Tex Mex schreef op 21 januari 2014 21:47:
[...]
Die FDA uitslag laat nog even op zich wachten, tot
2017, zo lijkt het. Tenminste, indien je het over de eventuele market approval van Glybera in de USA hebt (
BLA, Biological License Application). De huidige resultaten overtuigen de FDA niet. De post approval trial die de EMA wenst moet zo worden opgezet dat ook voor de FDA
meaningfulness voldoende kan worden aangetoond.
Onderstaande tekst is afkomstig uit de SEC-filing:
United States Planned Program for GlyberaWe met with the FDA in August and December 2013 to discuss the regulatory pathway in the United States for Glybera. In contrast with the European Union, the United States does not have a process to approve marketing of a drug under exceptional circumstances. In our meetings, the FDA advised that it would require data in addition to what we had submitted to obtain marketing approval for Glybera in the European Union. The FDA advised that severe hypertriglyceridemia is currently considered a hallmark of LPLD, and agreed that changes in chylomicron metabolism following a meal is a relevant marker of biological activity of Glybera.
However, the FDA also advised that changes in chylomicron metabolism following a meal alone would not be adequate for obtaining marketing approval in the United States at this stage, since it is
not yet sufficiently understood how this biological effect translates into clinical meaningfulness. The FDA recommended that we identify the clinical manifestations of LPLD for which Glybera might have the best prospects for demonstrating a meaningful impact in designing an adequate and appropriately controlled trial.
We plan discuss the details of the EU post-approval trial and patient registry with the FDA, and if applicable to seek to amend the protocols for the this post-approval trial and patient registry so that they also could serve as a clinical program with a design that addresses the FDA's requirements. We also plan to file an IND with the FDA for Glybera in the first half of 2014 so that we can include United States LPLD patients in the post-approval trial and registry. We believe the patient registry will provide valuable data for the FDA to consider as part of the totality of our United States regulatory submissions. Our current expectation, subject to satisfactory completion of regulatory discussions with the FDA, is to have sufficient data from a further clinical trial of Glybera and the patient registry to file a
BLA for Glybera with the FDA in 2017.