FDA Narrows Aduhelm Label Amid Controversy, Restricting Use to Early-Stage Alzheimer's Patients

Compiled and Translated by | Fan Dongdong

As a highly controversial new drug for Alzheimer's disease, one month after Biogen/Eisai's Aduhelm was approved, U.S. health regulators this Thursday updated the new prescribing information restricting its use.

Recently, the U.S. Food and Drug Administration (FDA) stated that changes to the indication labeling for Biogen/Eisai's Alzheimer's drug Aduhelm are intended to address confusion among physicians and patients regarding which patients should receive the drug. Since its approval last month, the medication has faced intense public backlash.

The updated FDA label emphasizes that Aduhelm is indicated for patients with mild or early-stage Alzheimer's disease, but has not been studied in patients with more advanced stages of the disease. This represents a significant change from the drug's originally approved indication, which previously authorized its use for the general treatment of Alzheimer's disease.

Biogen announced changes to the drug label in a press release on Thursday, stating that it had reached an agreement with the FDA on the label modifications following communications. “Having heard external concerns regarding the drug, the FDA has decided to clarify the prescribing information to resolve this confusion.” Despite the updated label, the FDA added that if “some patients progress to more advanced Alzheimer's disease, they may continue to benefit from treatment with Aduhelm.”

When the drug was first approved, a senior FDA official told reporters that the therapy was "relevant to all stages of Alzheimer's disease." The FDA's accelerated approval of the drug had previously sparked public controversy over its annual price of up to $56,000 and its questionable therapeutic benefits. Three external FDA advisors resigned over the decision to approve the drug, which has even been labeled "the worst drug approval decision in recent U.S. history."

It is highly unusual for an approved drug’s label to undergo a complete revision, particularly within just a few weeks of its initial approval. Dr. Ronald Petersen of the Mayo Clinic, who has previously consulted for Biogen, stated that the drug’s side effects include cerebral edema and hemorrhage. “This action reflects the responsible approach taken by both the FDA and Biogen, aimed at maximizing treatment safety while providing Aduhelm with the opportunity to deliver optimal therapeutic benefits.”

It is worth noting that, to date, Aduhelm has not been proven to reverse or slow Alzheimer's disease. However, the FDA states that the drug can reduce plaques in patients' brains, which may slow the progression of dementia. Nevertheless, many medical experts argue that there is little evidence to support this claim. Biogen is required to conduct follow-up studies to determine whether the drug will slow cognitive decline. Other Alzheimer's medications currently on the market can only temporarily relieve symptoms.

Following the drug's approval, analysts worry that Aduhelm could add tens of billions of dollars in new costs to the U.S. healthcare system, particularly burdening the federal Medicare program. There are approximately 6 million Alzheimer's disease patients in the United States, the vast majority of whom are old enough to qualify for Medicare.

The U.S. House of Representatives has launched an investigation into the FDA’s review and approval process for this drug. Lawmakers in the U.S. Senate have called for a hearing on the drug’s cost and its impact on federal spending. The FDA’s decision to narrow the drug’s label may help alleviate some of the financial burden by reducing the number of patients eligible to receive it. Representative Katie Porter of California has even accused Biogen of exerting “undue influence” on the FDA’s review process.

Many hospitals have already indicated plans to restrict the use of this drug to patients with early-stage disease. Physicians may still prescribe the medication for patients with more advanced disease, but insurers may deny coverage, citing the FDA’s revised label. Wall Street analysts stated that this change is unlikely to significantly impact the projected sales of Biogen’s drug. In a research report, Jefferies analyst Michael Yee noted that the company had already planned to focus its marketing efforts on the 1 million to 2 million U.S. patients with mild Alzheimer’s disease.

Reference:

1、Biogen,FDA walk back controversial Aduhelm label after weeks offiercecriticism

2、FDA updates label on controversial Alzheimer's drug, saying it's only meant for early-stage patients