FDA approval of Bluebird Bio’s Zynteglo makes it the first gene therapy in the U.S. for the rare blood disorder beta thalassemia. The biotech company set a $2.8 million price for the one-time treatment, but it also came up with a reimbursement strategy designed to ease the financial risk to payers.
An analyst wrote that it is unlikely bluebird would provide full data from its registration-directed study of the CAR-T bb2121 by year end, which is more likely to come early next year.
The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients.
An analyst wrote that bluebird bio would likely launch Zynteglo first in Germany, followed by other countries, but scaled back sales forecasts due to the therapy's high price tag of 1.575 million euros.
On a quarterly basis, companies in areas like cell and gene therapy raised less in the second quarter than during the same period last year, but the number of clinical trials underway saw an uptick. European and Israeli firms also saw a boost in funding.
The biotech company said it would price Zynteglo for beta-thalassemia at about $1.8 million for the European market. Data for the therapy in that disease and also sickle cell disease are being presented at EHA.