[Ip-health] World's most expensive medicine Glybera goes on sale with $1m price tag

Tahir Amin tahir at i-mak.org
Thu Nov 27 04:15:06 PST 2014


The western world’s first gene therapy drug is set to go on sale in Germany
with a €1.1m ($1.4m) price tag, a new record for a medicine to treat a rare

The sky-high cost of Glybera, from Dutch biotech firm UniQure and its
unlisted Italian marketing partner Chiesi, shows how single curative
therapies to fix faulty genes may upend the conventional pharmaceutical
business model.

After a quarter century of experiments and several setbacks, gene therapy
is finally throwing a life-line to patients by inserting corrective genes
into malfunctioning cells – but paying for it poses a challenge.

The new drug fights an ultra-rare genetic disease called lipoprotein lipase
deficiency (LPLD) that clogs the blood with fat. The medicine was approved
in Europe two years ago but its launch was delayed to allow for the
collection of six-year follow-up data on its benefits.

Now Chiesi has filed a pricing dossier with Germany’s federal joint
committee, or G-BA, which will issue an assessment of the drug’s benefits
by the end of April 2015.

The company is seeking a retail price of €53,000 per vial, or €43,870

That equates to €1.11m for an typical LPLD patient, averaging 62.5kg in
clinical trials, who will need 42 injections from 21 vials. This price will
be subject to a standard 7% discount under Germany’s drug pricing system.

Under German rules, the launch price for a new drug is valid for the first
12 months.

A Chiesi spokeswoman confirmed the launch price, in response to inquiries
from Reuters, prompted by information from health insurance sources.

She added that a final figure would be set after the G-BA gives its verdict
and negotiations are held with statutory health insurance funds.

“First commercial treatments are expected in the first half 2015,” she

UniQure, which will get a net royalty of between 23% and 30% on sales, said
EU pricing was a matter for its Italian partner, although the Dutch firm
does plan to discuss Glybera pricing during an investor meeting in New York
on Dec 1.
With only 150 to 200 patients likely to be eligible for Glybera across
Europe, the impact on healthcare budgets will be small, even at a very high
price – but this case will be watched closely as a benchmark for future
gene therapies.

UniQure also has plans to seek approval for Glybera in the United States,
which it hopes to get in 2018.

Although there is already a gene therapy for cancer on the market in China
that drug has not been rolled out to other countries, making Glybera a
first for the western world.

Proponents of the gene-fixing technology insist it stacks up as a
cost-effective treatment, despite the high cost, since it could permanently
cure many patients.

In the case of Glybera, Chiesi said the annualised cost was no more than
that charged for some expensive enzyme replacement therapies used in other
rare diseases, taking into account the drug’s proven benefits of at least
six years.

The drug consists of a harmless modified virus that carries a corrective
gene into the body’s cells.

UniQure is also working on gene treatments for haemophilia and has an
early-stage project in heart failure, which would take gene therapy beyond
the rare disease space.

Assuming trials are successful, analysts expect gene medicines treating
more common conditions to cost less, since manufacturers should be able to
recoup their research and development investment from a larger patient

Rivals in the gene therapy market include privately owned Spark
Therapeutics, which has an eye drug in late-stage clinical tests, and
Bluebird Bio, which is working on drugs for neurological and blood

Bluebird Bio and UniQure both staged successful floats on the Nasdaq market
in the past 18 months, reflecting growing investor interest in the field.

Among major pharmaceutical companies, Bayer struck a gene therapy deal with
Dimension Therapeutics in June, while Novartis recently established a new
cell and gene therapies unit, and Sanofi has a long-standing tie-up with
Oxford BioMedic.

Tahir Amin
Co-Founder and Director of Intellectual Property
Initiative for Medicines, Access & Knowledge (I-MAK)
*Website:* www.i-mak.org
*Email:* tahir at i-mak.org
*Skype: *tahirmamin
*Tel:* +1 917 455 6601/+1 646 884 7418/+44 771 853 9472

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