Bluebird prices gene therapy at 1.575 mln euros over five years

Orchard Therapeutics

14/06/2019 13:00

By Deena Beasley and Tamara Mathias
    June 14 (Reuters) - Bluebird bio Inc  BLUE.O  on Friday set
a price for its gene therapy, Zynteglo, at 1.575 million euros
($1.78 million) over five years, after winning conditional
approval in Europe this month to treat a rare genetic blood
disorder.
    The company proposed a five-year installment plan, with
315,000 euros paid up front and additional annual payments due
only if the treatment continues to be effective.
    Zynteglo was approved in Europe for patients age 12 and
older with beta thalassemia who require regular blood
transfusions to manage their disease and have no matching donor
for a stem cell transplant.  urn:newsml:reuters.com:*:nL4N23A3NC
    Bluebird, which is also testing Zynteglo for sickle cell
disease, said it expects the therapy to be approved for beta
thalassemia in the United States in 2020. Wall Street analysts
forecast Zynteglo beta thalassemia sales of about $828 million
by 2024, according to data from Refinitiv.
    Pharmaceutical companies have been investing heavily in
potentially life-changing gene therapies over the past few
years, but they come with hefty price tags. As a result, some
are experimenting with so-called value-based pricing, which in
some cases calls for refunds if a therapy fails to deliver on
its promise. 
    Bluebird said it believes the "intrinsic value" of its
one-time infusion is about $2.1 million per patient, and the
price reflects the ability to avoid costly blood transfusions,
improve quality of life and potentially cure patients.
    Patients with beta thalassemia typically receive lifelong
blood transfusions every few weeks.
    Bluebird said it aims to price Zynteglo in developed
nations, including the United States, within a "reasonably
close" range. The company said patient access talks are underway
with health authorities in Germany, Italy, France, and Britain.
    "The one-time potentially curative nature of what we have on
our hands here sort of warrants this type of a (pricing) model
in a more aggressive way," bluebird Chief Executive Officer Nick
Leschly told Reuters in a phone interview.
    Gene therapies use engineered viruses to carry healthy
genetic material into a person’s cells to replace faulty or
mutated genes that cause a disease or condition.
    Whether these high priced new therapies can succeed in
Europe, where countries have stringent price controls, remains
to be seen.
    The first two gene therapies were approved in Europe -
UniQure NV's  QURE.O  Glybera in 2012 for a very rare blood
disorder and GlaxoSmithKline's  GSK.L  Strimvelis in 2016 for
"bubble boy" disease - and both were eventually abandoned by the
companies.
    But drugmakers are forging ahead with promising new gene
therapies that will continue to test pricing limits.
    In May, Swiss drugmaker Novartis  NOVN.S  won U.S. approval
for its gene therapy for spinal muscular atrophy and priced it
at $2.125 million, making it the world's most expensive drug.
Novartis defended the price by saying that a one-time treatment
was more valuable than expensive long-term treatments.
 urn:newsml:reuters.com:*:nL2N2300QT
    Spark Therapeutics Inc  ONCE.O , which is being acquired by
Roche Holding AG  ROG.S , won U.S. approval for its gene therapy
to treat a rare form of blindness in 2017, pricing it at
$850,000.
 ($1 = 0.8869 euros)

 (Reporting by Deena Beasley, Tamara Mathias and Aakash
Jagadeesh Babu; editing by Bill Berkrot)
 ((Tamara.Mathias@thomsonreuters.com; within U.S. +1 646 223
8780, outside U.S. +91 806749 1208; Reuters Messaging:
tamara.mathias.thomsonreuters.com@reuters.net))