bluebird's Zynteglo becomes world's second most expensive therapy at €1.575 million

Country : France, Germany, Italy, U.S., UK

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LONDON, 14 June (APM) - bluebird bio's beta thalassemia gene therapy Zynteglo (lentiglobin) has been priced at €1.575 million spread out over five years, the company said on Friday.
bluebird, which is in talks with national authorities and payers in the EU, has proposed a plan whereby €315,000 is paid upfront and the remaining in annual payments over five years only if the treatment continues to be effective, the company told Thomson Reuters.
The price makes Zynteglo the second most expensive in terms of list price, behind another gene therapy - Novartis' Zolgensma (onasemnogene abeparvovec), approved only in the U.S., priced at $2.1 million.
Zynteglo was approved in the EU for patients with the rare genetic blood disorder requiring regular blood transfusions and who have no matching donor for stem cell transplant on a conditional basis earlier this month (APMHE 63203). The company told APM earlier this week that it would launch first in Germany, following by France, the UK and Italy (APMHE 63273).
The company expects Zynteglo to be approved in beta thalassemia in the U.S. in 2020 (APMHE 61386). bluebird is also testing the therapy for sickle cell disease (APMHE (APMHE 61993).
Wall Street analysts expect its beta thalassemia sales will reach around $828 million by 2024, Reuters cites Refinitiv data as saying.

Gene therapy pricing

The price of Zynteglo is higher than analyst estimates of around €900,000 for the European market.
It places the therapy as one of the priciest treatments in the world behind Zolgensma, and just ahead of what is now the third most expensive, another gene therapy, Spark's Luxturna (voretigene neparvovec), which is priced at $850,000 (APMHE 56276).
The three therapies are all part of a new wave of one-time gene therapies with purported long-term - even curative - effects which are coming to market with high price tags.
The "intrinsic value" of the one-time infusion is around $2.1 million per patient, given the treatment means patients should be able to avoid costly blood transfusions and improve their quality of life, bluebird said in January (APMHE 61386).
"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 Nick Leschly told Reuters in a phone interview.
His comments are in line with those of Dave Lennon, president of AveXis - the Novartis company behind Zolgensma. Earlier this week, he told APM that health systems have the capacity to cope with high cost, one-time, potentially curative therapies, and that they are already paying high costs to treat the diseases they target (APMHE 63309).
Like Novartis, bluebird is offering risk sharing for the treatment to handle long-term uncertainty.
However, Reuters noted that the whether these new high prices therapies can succeed in the European market - where countries have stringent price controls - remains to be seen.
It cited the first two gene therapies approved in Europe, UniQure's Glybera (alipogene tiparvovec) and GlaxoSmithKline's Strimvelis, and remarked that both were eventually abandoned by their companies.
"But drugmakers are forging ahead with promising new gene therapies that will continue to test pricing limits," according to Reuters.



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