2019-11-11_Bloomberg_Businessweek

◼ BUSINESS Bloomberg Businessweek November 11, 2019

19

loftypricetag—€1.58million($1.8million)per
person—which puts into sharp focus an increas-
ingly common dilemma.
Dozens of gene therapies for a range of devas-
tating illnesses are on their way. These single-dose
drugs, tailored to each patient, can potentially
deliver a lifetime of benefits. But that’s reflected in
their prices, which are likely to increase pressure
on already stretched budgets. To make it easier for
government payers to digest Zynteglo, Bluebird
planstospreadoutthecostoverfiveyears,with
paymentscontingentonitssuccess.
Asa one-timetherapy,Zynteglocouldsavegov-
ernmentsmoneyinthelongrunbycuttingtheneed
forexpensiveongoingcare.Treatingonebetathal-
assemiapatienttodaycancostasmuchas€60,000
a year,saysAurelioMaggio,a blood-diseasespecial-
istatthePalermocenter.That’s€3millionoverfive
decades.Withmultiplewonderdrugsforothercon-
ditionssettoreachthemarketsoon,theupfront
billcouldtakea heavytollonItaly’sfinances.The
pricetagforthetherapyis twicethe$900,000that
SVBLeerinkanalystManiForooharexpected.Given
thelargenumberofpatientsinthecountrywith
theailment,“thestakesaremuchhigher,”hesays.
Regulatorspredicttherecouldbeasmany
as 20 cellandgenetherapyapprovalsannually
by2025.NovartisAG’sZolgensma, whichwas
approvedintheU.S.inMayfora childhoodillness
calledspinal muscular atrophy, is priced at $2.1 mil-
lion. U.K.-based Orchard Therapeutics Plc is also
developing a treatment for beta thalassemia, which
afflicts almost 300,000 people globally. Zynteglo
could gain approval in the U.S. next year and reach
$1 billion in annual sales in 2025. “We’re benefiting
from an explosion of innovation and have crossed
a lot of the key scientific hurdles,” Foroohar says.
“Getting paid for that requires slower-moving enti-
ties such as governments, payers, and regulators
to catch up.”
In Italy, adoption of Zynteglo could have a sig-
nificant impact. More than twice as many of its cit-
izens suffer from beta thalassemia as in France,
Germany, the U.K., and the U.S. combined,
Bluebird estimates, and many live on the islands of
Sicily and Sardinia. But fewer than a third of Italian
patients may be immediately eligible, because regu-
lators approved it only for those 12 years and older
who don’t have a serious type of the disease that
leaves them unable to make any hemoglobin, the
key protein in the blood. Paying for a limited num-
ber of people initially will make it easier for govern-
ments to afford, says Andrew Obenshain, head of
Bluebird’s Europe business. The company intends
to eventually reach a broader pool of patients.

Peoplewithbetathalassemia,whichcanleadto

life-threatening anemia, extreme fatigue, and other

problems, require transfusions as often as every

two weeks. One side effect is excess iron, result-

ing in possible liver and heart damage if untreated.

The standard therapy has improved over the years,

but the prospect of a cure “represents a dream for

the thalassemia population,” says Franco Locatelli,

a doctor at Sapienza University of Rome who was

involved in the Zynteglo trials.

The blood disorder, related to sickle cell disease,

has burdened Sicily for centuries and is common

in Africa, Asia, and other regions where malaria

historically was also prevalent. Scientists say the

gene thrived because it offered protection against

the mosquito-borne illness. Carriers who inherited

only one copy of the flaw were more likely to sur-

vive and pass on the gene to their children, lead-

ing to higher prevalence in successive generations.

Bluebird, based in Cambridge, Mass., spent

decades developing a way to use a modified virus

to transport a functioning version of the gene into

a patient’s cells to restore production of hemoglo-

bin. That work has finally paid off. After refining the

process for manufacturing the therapy, the com-

pany is working with governments to hammer out

coverage based on its novel five-year installment

plan. The drugmaker expects to introduce Zynteglo

in Europe in 2020. Getting the product to patients

in Italy will likely take about a year from the time it

was cleared, Bluebird’s Obenshain says.

In the meantime, patients and family mem-

bers are eager to get the drug. The therapy gives

Valentino Orlandi new hope as his 60th birthday

looms. The Ferrara-based entrepreneur may be

able to spend the rest of his life without the bur-

den of blood transfusions. “We have been waiting

for Zynteglo for over 40 years,” he says. “We are now

in a hurry.” �James Paton and Chiara Albanese, with

LucaCasiraghiandAlbertinaTorsoli

THE BOTTOM LINE A gene therapy from Bluebird Bio will cost

almost $2 million per patient. A growing number of such expensive

cures could put national health services and insurers in a bind.

DATA:COMPILEDBYBLOOMBERG

The High Cost of One-Shot Therapies

Drug

Zolgensma

Zynteglo

Glybera

Luxturna

Strimvelis

Kymriah

Yescarta

Indication

Spinal muscular atrophy

Beta thalassemia

Lipoprotein lipase deficiency

Inherited retinal disease

Immunodeficiency

Lymphoblastic leukemia

Non-Hodgkin lymphoma

Company

Novartis

Bluebird Bio

UniQure

Spark/Roche

Orchard

Novartis

Gilead

Costperdose

$2.1m

1.8m

1.0m

850k

663k

475k

373k