US FDA approved new drug for Alzheimer's disease - Advisory Committee said "insufficient evidence"

Recently CNN.co.jp reported the following:

アルツハイマー病の新薬、米ＦＤＡが承認　諮問委は「証拠不十分」

2021.06.08 Tue posted at 15:09 JST

（ＣＮＮ） 米食品医薬品局（ＦＤＡ）は７日、初期段階のアルツハイマー病に使われる実験的新薬「アデュカヌマブ（別名アデュヘルム）」の使用を承認した。同薬についてはＦＤＡの諮問委員会が昨年、治療効果を裏付ける証拠が不十分だと結論付けていた。

アデュカヌマブは重度の認知症ではなく、軽度の認知障害をもつ患者向けに開発された。症状を和らげるだけでなく、アルツハイマー病の進行を遅らせることを目指している。

ＦＤＡがアルツハイマー病の新薬を承認するのは２００３年以来。

アデュカヌマブは、効果に関してさらなる研究が必要な段階でも、重大な疾患や命を脅かす疾患については承認を早める「迅速承認」の制度を使って承認された。

ＦＤＡ医薬品評価研究センターのパトリツィア・カバゾーニ所長は、アデュカヌマブを承認すべきかどうかについては相当の論議があったとした上で、「アルツハイマー病の患者にとって、アデュヘルムにはリスクを上回る効果があると判断した」と迅速承認の理由を説明した。

迅速承認制度では、「承認後研究」と呼ばれるフェーズ４の臨床試験を実施して、臨床効果を検証することが医薬品会社に対して義務付けられている。もし効果が確認されなかった場合、ＦＤＡがその薬品を市場から締め出すこともある。

ＦＤＡの末梢（まっしょう）・中枢神経系薬物諮問委員会は昨年１１月、アデュカヌマブの効果に関する複数の疑問について判断を求められた。初期のアルツハイマー病に対するアデュカヌマブの治療効果については、１つの有望な研究結果を治療効果を裏付ける主な証拠とみなすことの合理性を問う投票を行った結果、合理性を認めた票は皆無で、合理性を否定する票が１０票、不確実とする票が１票だった。

アデュカヌマブは製薬大手のエーザイと米バイオジェンが共同開発した。

Translation

(CNN) On the 7th, the US Food and Drug Administration (FDA) approved the use of a new experimental drug, aducanumab (also known as Aduhelm), used for early-stage Alzheimer's disease. Last year, the FDA's advisory board concluded that there was insufficient evidence to support the therapeutic effect of the drug.

Aducanumab was developed for patients with mild cognitive impairment rather than severe dementia. It aimed not only to relieve symptoms but also to slow the progression of Alzheimer's disease.

For the FDA to approve a new drug for Alzheimer's disease, this was the first time since 2003.

Aducanumab was approved under a "fast-track" system that expedited drug approval for serious and life-threatening illnesses, even it was at a stage where further research would be needed on its efficacy.

Patrizia Cabazoni, director of the FDA Center for Drug Evaluation and Research, while saying that there was considerable debate over whether aducanumab should be approved, also explained the reason for the rapid approval in that "we have determined that the effect of Aduhelm outweighs the risk for patients with Alzheimer's disease".

In the accelerated approval system pharmaceutical companies were required to conduct Phase 4 clinical trials known as "post-approval studies" to verify clinical efficacy. If no effect could be confirmed, the FDA might keep the drug out of the market.

The FDA's Peripheral and Central Nervous System Drug Advisory Board was asked last November to judge several questions about the effects of aducanumab. Centering around the therapeutic effect of aducanumab on early Alzheimer's disease and on the therapeutic effect of aducanumab, a vote was cast to inquire the reasonableness of considering one promising study as the main evidence to support the therapeutic effect, there were no votes that recognized reasonableness, 10 votes denied reasonableness, and one vote was for uncertainty.

Aducanumab was jointly developed by pharmaceutical giant Eisai and US Biogen.

So, the US Food and Drug Administration has approved the use of a new experimental drug to cure early-stage Alzheimer's disease despite the fact that there is insufficient evidence to support the supposed therapeutic effect of this drug. I am interested in knowing how this drug will perform in its Phase 4 clinical trials known as "post-approval studies".