Pink Sheet's monthly Drug Review Profile looks at Marathon's (now PTC's) Emflaza for Duchenne muscular dystrophy, and finds that before the steroid's price ignited controversy it received an unremarkable review – unremarkable, that is, for rare and serious illnesses. The US FDA accepted old and flawed trial data because signals of clinical benefit were consistently seen and safety profile was understood.
The US FDA's approval of Marathon Pharmaceuticals LLC's Emflaza (deflazacort) for Duchenne muscular dystrophy quickly set off a controversy about orphan drug pricing and value, but agency review documents show that the decision about the drug's efficacy and safety was anything but controversial.
"The benefits of deflazacort plainly outweigh its risks," Office of Drug Evaluation I Deputy Director Robert Temple concluded in his...
The Pink Sheet Drug Review Profile explores the US FDA’s approval of vadadustat to treat anemia in chronic kidney disease patients on dialysis. A complete response letter cited the risk of drug-induced liver injury, but postmarketing data from Japan reassured reviewers.
The Pink Sheet’s Drug Review Profile looks at the timeline behind the development and US FDA approval of Akebia’s Vafseo, a treatment for anemia in chronic kidney disease patients on dialysis.
Manufacturers likely will have to determine when to provide a 340B discount on a drug assigned a Medicare negotiated price under CMS guidance. PhRMA anticipates the job will not be easy.
<p>Executive Summary</p> <p>Zanidatamab, Jazz and BeiGene’s investigational treatment for biliary tract cancer, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.</p>
<p>Executive Summary</p> This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.
<p>Executive Summary</p> <p>A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.</p>
