A Food and Drug Administration (FDA), the health regulatory agency of the United States, approved this Monday (07) – after many controversies – a new drug that promises to slow the progression of Alzheimer’s. the medicine Aduhelm, made with aducanumab, showed evidence that it reduces amyloid plaques in the brain – the increase of which is associated with Alzheimer’s disease. The drug, manufactured by the pharmacist Biogen, is the first launched against the disease since 2003.
Until the drug was approved by the FDA, it took years of research and billions of dollars in investments. The drug’s sale offers hope to millions of families who have carriers of the disease – which affects memory and is known for the lack of good treatment options. But not all people with the disease should benefit from aducanumab: doctors believe the drug will have little or no effect for people with more advanced stages of the disease.
Richard Isaacson, director of the Alzheimer’s Prevention Clinic at New York Presbyterian Hospital and Weill Cornell Medical Center, told CNN International in an interview that aducanumab targets the earliest symptomatic stage of the disease, called mild cognitive impairment. “We have to moderate expectations and explain to people that this drug is intended for the first symptomatic stages,” he explained.
For the manufacturer Biogen, the approval came at a good time: the company has been facing declining sales and the loss of patent protection for its best-selling drug, the Tecfidera. The drug fights multiple sclerosis and is responsible for approximately a third of the drugmaker’s revenue today.
A Biogen developed the drug in partnership with the Japanese Eisai. The drug is given via an intravenous infusion as an early treatment for Alzheimer’s disease and is designed for patients with mild cognitive impairment – not for advanced stages of dementia – and is intended to slow the progression of the disease, not alleviate symptoms.
In March 2019, Phase 3 clinical trials were stopped after an analysis concluded that the research would not achieve its primary objective at completion. “The futility analyzes showed that the studies were more likely to fail,” Isaacson said. Months later, Biogen announced a new, more comprehensive analysis and showed that patients who received high doses of aducanumab had a reduction in clinical decline – 22% less in approximately 18 months.
But in November 2020, the FDA’s Central and Peripheral Nervous System Drug Advisory Committee concluded that there is insufficient evidence to support the treatment’s effectiveness. The committee held a vote on the drug’s efficacy, and when asked about the reasonableness of considering data from a positive trial as primary evidence of the efficacy of aducanumab for the treatment of early-stage Alzheimer’s disease, ten members voted no and one was uncertain .
Isaacson explained that it would take years to repeat the studies and, given the lack of available therapies to treat the pre-dementia phase of the disease, Biogen made the atypical decision to apply to the FDA for registration of the drug in July 2020. The cost of treatment, according to the American Institute of Clinical and Economic Review, should run between US$ 2,560 and US$ 8,290 – more than R$ 12 thousand up to R$ 41.6 thousand per year.
Medicine will have a high cost, according to a specialized entity.Source: Pixabay
Despite the chorus of non-approval by entities such as the Public Health Research Group for US Citizens – a non-profit organization that drew attention to the drugmaker’s close connection with the FDA, and the high price of the therapy, the agency gave a positive response to the company, which is now expected to continue with Phase 4 trials after commercialization. If the drug fails in these studies, the FDA can revoke approval.
Other organizations, such as the American Alzheimer’s Association, supported the drug’s approval. “Whatever the FDA’s final decision is, this is an important moment. We’ve never been so close to approving an Alzheimer’s drug that could change the progression of the disease, not just the symptoms. Together, we can continue to work towards our vision of a world without Alzheimer’s and all other dementias,” he said in a statement.
In Brazil, the manufacturer filed a regulatory review request with the National Health Surveillance Agency – Anvisa. Biogen is also seeking authorization to market Aduhelm in other countries, in the European Union and in Japan.