The annual cost exceeds 21 million, and the world's most expensive medicine record is refreshed

The annual cost exceeds 21 million, and the world's most expensive medicine record is refreshed

Source: Sai Blue/Taro

01

"The most expensive drug in the world" record, it was refreshed twice a month

On September 16, the American Blue Bird Bio announced that the FDA has accelerated the company's gene therapy drug Skysona (ElivalDogene AutoteMCE, Eli-Cel). It is the first and the only one -time gene therapy in the early CALD approved treatment in the world.

Bluebird creatures set SKYSONA's wholesale acquisition in the United States to $ 3 million (at the current exchange rate equivalent to about RMB 2.123 million, the same below), becoming the world's most expensive treatment method.

A month ago, this record was just refreshed by the Treatment cost of $ 2.8 million by the blue bird creature.

Prior to the treatment of SKYSONA treatment, the effective choice of CALD patients is limited to Allo-HSCT transplantation (Allo-HSCT), which is accompanied by severe potential complications risk, and even death, especially in the same manner without human white blood cell antigen (HLA). Among patients, such risks may increase sharply.

As one of the conditions for SKYSONA's accelerated approval, Blue Bird Biological has agreed to provide FDA with confirmation long -term clinical data. Blue Bird Bio said that this will include the ongoing long-term follow-up research (LTF-304) data, which tracks patients who have been treated in clinical trials for 15 years and patients from commercial treatment.

Blue Bird Biological estimates that Skysona will provide Boston Children's Hospital and Philadelphia Children's Hospital by the end of 2022 by the end of 2022.

02

There are five drugs, and the annual treatment costs exceed $ 1 million

Combined with the statistics released by the US prescription drug comparison platform Goodrx this year, including Skysona of Blue Bird Bio, there are currently five drugs for the annual treatment costs of more than $ 1 million.

Blue Bird Bio "ZyNTEGLO": 2.8 million US dollars (equivalent to about 19.81 million yuan)

ZyNTEGLO was approved by FDA on August 17 this year. It is a one-time gene therapy that is used to treat patients with β-thalassemia who requires regular blood transfusion. The first gene therapy for the disease.

Before being approved for listing in the United States, Zynteglo was approved for listing in May 2019. At that time, the price was about 1.8 million US dollars, but the commercialization expansion was not good. In April 2021, due to the failure to reach a price consensus with the German regulatory agency, Blue Bird Bio announced that ZyNTEGLO withdrew from Germany.

At present, Chinese companies are also developing the treatment of poor gene therapy. In August of this year, Bangyao Biological announced that it had a clinical trial application of the genetic therapy product "BRL-101 autologous hematopoietic ancestor injection" of the genetic therapy product "BRL-101 autologous hematopoietic ancestral cell injection", which was officially obtained on the day The approval of the center.

Novartis "ZOLGENSMA": US $ 2.125 million (equivalent to about 15.04 million yuan)

ZOLGENSMA was approved by FDA in 2019 and used to treat spinal muscle atrophy. This is a rare children's disease that can cause muscle erosion and may cause lung infection and muscle weakness.

ZOLGENSMA is a one -time treatment, and many children who have received the treatment of the drug have not re -showed signs of illness. However, paying for Zolgensma is not easy, because many insurance companies may not underwrite, or have restrictions on the insured, and can only obtain insurance on the premise of meeting certain requirements in the insured.

In order to encourage insurance companies to pay this genetic treatment cost, Zolgensma's manufacturer Novartians allow certain insurance companies to pay for 5 years, and each phase of drug treatment costs 425,000 US dollars. Novartis can also provide Onegene services to provide guidance to patients and help them meet insurance requirements.

China ’s drug clinical trial registration and information publicity platform shows that in April 2022, ZOLGENSMA has officially launched the third phase of the clinic in China. 8 domestic institutions including Peking University Hospital and Xiehe Hospital participated in it.

Eiger biopharmaceutical "ZOKINVY": US $ 10.74 million (equivalent to about 7.6 million yuan)

Zokinvy is an orphan drug, which was approved by the FDA in November 2020.

This is the Harchienson-Gilford Pungeon Parment Syndrome-a rare genetic disease that leads to premature aging, which is approved, and the only treatment method.

Patients with different heights and weight are different. Generally speaking, patients generally take about 200 mg of ZOKINVY per day. Based on $ 746 per 50 mg of capsules, the cost of drug treatment for patients per month is $ 89,480.

Although this drug does not have a common payment card, the EigeroneCare program launched by the manufacturer can help provide insurance scope and financial support options.

In February 2022, ZOKINVY was survived by Hong Kong Victoria Pharmaceutical Group Co., Ltd. for importing clinical urgent need for drugs. Y-MABS "Danyelza": US $ 1.012 million (equivalent to about 7.16 million yuan)

The FDA approved Danyelza in November 2020 to treat pediatric and adult patients' skeletal or neuroblastoma in the marrow.

In August 2021, Sesheng Pharmaceutical and the United States Y-MABS reached an exclusive indefinite authorization license agreement, introducing the world's first person in the world to Danyelza (Nart Midam, HU3F8) to China, and took the lead Hainan Boao Le City's International Medical Tourism Pioneer area landed.

In December 2021, the price of Danyelza rose 3.5%, making the cost of a single small bottle reached $ 2,081. Patients usually make about 48 bottles per year.

Manufacturer Y-MABS offers Y-Mabsconnect, which is a plan that can help people choose insurance and provide assistance to patients without insurance and meet other conditions.

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