Recently, Bluebird Bio announced that the FDA has accelerated the approval of the company's gene therapy drug Skysona (elivaldogene autotemce, eli-cel), which is used to treat a rare neurological disease cerebral adrenoleukodystrophy (CALD), and is the world's first approved one-time gene therapy
for the treatment of early CALD.
It is understood that Bluebird Bio set the wholesale acquisition cost of Skysona in the United States at $3 million (equivalent to about RMB 21.
23 million at the current exchange rate, the same below), which has become a very expensive treatment method
in the world.
In fact, in addition to Skysona, Bluebird Bio has another Zynteglo that costs $2.
8 million
a year.
According to the data, Zynteglo, which was approved by the FDA on August 17 this year, is a one-time gene therapy for the treatment of patients with β-thalassemia who require regular blood transfusions, covering adults, adolescents and children of all genotypes, which is also the first gene therapy
approved in the United States to treat the disease.
At present, domestic pharmaceutical companies are also actively developing gene therapies
to treat poverty.
In August this year, Bangyao Bio announced that its clinical trial application for the gene therapy product "BRL-101 Autologous Hematopoietic Stem Progenitor Cell Injection" for transfusion-dependent β-thalassemia was officially approved by
the Drug Evaluation Center of the State Food and Drug Administration on the same day.
It is worth noting that combined with the statistics released by GoodRx, the US prescription drug comparison platform in May this year, in addition to Bluebird Bio's Skysona and Zynteglo, there are currently 3 drugs with annual treatment costs of more than $1 million
.
Among them, the three drugs are Novartis's "Zolgensma", Eiger Biopharma's "Zokinvy", and Y-mAbs' "Danyelza"; The annual cost of treatment was US$2.
125 million, US$1.
074 million and US$1.
012 million
, respectively.
In fact, in recent years, with the domestic pharmaceutical industry setting off a wave of innovation, a number of high-priced drugs have begun to emerge in China
.
For example, the cell therapy product CARVYKTI (Cilta-cel), independently developed by China Legend Biologics, was approved by the US FDA on February 28 for the treatment of patients with
recurrent or refractory multiple myeloma (R/R MM).
According to the data, CARVYKTI is a CAR-T cell immunotherapy with two single-domain antibodies targeting B-cell mature antigen (BCMA), which is administered as a one-time infusion, and the one-time treatment cost is equivalent to about 3.
25 million yuan
.
In addition, the price of 30 tablets of respirinib of Zaiding Pharmaceutical is also as high as 33266 yuan, according to the instructions of the drug, 3 tablets per day, the annual treatment cost of the corresponding patients is also as high as 1.
198 million yuan
.
However, in order to enter medical insurance, since this year, the repatinib of Zaiding Pharmaceutical has reduced prices
in many places.
In the list of price reductions previously released by the Yunnan Medicine Harvesting Center, the price of 30 pieces of repatinib was reduced from 33266 yuan to 13315 yuan, which means that the annual treatment cost will also drop to 479,000 yuan
.
