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AN EXPERIMENTAL GENE THERAPY COULD BECOME THE MOST EXPENSIVE DRUG EVER.
BIOMARIN'S CEO SAID A PRICE AS HIGH AS $3 MILLION WOULD SAVE THE HEALTHCARE
SYSTEM MONEY.

Andrew Dunn
2022-01-20T13:00:00Z
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BioMarin Pharmaceutical CEO Jean-Jacques Bienaimé. BioMarin Pharmaceutical

THIS STORY IS AVAILABLE EXCLUSIVELY TO INSIDER SUBSCRIBERS. BECOME AN INSIDER
AND START READING NOW.

 * A biotech is planning to price its gene therapy as high as $3 million, its
   CEO told Insider. 
 * The one-time treatment has shown it can help patients with the blood-loss
   disorder hemophilia.
 * BioMarin CEO Jean-Jacques Bienaimé said the treatment would lower healthcare
   costs.

A California biotech is planning to price an experimental gene therapy for a
bleeding disorder as high as $3 million, even though the benefit appears to wane
after several years.

BioMarin Pharmaceutical CEO Jean-Jacques Bienaimé first floated a $2 million to
$3 million price range two years ago in an interview with The Wall Street
Journal. Even as new results indicated the one-time treatment wouldn't cure
hemophilia, the CEO of the $16 billion company said that price range was still
justified.

That amount "looks like a lot of money, but at the same time, in the context of
what it's costing today to treat these patients, it's not a lot of money,"
Bienaimé told Insider.

The treatment will be marketed under the brand name Roctavian. The one-time
infusion delivers a working copy of a gene that produces a crucial protein to
help stop bleeding. Hemophilia patients lack this clotting protein. This makes
injuries and bleeding serious — sometimes life-threatening — events.

The Food and Drug Administration initially rejected BioMarin's application for
approval in August 2020, as it wanted to see more data on how long the treatment
worked and how safe it was. 

With those results now in hand, BioMarin plans to resubmit its application to
the FDA in the second quarter of 2022. Analysts expect US approval to come in
late 2022 or early 2023, with some forecasting more than $1 billion in peak
annual sales from the drug.

If approved, BioMarin's treatment would likely be the most expensive drug. The
Swiss pharma giant Novartis' gene therapy for spinal muscular atrophy,
Zolgensma, is currently the most expensive drug at $2.1 million. 


BioMarin Pharmaceutical is developing a gene therapy for hemophilia, an
inherited bleeding disorder. BioMarin Pharmaceutical


BIOMARIN CEO SAYS A 7-FIGURE PRICE TAG WOULD SAVE THE HEALTHCARE SYSTEM MONEY

Given the high cost of treating hemophilia patients, the biotech CEO said the
one-time treatment would save health insurers money.

Hemophilia patients typically need regular infusions of clotting factor, a
protein that helps stop bleeding. A newer option is Hemlibra, a drug developed
by Roche that was approved in 2017. It can prevent or reduce bleeding events. 
These treatments can be wildly expensive — Hemlibra is priced at about $450,000
a year, and factor infusions range from $300,000 to $500,000 a year for adults.

Bienaimé said health-insurance companies were largely on board with BioMarin's
pricing plan. 

"I don't think any of them were panicking at a $2 million to $3 million price
tag. They weren't," he said. "They know how much those patients cost them right
now."

Dr. Aaron Kesselheim, a professor at Harvard Medical School, said the key
consideration for pricing Roctavian was how well it worked and the durability of
that effect. In an email to Insider, he said the high costs for current
hemophilia treatments didn't justify a high price for a future gene-therapy
option. Instead, Roctavian's price should mainly come from its benefit,
Kesselheim said.

The high price will create challenges for the US healthcare system, Kollet
Koulianos, vice president of payer relations at the nonprofit National
Hemophilia Foundation, said. People often change health insurers, which could
leave some health plans paying for the upfront cost and not realizing the
long-term savings from the therapy, she said in an email.  This may be untenable
for smaller health plans, she added.  

"There is no doubt that for some patients living with hemophilia, gene therapy
has the potential to offer life changing hope, but for the health plans who will
be expected to assume the majority of costs, come sleepless nights," Koulianos
said. 

After this story published, a BioMarin spokesperson highlighted a study paid for
by the biotech that found the average patient with severe hemophilia stayed on a
commercial plan for about five years.  


A ONE-TIME TREATMENT MAY NOT LAST A LIFETIME

The promise of gene therapy is it that can be a one-time treatment that fixes
the root cause of certain genetic diseases. Dr. Francis Collins, a previous
director of the National Institutes of Health, said in a November blog post that
gene therapy "represents a possible cure for hemophilia."

It's unclear how long the therapy will work. BioMarin presented updated data on
January 9 that followed most of its trial participants for at least two years.
The results showed a dramatic benefit through two years, with the average rate
of factor infusions dropping 98% and the typical number of bleeding episodes
requiring treatment falling 85% from baseline levels.

While BioMarin's data is encouraging, the trial also showed the amount of
clotting protein made by treated patients dropping each year. BioMarin's top
scientists aren't sure what's behind the waning benefit. 

"We don't exactly have a smoking gun," Henry Fuchs, BioMarin's president of
worldwide research and development, told Insider.

Bienaimé previously justified a $2 million to $3 million list price by comparing
it to the lifetime cost of hemophilia treatment, which he estimated was $25
million.

Despite the diminishing benefit, the pricing plan is unchanged, and Bienaimé
referenced the cost of four years of standard treatment as the rationale for the
up to $3 million price tag.


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