Η Υπηρεσία Τροφίμων και Φαρμάκων των ΗΠΑ (Food and Drug Administration ή FDA) ενέκρινε την Τετάρτη τη γονιδιακή θεραπεία του bluebird bio για ασθενείς με μια σπάνια διαταραχή που απαιτεί τακτικές μεταγγίσεις αίματος.
The pharmaceutical company priced the new treatment at a record price of $2,8 million.
The approval lifted the company's shares by 8%. It is a treatment for beta-thalassemia, which causes a lack of oxygen in the body and often leads to liver and heart problems.
The sickest patients, estimated to be as many as 1.500 in the United States alone, require blood transfusions every two to five weeks. The drug, to be called Zynteglo, is expected to face pushback from insurers because of its very high price.
Bluebird is pitching Zynteglo as a one-time treatment that can eliminate the need for transfusions, resulting in savings for patients in the long run.
The average cost of all the transfusions required could be as high as $6,4 million, Chief Operating Officer Tom Klima told Reuters before the approval.
"We believe the prices we are looking at still provide significant value to patients."
Bluebird is currently in negotiations with insurers for a lump sum payment option.
"Potentially, up to 80% of that payment will be refunded if a patient continues to need transfusions, and insurers are very excited about that," Klima said.
The FDA warned of a possible risk of blood cancer with the treatment, but noted that studies have shown no such cases.
"Bluebird expects to begin the treatment process for patients in the fourth quarter," Reuters reports.
"However, no treatment revenue is expected in 2022, as the treatment cycle will take an average of 70-90 days from initial cell collection to final transfusion."