Insmed’s Amikacin Moves Closer To US Market In Refractory Lung Disease
Executive Summary
FDA panel's recommendation that liposome inhalation suspension be labeled only for refractory nontuberculous mycobacterial lung disease and not first-line use aligns with analyst expectations; panel cites lack of clinical studies in treatment-naive patients and adverse event profile as reasons for limiting drug to patients with limited or no treatment options.
You may also be interested in...
Rare Diseases: CBER Looks To ‘Lean Into’ Accelerated Approval, Align More With CDER
US FDA biologics center officials spoke about their efforts to increase collaboration and harmonization with the drugs center, and to internally involve more review disciplines in evaluating biomarker evidence, during a Reagan-Udall Foundation meeting that weighed potential use of accelerated approval for neuronopathic mucopolysaccharidoses disorders.
RWE: Non-Interventional Studies Must Be Able To Distinguish A True Treatment Effect, US FDA Says
Agency describes a host of issues sponsors should consider and address before pursuing observational or case-control studies to support regulatory decision-making on drug efficacy or safety; new draft guidance was issued under the FDA’s Real-World Evidence program.
Eye On ODAC: Former Members, FDA’s Pazdur Talk Pre-Meeting Mindsets, Impact Of Sponsor’s Experts
Oncologic Drugs Advisory Committee reps and the Oncology Center of Excellence director go behind the scenes to discuss FDA interactions with panelists, how opinions can change during a meeting, and why the voting question is important.