Drug price over 400 million yen - World's most expensive new drug approved by US FDA

Recently CNN.co.jp reported the following:

薬価４億円超　世界一高額の新薬、米ＦＤＡが承認

2022.11.24 Thu posted at 15:48 JST

  （ＣＮＮ） 米食品医薬品局（ＦＤＡ）は２２日、血友病治療の新薬「Ｈｅｍｇｅｎｉｘ」を承認した。開発元のＣＳＬベーリングが発表した価格は治療１回当たり３５０万ドル（約４億８６００万円）で、世界一高額な医薬品となる。

Ｈｅｍｇｅｎｉｘは、血栓を作るのに必要なたんぱく質が作られない遺伝性疾患、血友病Ｂの治療に使われる成人向けの遺伝子治療薬。男性を中心として、およそ４万人に１人がこの疾患を患っている。

一般的な治療では、不足・欠落している凝固因子を補うために定期的な点滴を必要とするが、新薬を使った治療では１回の点滴で済む。

ＦＤＡは新たに承認された新薬を「血友病Ｂ患者の治療の新たな選択肢」と位置づけ、「重い負担を経験している患者のための革新的な治療法の開発における重要な進展」としている。

処方薬などの価格を調査している非営利研究機関ＩＣＥＲは、Ｈｅｍｇｅｎｉｘが世界一高額な医薬品になることを確認した。

ＩＣＥＲは最近行った費用対効果分析の中で、同薬の効果と相殺される費用を比較して、適正価格を２９３万ドル～２９６万ドルと算定していた。

米国でこれまで最も高額だった治療薬は、ＦＤＡが脊髄（せきずい）性筋萎縮症（ＳＭＡ）の治療薬として２０１９年に承認した「ゾルゲンスマ」で、価格は治療１回当たり２１０万ドル。

ＦＤＡは、成人男性約６０人を対象とする２回の研究で安全性と効果を検証した結果に基づき、Ｈｅｍｇｅｎｉｘを承認した。主な副作用として、肝酵素の上昇、頭痛、インフルエンザのような症状、輸液に関連した軽度の反応などが指摘されている。

Translation

  (CNN) The US Food and Drug Administration (FDA) today approved a new drug, Hemgenix, to treat hemophilia. The price announced by developer CSL Behring was $3.5 million (about 486 million yen) per treatment, making it the most expensive drug in the world.

Hemgenix was ​​an adult-use gene therapy drug used to treat hemophilia B, a genetic disorder in which the proteins that was needed to form blood clots were not produced in the body. Approximately 1 in 40,000 people, mainly men, suffer from this disease.

In general treatment, regular infusions were required to compensate for insufficient or missing clotting factors, but treatment using new drugs required only one infusion.

The FDA positioned the newly approved drug as a "new treatment option for patients with hemophilia B" and called it a "significant advance in the development of innovative treatments for patients now experiencing a heavy burden."

ICER, a non-profit research organization that tracked the prices of prescription drugs and other drugs, had confirmed that Hemgenix could become the world's most expensive drug.

In a recent cost-benefit analysis, ICER in comparing the benefits of the drug and the offsetting costs. calculated a fair price of $2.93 million to $2.96 million.

Zolgensma, which was approved by the FDA in 2019 to treat spinal muscular atrophy (SMA), was the most expensive drug in the United States to date, costing $2.1 million per treatment.

The FDA approved Hemgenix based on two studies that tested its safety and efficacy in approximately 60 adult men. Common side effects pointed out would include elevated liver enzymes, headache, flu-like symptoms, and mild transfusion-related reactions.

So, FDA has approved a new drug, Hemgenix, to treat hemophilia. The price will be $3.5 million per treatment, making it the most expensive drug in the world. I am wondering how many people can afford that.