Alsof de duvel ermee spelt: QURE heft net een form 6-K voor Glybera ingediend,met een nieuwe risk factor. Het is een lang verhaal, maar het komt erop neer dat: On April 8, 2015, we received a copy of an assessment report prepared by the rapporteur designated by the Committee for Advanced Therapy Medicinal Products, or CAT, which is the committee that advises the EMA’s Committee for Human Medicinal Products for Human Use of the EMA, or CHMP, on gene therapies. The CHMP is comprised of 32 members and the report represents only the views of the rapporteur and does not bind the CAT or the CHMP in any way. Based on our final clinical study report including the six-year follow-up data, the assessment report states that the rapporteur considers [b]that Glybera lacks efficacy and therefore that the benefit-risk balance of Glybera is negative. However, the rapporteur recommends that our application for variations be subject to a request for supplemental information before a final recommendation can be made.[/b]
Er is dus een kans dat de toelating van Glybera wordt ingetrokken.....
Meer text hieronder.
The following risk factor on page 14 of uniQure’s 2014 Annual Report on Form 20-F, filed with the Securities and Exchange Commission on April 7, 2014, has been revised to reflect information received on April 8, 2015, with respect to the ongoing post-approval review of our Glybera program by the European Medicines Agency. The revised risk factor reads as follows:
We are subject to potentially costly post-approval obligations, review and other regulatory requirements for Glybera in the European Union, and any of our product candidates for which we obtain marketing approval in the future could be subject to similar requirements, which may restrict or eliminate the commercial success of Glybera or our other product candidates.
Glybera and any of our product candidates for which we obtain marketing approval in the future, as well as the manufacturing process, post-approval studies and measures, labeling, advertising and promotional activities for such products, will be subject to continued requirements of and review by the FDA, EMA and other regulatory authorities.
As part of our marketing approval under exceptional circumstances in the European Union, the EMA has imposed ongoing requirements for a potentially costly post-approval study and market surveillance activities. Specifically, as a condition to approval of Glybera we are required to complete a post-approval clinical trial and implement a disease registry for long-term surveillance of patients, as well as implement risk management procedures, distribute educational materials to healthcare professionals and patients, implement an additional manufacturing process step, comply with certain notification obligations and undergo annual reassessment, any negative outcome of which could potentially lead to a withdrawal of marketing approval for Glybera. The expense and uncertain result of these post-approval requirements may delay, limit or terminate our commercialization plan for Glybera and adversely affect our financial position, particularly in light of the relatively small market for this orphan indication. Non-compliance with EU requirements regarding safety monitoring or pharmacovigilance can also result in significant financial penalties.
In addition, we have submitted several Type II variations to the EMA, which seek to update the summary product of characteristics, SmPC, of Glybera to include additional six-year follow-up and other clinical data. Following our submission of the Type II variations, and a voluntary disclosure of accidental destruction of some historical source data at a site in Canada, the EMA requested a good clinical practices, or GCP, inspection of our Glybera trial program. The Dutch and UK regulatory authorities conducted the inspection on behalf of the EMA in early 2015. The inspectors reported to the EMA on the quality control mechanisms that were in place in our company during data acquisition and processing in 2009 and 2010 with respect to maintaining patient and trial data obtained prior to approval of Glybera, and the integrity of the historical trial data as a whole. We have already implemented corrective quality control actions to rectify the oversight issues identified by the inspection and continue to refine our quality system. We believe that the events in the past do not materially affect the previously reported results of our historical trials. The inspection team also concluded “that the quality of the data and the level of GCP compliance both are acceptable” and that the trial data can be used for the submitted Type II variations.
As part of the ongoing variation procedure, the EMA is currently reviewing the benefit-risk analysis of Glybera in light of the additional six-year follow-up data we provided and the findings of the GCP inspection. On April 8, 2015, we received a copy of an assessment report prepared by the rapporteur designated by the Committee for Advanced Therapy Medicinal Products, or CAT, which is the committee that advises the EMA’s Committee for Human Medicinal Products for Human Use of the EMA, or CHMP, on gene therapies. The CHMP is comprised of 32 members and the report represents only the views of the rapporteur and does not bind the CAT or the CHMP in any way. Based on our final clinical study report including the six-year follow-up data, the assessment report states that the rapporteur considers that Glybera lacks efficacy and therefore that the benefit-risk balance of Glybera is negative. However, the rapporteur recommends that our application for variations be subject to a request for supplemental information before a final recommendation can be made.
The report will be submitted to the CAT for review and consideration at meetings scheduled to be held on April 16-17, 2015, and then to the CHMP for review and consideration at meetings scheduled to be held on April 21-23, 2015. These committees will reach their own conclusions, which may or may not be in line with those of the rapporteur. After the April meetings, we anticipate receiving a final assessment report, which may include requests for additional information as part of the variation procedure and may require a further response from us to support our position.