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[tahirkhan]

bluebird bio visited the CAF office last week to discuss the development of LentiGlobin gene therapy for the treatment of transfusion-dependent beta thalassemia. Here are a few of their updates:

(1) bluebird bio recently filed a Marketing Authorization Application with the European Medicines Agency (EMA) to determine whether gene therapy might be approved for use in the European Union for the treatment of transfusion-dependent beta thalassemia. The length of time this review process may take is up to the EMA, but bluebird expects to hear an answer from them sometime in 2019.

(2) bluebird bio has not yet filed a New Drug Application (NDA) for LentiGlobin with the U.S. FDA. However, they are continuing to engage with the FDA. To date, the FDA has granted LentiGlobin both Orphan Drug status and Breakthrough Therapy designation for the treatment of transfusion-dependent thalassemia.

(3) bluebird bio is actively engaging with health technology assessment (HTA) bodies, payers, and other authorities in the U.S. and Europe to discuss models that recognize the value of gene therapy treatments and how to align payment with value over time. Their goal is to potentially create new pricing and reimbursement models that responsibly reflect the value that a one-time transformational treatment brings to patients, families, the healthcare system and society.

(4) bluebird bio will be providing an update on the latest findings from their clinical trials at the upcoming American Society of Hematology (ASH) meeting in early December.

[sanjuna]

I am from india, I like to know more about gene therapy. Is this treatment will be available in India. if yes like to know some more details. is this treatment like BMT? risk on life. and how much it will cost. my son is 4 yrs old now, we got matching from out side donor but afraid to due to life risk, since some doctor said , in india success of BMT from outside donor is 50-50.

[Andy Battaglia]

Gene therapy uses a patient's own blood to insert good beta globin genes into the patient, where they can multiply and produce good red blood cells. The preparation is similar to that for a BMT, using chemo, and has all the same side effects, although this may change with new preparations being developed. There is no risk of graft vs host disease in gene therapy, as no foreign cells are introduced, so it is not the same as BMT afterwards. The cost looks like it will be very high, but once it gets to India, I imagine that will drop quite a bit. I have no idea about how long it will take, as it is not yet approved anywhere.