Is the FDA Becoming More Flexible in Drug Approvals? Ali-Urman By Ali Urman | @aurmanARK Analyst
This week, the U.S. Food and Drug Administration (FDA) approved Biogen’s Aducanumab, a drug targeting amyloid plaque buildup in the brains of Alzheimer’s patients. As many families know all too well, Alzheimer’s is a devastating disease during which patients can lose both their memories and their abilities to perform daily tasks.
According to the Center for Disease Control and Prevention (CDC), the number of people aged 65+ suffering from Alzheimer’s disease in the US will nearly triple during the next 50 years, from an estimated 5.8 million people today to 14 million in 2060.
The approval of Aducanumab was surprising for several reasons:
Broad Label: Doctors will be able to prescribe Aducanumab for any Alzheimer’s patients whether they suffer from mild or severe forms of the disease. This broad label was a surprise because the clinical trial included only patients suffering from a mild form of Alzheimer’s.
Cost: Aducanumab will cost $56,000 annually, a high sticker price given its low efficacy.
Toxicity: In its approval, the FDA seemed to place little to no emphasis on toxicity, which is puzzling because Alzheimer’s patients will have difficulty communicating ill effects to their families and care givers.
Clinical Benefit: Biogen will have nine years to complete the trial and demonstrate that Aducanumab delivers clinical benefit.
Imaging Requirements: By the end of their first year on Aducanumab, patients will have to undergo an MRI instead of a positron emission tomography (PET) scan to confirm beta-amyloid, the drug’s target.
Advisors: Three of the nine advisors on the Peripheral and Central Nervous System Drugs Advisory Committee - Aaron Kesselheim, Joel Perlmutter, and David Knopman – stepped down following the approval.
If the FDA seems to be approving therapeutic agents targeted at diseases with high unmet needs before confirmatory trials end, we believe the value of other such therapies showing little clinical efficacy could be much higher than most investors expect.
Submitted June 14, 2021 at 10:56PM by ErinG2021 https://ift.tt/3xpOfqz
