[Ip-health] Richard Knox in NPR: With A Life-Saving Medicine In Short Supply, Patients Want Patent Broken

James Love james.love at keionline.org
Thu Aug 5 03:15:39 PDT 2010

Richard Knox in NPR: With A Life-Saving Medicine In Short Supply,
Patients Want Patent Broken


With A Life-Saving Medicine In Short Supply, Patients Want Patent Broken
August 4, 2010, Richard  Knox

Listen to the Story Morning Edition [3 min 44 sec]

The initial version of this post misstated the relationship of two of
the petitioners. Anita Bova is Anita Hochendoner's daughter -- not her

The Massachusetts biotech company Genzyme showed everybody else the way
to make money by developing ultra-high-priced drugs for people with rare
diseases. But lately it’s discovered the downside of that strategy.

And boy, is it a big one.

In the year since Genzyme discovered viral contamination in equipment
used to  produce the $300,000-a-year drug Fabrazyme, and then foreign
particles like bits of stainless steel in vials of the drug, the company
has been fined $175 million by the Food and Drug Administration and
become a takeover target.

Fabrazyme replaces a missing enzyme in patients who have a genetic
disease that can lead to an early death. But Genzyme's manufacturing
woes mean the medicine is in short supply — less than a third of the
needed amount is available.

Now patients who depend on Fabrazyme are asking Health and Human
Services Secretary Kathleen Sebelius to exercise "march-in rights" under
a 1980 law to abrogate Genzyme’s patent on the drug, allowing another
company to fill the demand.

Such petitions have been filed several times – never successfully. But
this is the first time patients have made the case, alleging that a
patent-holder isn’t satisfying their needs.

"They have not taken proper care of those patients that have these rare
diseases," says Joseph Carik, a 53-year-old Fabrazyme patient in Las
Vegas who filed the petition yesterday along with his cousin Anita
Hochendoner and her daughter Anita Bova.

All three have Fabry disease, a rare genetic disorder that interferes
with fat metabolism. And, all told, 22 members of Carik's family have
Fabry disease.

Fats build up in virtually every tissue of the body, leading to
premature death from heart and kidney failure. Along the way, though,
patients suffer pain in the arms and legs, severe intestinal problems,
profound fatigue and other maladies.

There may be as many as 50,000 people with the disorder, but only about
1,500 depend on Fabrazyme, which was approved in 2001. The rest are
presumably undiagnosed or misdiagnosed.

Carik and other Fabrazyme patients are currently able to get only 30
percent of the needed dose. That’s because Fabrazyme production is only
30 percent of normal. Genzyme says it hopes to begin increasing supplies
by late this year, but it’s making no promises about when stocks will be
in full supply.

"We sympathize with the challenges patients are facing," Genzyme said in
a statement yesterday, "and we share a common goal of restoring reliable
supplies of product for all patients."

The company said it's making "excellent progress" on a new manufacturing
plant "which is on track for approval next year." Genzyme declined to
make anyone available for an interview.

But a string of broken promises has alienated many patients who were
once loyal to the company and led to this week's petition.

"Since the shortage in 2009, three to six months turned into six to
nine, nine to 12, and now we’re (talking about) late 2011," Carik says.
"I would like to have a backup plan, for myself, for my famly, and for
those in the Fabry community. I do not know of a backup plan"

Patients are especially anguished because one backup plan has evaporated
– while they waited for Genzyme to get past rationing of Fabrazyme.

The British drugmaker Shire had offered to supply its Fabry
enzyme-replacement drug, Replegal, free of charge to U.S. patients under
an emergency-use provision. Shire applied to the FDA for market approval
of Replegal earlier this year.

However, a Shire spokeswoman says only about 100 Fabry patients in this
country have accessed Repligal so far. And this summer – just around the
time Genzyme announced another delay in increasing Fabrazyme supplies –
Shire said it could no longer provide Replegal to new patients.

Allen Black, a Pittsburgh patent attorney who’s representing the Fabry
disease petitioners on a pro-bono basis, says many Fabrazyme patients
were holding back from accepting Shire’s earlier offer.

"They’ve been told by Genzyme that drug was on its way, that the problem
is going to be fixed soon, there’s nothing to worry about," Black
says."They probably wish they had applied (to Shire) for treatment. They
were led to believe they would be OK, essentially."

Carik especially regrets not urging his cousin's daughter Anita Bova to
take Shire up on its offer. She’s only in her late 40s, and since
Fabrazyme has been rationed, she has developed heart failure, Carik

Carik and Allen acknowledge they face an uphill battle in getting HHS,
or the National Institutes of Health, which funded initial studies that
led to Fabrazyme, to exercise their march-in rights. It’s never happened

But even if their petition is granted, it’s not at all clear what
practical effect it would have. No other company is waiting in the wings
to take advantage of an opportunity to make Fabrazyme, and even if there
were, it would take time to gear up to make it and get past various
regulatory hurdles.

And as Genzyme’s experience amply demonstrates, Fabrazyme is not a snap
to make.

Still, the petitioners hope they will bring attention to the need for a
back-up supply for life-saving drugs – either stand-by capacity from
more than one supplier, or inventory requirements to address
manufacturing glitches.

Meanwhile, frustrations are running high in the small community of Fabry
disease patients and caregivers – and anxiety may be mounting among
other sufferers of "orphan" diseases who depend on the biotech industry.

"We’re been promoting awareness of Fabry disease for several years
because we think it’s potentially life-saving to get on therapy," says
Dr. Robert Hopkin of Cincinnati Children’s Hospital Medical Center. "And
now we have patients who want treatment, who qualify for treatment and
there's just no medicine available."
James Love, Director, Knowledge Ecology International
http://www.keionline.org | http://www.twitter.com/jamie_love
Wk: +1.202.332.2670 | US Mobile +1.202.361.3040 | Geneva Mobile +41.76.413.6584

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