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Bluebird’s first gene therapy gets European fast track

For once, Europe set for approval and launch ahead of US


Gene therapy company Bluebird Bio is on course to file its first product later this year, and has just been granted an accelerated review by the European Medicines Agency.

The Cambridge, Massachusetts-based firm will file its gene therapy LentiGlobin TDT with the EMA by the end of 2018, a treatment of adolescent and adult patients with transfusion-dependent β-thalassemia (TDT) and a non-β00 genotype,

Pivotal data from two studies in the severe inherited blood condition suggest the gene therapy could transform care of the disease by providing a cure for many patients.

In one recently presented study, seven out of eight patients no longer needed long-term blood transfusions, and continued to show normal levels of haemoglobin.

Unusually, Bluebird plans to file its groundbreaking treatment in Europe ahead of the US. The company says this was in part thanks to LentiGlobin being accepted on to the EMA’s PRIME fast track designation, which allows early discussions about data requirements and trial design in groundbreaking therapies.

Today’s announcement from the EMA means an approval for the gene therapy could come in 2019.

David Davidson

“Transfusion-dependent β-thalassemia is a severe genetic disease that requires a lifetime of chronic blood transfusions for survival, and while these transfusions are life-saving, they are also associated with serious medical complications such as organ failure from iron overload,” said David Davidson (pictured) M.D., chief medical officer at Bluebird. “Receiving accelerated assessment for LentiGlobin helps support our goal of delivering the first gene therapy to patients with TDT. We look forward to working in collaboration with the regulatory authorities on this potentially transformative treatment option.”

The company has now confirmed its intention to file LentiGlobin in TDT with the EMA in 2018.  The agency’s accelerated assessments can cut the active review time of a marketing authorisation from 210 days to 150 days once it has been validated by the EMA.

An accelerated assessment is granted to products deemed by the CHMP to be of major interest for public health and represent therapeutic innovation.

The therapy also has Breakthrough Therapy Designation status with the FDA, and US filing is expected to follow close behind Europe.

Analysts William Blair forecast that LentiGlobin could eventually hit global peak revenues of $800m in TDT, but this would depend on its efficacy in the harder to treat β00 genotype patients, who have more severe thalassemia.

The analysts predict the drug will be priced at around $550,000 (€472,000) per treatment, a level which compares favourably with cost of conventional stem cell therapy of $500,000 to $1m.

Bluebird’s products are part of a wave of new cell and gene therapies reaching the European market: Novartis’ Kymriah and Gilead/Kite’s Yescarta CAR-T therapies recently gained CHMP recommendation, while Spark Therapeutics’ gene therapy Luxturna looks set to gain CHMP recommendation by the end of 2018.

Read Pharmaceutical Market Europe’s in-depth look at Bluebird, including an exclusive interview with its CEO Nick Leschly.

Bluebird Bio: on the cusp of a gene therapy revolution (PME July/August Digital Edition)

Article by
Andrew McConaghie

27th July 2018

From: Regulatory



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