You can file this story in the Not Surprised folder.
Austin-based XBiotech’s application to start selling its lead cancer therapy has been spurned by European regulators.
That couldn’t have been completely unexpected as their lead drug — Xilonix — had offered up some dubious data. Investigators reported that the Phase III was marred by a mixup in treating the placebo and drug arms, a number of dropouts and improper patient evaluations. Investigators then used a surrogate endpoint on conserving patients’ health status as a measure of Phase III success.
A month ago, a trend vote by the EMA’s Committee for Medicinal Products for Human Use flagged the members’ opinion that the data just wasn’t there for an approval. The formal CHMP opinion didn’t change. And its opinion was brutal.
The CHMP had a number of concerns. First, the committee noted that the study did not show clear improvements in either lean body mass or quality of life. Secondly, there was an increased risk of infection in patients taking the medicine, which was not considered acceptable in vulnerable patients who will be receiving palliative care. Lastly, there were inadequate controls of the manufacturing process to ensure the medicine would have the same quality as the product used in clinical trials.
Therefore, the CHMP was of the opinion that the benefits of this medicine did not outweigh its risks and recommended that it be refused marketing authorisation.
XBiotech $XBIT, though, indignantly insists that the EMA is off base and wants a do-over. The biotech noted the rejection in an SEC filing Thursday, saying:
The Company plans to promptly notify the EMA of its request for a re-examination and will have 60 days to submit its grounds for appeal. The Company firmly believes it has a strong position for re-examination.
XBiotech had burned through about $194 million by the end of Q1. Its shares are down a little more than 10% this morning.
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