Pink Sheet - News And Expert Analysis On Pharma Policy And Regulation since 1939

Roche’s filing for Elevidys in the EU followed applications in Middle Eastern countries because those markets accept applications based on approval by the US Food and Drugs Administration.

An ongoing proof-of-concept study by the European Medicines Agency testing the use of US-style standards for submitting non-clinical raw data in marketing applications has received positive feedback from both industry and regulatory assessors so far.

Sponsors should investigate signals of possible fraud in a manner that does not raise alarms or else evidence might disappear, UK and US regulators said.

The US FDA will not be in its usual position as the first regulator to approve novel drugs, with only one of the six novel candidates on the July user fee calendar seeking its first approval worldwide in the US

The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.

Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.

The Pink Sheet drug review profile investigates the long and rigorous process behind the FDA’s recent relaxation of the cardiovascular safety warning for testosterone products.

The Council of the EU has not taken forward a proposal from the European Parliament that would require companies to disclose the transfers of value they make to health care professionals and health care organizations – lawyers weigh in on the diverging proposals.

Lawyers explain how changes to rules around drug advertising and promotional activities under the EU pharma reform package could impact companies operating in the bloc, with one legal expert advising companies to “monitor developments closely.”

John Crowley discussed how he prioritizes industry’s many competing challenges in a Pink Sheet interview at the BIO International Convention.

Too many “uninformative” drug trials fail to justify the excessively high prices of many medicines, while there is too much evidentiary uncertainty in European pricing and reimbursement systems, warned Anja Schiel from Norway’s NOMA.