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A first for Bluebird and Europe as gene therapy Zynteglo approved

Will product be priced over $1m?


Bluebird Bio is celebrating its first ever drug approval, with the European Commission yesterday granting conditional marketing authorisation for its groundbreaking gene therapy Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene).

The one-time gene therapy has been approved for patients 12 years and older with transfusion-dependent β-thalassaemia (TDT), and has been shown in a series of small studies to free a majority of patients from the need to have these regular blood transfusions.

This first conditional approval is in the moderately severe TDT form of the disease (and not those with the more acute β0/β0 genotype) for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

The approval is not only special for Boston, Mass-based Bluebird Bio, but also for Europe, as the company chose to file its gene therapy with the European Medicines Agency first, rather than the FDA.

This was thanks to the EMA’s PRIME designation, a fast-track appraisal system particularly focused on cutting edge therapies from smaller-to-medium sized pharma and biotech firms.

Similar to other cell and gene therapies, much of Bluebird’s planning to date has been in setting up the gene therapy manufacturing and partnerships with hospitals for the necessary collection of cells from patients and subsequent readministration of the gene therapy back into patients.

The other major preparation has been in preparing Europe’s healthcare systems for the high cost of the treatment. The company hasn’t yet disclosed its price, but analysts expect it to be anywhere between €450,000 and €1 million ($500,000 - $1.12m)

That’s because Bluebird believes it can offer a compelling cost-effectiveness case for its treatment in beta thalassemia and sickle cell cases versus conventional stem cell treatment.

Conventional stem cell transplants cost anywhere between $500,000 and $1m, while also carrying the risk of having to treat adverse events such as graft versus host disease.

The company may well feel emboldened by Novartis, which in late May gained FDA approval for its SMA gene therapy treatment Zolgensma, and unveiled a world-recording breaking $2.12m price tag.

There are great similarities too in the pricing plans: Novartis and Bluebird have both proposed a system of spreading payments across five instalments over five years.

Bluebird’s plans involves all but the first payment being paid based on outcomes, in this case being transfusion free – a gamble based on small trials of little more than a dozen patients – and one that will now be tested out across European markets, once the continent’s payers agree deals.

“EC authorisation of Zynteglo brings a new treatment option, in the form of the first one-time gene therapy for a subset of TDT patients one step closer to being available to patients across Europe,” commented Andrew Obenshain, Bluebird Bio’s European general manager.

He thanked the TDT community, patients and clinical investigators in helping the company achieve the approval and added: “We will now be prioritising reimbursement, ensuring that we work with national payer agencies in each country to enable access to this treatment across Europe.”

As Obenshain explained to Pharma Market Europe earlier this year, the company will be prioritising Germany, Italy, France and the UK as its initial launch markets, as 90% of Europe’s beta thalassemia patients live in these four count

The approval comes just days ahead of a number of important phase 3 read outs for Zynteglo at the European Hematology Association (EHA) congress in Amsterdam. 14 June sees results from several trials, including the ongoing Phase 3 Northstar-3 (HGB-212) study in the most difficult to treat sub-group of patients who have β00 genotype or an IVS-I-110 mutation.

Analysts says peak sales forecasts for Zynteglo in TDT could exceed $800m, but this will depend on whether data shows it can provide curative treatment in these harder-to- treat patients.

Article by
Andrew McConaghie

4th June 2019

From: Regulatory



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