From 2.8 million to 3 million, the world's most expensive medicine break record is only 30 days!

From 2.8 million to 3 million, the world's most expensive drug records were broken! It took only 30 days!

Source: Medicine Age/Medicine Times Team

Preface

On August 17, 2022, Bluebird Bio, the American Blue Bird, announced that the US FDA approved its gene therapy product ZyNTEGLO to treat patients with β-thalassemia. The product is priced at 2.8 million US dollars, which is more than $ 2.125 million than the Novo Pharmaceutical Gene therapy product ZOLGENSMA. This means that the record of the most expensive drugs in the world has been broken, and the newly priced drug king was born.

When many people are still booing this 2.8 million, on September 16, 2022, the blue bird creature issued another press release, and the official announced another shocking news, that is: FDA has accelerated approval. The company's gene therapy drug Skysona (EliValdogene AutoteMce, Eli-Cel) is listed to slow down the progress of boys' neurological dysfunctional dysfunctional dysfunctional dysfunction (CALD) in early 4-17 years old. Skysona is priced at US $ 3 million in the United States. This means that the record of the world's most expensive drugs was broken again in just 30 days, and the latest sky -high medicine king was born. Borrowing a sentence that everyone often hangs on the mouth, "There is no highest, only higher!". If we hadn't seen each other, we would never believe it! ~~ The following are some details.

2.8 million records

On August 17, 2022, American time, Blue Bird announced that the FDA approved its gene therapy product Zynteglo (Betibeglogene AutoteMcel, Beti-Cel) for the treatment Type adults, adolescents and children.

Picture source: official website of Blue Bird Biological Company

ZyNTEGLO was approved to be listed in the EU in June 2019. Later, due to the differences with the European medical insurance company in the selling price, it overall withdrawal from the European market in October 2021. ZyNTEGLO was suspended by the FDA due to the CMC problem. On June 10, 2022, the FDA held an expert consulting committee to discuss whether the benefits of ZyNTEGLO were greater than risks.

According to Blue Bird Bio, patients with blood transfusion dependent β-thalassemia are based on traditional therapy. The cost of lifelong treatment is about $ 6.4 million, and the price of ZyNTEGLO is set to $ 2.8 million. ZyNTEGLO is currently the most expensive drug in the world. The most expensive drugs were the gene therapy products of Norhua Pharmaceutical Zolgensma, priced at $ 2.125 million.

In addition to obtaining priority review (PR), fast channel (FT), breakthrough therapy (BT), and orphan drug qualifications, ZyNTEGLO also obtained rare pediatric diseases. After obtaining priority review and approval, Bluebird BIO also received a priority review voucher, namely Priority Review Voucher. According to reports, PRV value is high and exceeds 100 million US dollars.

3 million records

On September 16, 2022, the Blue Bird Biological Website announced that FDA has accelerated the company's gene therapy drug Skysona (ElivalDogene AutoteMce, Eli-Cel) to slow down to slow down the early active brain adrenal and encephalopenar malnutrition ( CALD) Progress of Boys' Neurological Dioxide. This is the first drug approved by FDA for patients with CALD.

Picture source: official website of Blue Bird Biological Company

It is reported that about 40 to 50 patients in the United States are diagnosed with CALD. Bluebird is expected to treat about 10 patients per year.

The company announced that Skysona's pricing in the United States is $ 3 million.

Andrew Obenshain (Picture Source: Company Official Website)

Mr. Andrew Obenshain, president and CEO of Blue Bird Bio, said, "Since the company was established more than ten years ago, children and their families have been the core of the blue bird mission. For the CALD community, this long -awaited approval representative With huge hope, it provides a new choice for the family.

We thank everyone who participated in the development of Skysona and will work in cooperation with all parties to provide this important treatment option for patients and their families. "From June 9-10 this year, FDA invited external experts to participate in the meeting of cells, tissue, and gene therapy consulting committees (CTGTAC) to discuss the two-gene therapy product of Blue Bird Bio Eli-Cel and Beti-Cel for listing applications. The results of the meeting were positive, and both products were supported by the expert committee.

It is not easy to develop, and the company has faced difficult situations many times, including the ELI-Cel clinical development project has been released by the FDA clinical restrictions. On September 16, the company confirmed that the previous FDA restrictions have been lifted.

Reference materials:

$ 2.8 million! The most expensive drug breaks! Oral SERD tracks are frustrated; the ADC track competition has intensified, and the star company changes CEO. Essence Essence | "Pharmaceutical Medicine Newsletter" (No. 2) Bluebird Biological Paragraph 2 gene therapy was approved by FDA, priced at 3 million US dollars

$ 3 million! FDA approves the most expensive genetic treatment in history

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