the APOLLO-B trial show evidence of a clinically meaningful treatment effect of patisiran in patients with ATTR-CM; and
whether there is a population of patients for which the APOLLO-B results support using the drug as monotherapy or in combination with tafamidis (Pfizer’s Vyndamax) to treat ATTR-CM.

Onpattro was approved by FDA in 2018 to treat hereditary ATTR amyloidosis polyneuropathy.

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