Drugs

Don’t License Mesoblast’s Ryoncil: Petition

An unnamed client represented by Lassman Law & Policy is asking FDA not to issue a BLA to Mesoblast for its Ryoncil (remestemcel-L) (ex-vivo culture-expanded adult human mesenchymal stromal cells suspension for intravenous infusion) to treat pediatric patients with steroid-refractory acute graft-versus-host disease (aGVHD). The petition expresses concern that the data submitted in the BLA to establish effectiveness fails to meet the rigorous standards necessary to qualify as “substantial evidence” of effectiveness under the Public Health Service Act and FDA policies.

It asks FDA to:

refuse to issue a license to Ryoncil until the sponsor provides “substantial evidence” of effectiveness that consists of, at a minimum, at least one successful pivotal clinical trial that is prospective, randomized, blinded, and uses an appropriate concurrent control; and
provide a copy of the petition to members of any advisory committee scheduled to discuss the BLA.

Lassman said it “believes that remestemcel-L’s ill-defined mechanism of action in combination with a pair of prior, failed, Phase 3 trials and a third trial featuring a confounded and uncontrolled historical control arm does not constitute ‘substantial evidence’ of Ryoncil’s effectiveness. It is particularly important for FDA to be confident in remestemcel-L’s efficacy given alternative, approved therapies on the market and potential development-stage therapies that could be delayed or passed over altogether, possibly causing harm to patients who have potentially life-threatening aGVHD.”

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