The National Health Surveillance Agency (Anvisa) granted a special permission this Friday (4th) to import the experimental drug ALN-6457. This authorization was intended for the treatment of influencer Lito Sousa, who was diagnosed with Creutzfeldt-Jakob disease (CJD).
This compound, developed by the pharmaceutical company Regeneron Pharmaceuticals, does not yet have commercial registration in Brazilian territory nor an established legal representative in the country. Due to these circumstances, the import release occurred outside of usual procedures for medications.
ALN-6457 is in the preclinical phase for treating prion diseases. It is important to note that formal clinical studies in humans have not yet been conducted to evaluate this substance specifically against CJD, making Lito Sousa the first person to receive this test in this context.
The import request was formalized by the Israelita Albert Einstein Hospital, the institution responsible for monitoring the influencer, after he was accepted into a compassionate use program for the drug. This access modality allows individuals with serious illnesses and no other available therapeutic options to gain exceptional access to drugs that are still under development.
More information about the authorization
The Anvisa endorsement is classified as exceptional and does not equate to a commercial approval of ALN-6457. Since the substance has not completed formal clinical studies initiated in humans for prion disease, there is no evidence that it can reverse or stop the progression of CJD.
This situation differs from that of an already approved medication that has undergone the necessary clinical trials to prove its safety and efficacy. In Lito's specific case, access is provided through compassionate use, given the severity of the condition and the absence of therapeutic alternatives.
CJD is characterized as a prion disease, originating from the anomalous modification of a protein called a prion. This altered protein replicates in the brain, causing neuron destruction and generating a pattern of small holes in the brain tissue, which gave rise to the term 'spongiform.'
The illness is rare, shows continuous progression, and currently, there is no treatment capable of stopping or reversing its course. According to neurologist José Bauab, the prion lodges inside the neuron, causing gradual cellular intoxication. This process also affects glial cells, the tissue essential for supporting and maintaining neurons. Bauab detailed: 'The protein particles enter the neuron, intoxicate the neuron, and exhibit an absolutely destructive and toxic behavior in the cell.'
In Brazil, the disease predominantly affects older people. The age group between 55 and 74 years represented 60.2% of notifications, with an average age of 66 years among suspected cases. This profile differs from Lito's, as the sporadic form of the disease tends to affect individuals between 60 and 80 years old.
Data provided by the Ministry of Health indicates that approximately 90% of patients develop death within a period of six months to one year after the onset of symptoms, with an average survival of about five months. 290 deaths linked to the disease were registered in the analyzed notifications, despite the bulletin pointing out inconsistencies in data completion and monitoring.
Between 2005 and 2021, Brazil recorded 547 confirmed diagnoses of Creutzfeldt-Jakob disease. This period covers the first 17 years since CJD was included in the list of mandatory notification diseases.
