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Only one patient has been treated commercially since the drug was
first approved in Europe nearly five years ago, a spokeswoman for
Dutch-based UniQure said on Thursday.
UniQure went into the record books when its gene therapy Glybera was
approved by European regulators for an ultra-rare blood disorder in
2012, and the drug was finally launched two years later with a price
tag of around $1 million per treatment.
But the number of patients eligible for treatment was always tiny
and, with no sign of demand improving, the company said it had
decided not to renew Glybera's five-year European marketing
authorisation, which is due to expire on Oct. 25.
"Glybera's usage has been extremely limited and we do not envision
patient demand increasing materially in the years ahead," said
UniQure Chief Executive Matthew Kapusta.
The group, which had already decided not to pursue a U.S. approval
for the drug, said the decision was not related to any safety
concerns.
Glybera is given as a series of injections to fight lipoprotein
lipase deficiency (LPLD), a disabling condition that clogs the blood
with fat. The drug is sold in Europe on UniQure's behalf by Italian
drugmaker Chiesi Farmaceutici.
The commercial flop is a reminder of the economic challenges facing
the emerging field of gene therapy, which seeks to cure rare genetic
diseases by offering a one-time fix of a faulty DNA but inevitably
comes at a very high price.
However, the setback is unlikely to derail rising investor interest
in gene therapy, which has been triggered recently by a number of
advances in treating a range of genetic diseases, most of which
affect far more patients than LPLD.
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Industry analysts said the decision to pull the plug on Glybera
would make little difference to UniQure's financial outlook. In
fact, the move will save some $2 million in annual costs and help
UniQure focus on other gene medicines.
The Nasdaq-listed firm has high hopes for its next wave of gene
therapies against haemophilia, Huntington's disease and congestive
heart failure, where it has a collaboration with Bristol-Myers
Squibb.
UniQure, whose shares slipped 2 percent in early trading, said it
would continue to make Glybera available to Chiesi to treat any
patients approved for therapy in Europe before Oct. 25.
Scientists have been working on gene therapies for more than a
quarter of a century but it is only recently that the approach has
started to become a commercial reality, although the U.S. Food and
Drug Administration has yet to approve any.
(Editing by Adrian Croft)
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