Drug Regulation does not work! When it discovers a harmful drug it does little/nothing. It is failing to address its primary purpose - to safeguard patients from harm
This is Medscape’s headline. “FDA Issues Alert on Liver Injuries Linked to Vasculitis Drug, Following Withdrawal Request”.
The FDA is the USA’s Drug Regulator, but its decisions are widely influential with drug regulators around the world. So its decisions affect all of us. It issued an alert about liver injury, including fatal cases, that have been linked to avacopan (Tavneos), a drug used for a rare autoimmune disease “that the agency had already asked to be voluntarily removed from the market”
Dangerous enough, you might think, for it to be withdrawn by the drug company, and/or banned by the regulator?
The Medscape article went on to say that on 31st March 2026 the FDA issued a public notice outlining their serious concerns about post-marketing cases of drug-induced liver injury that have been associated with avacopan. However, this seems to have carried little weight with Amgen, the drug’s manufacturer, who
“..announced in January that it intended to continue to market this drug even though the FDA requested a voluntary withdrawal of avacopan. Separately, European regulators in January announced a safety review of the drug”.
The article goes on to state that the problem about this drug had been known for over 5 years.
“In 2021, FDA staff expressed deep concern about liver risk even while approving avacopan for a serious and sometimes fatal autoimmune condition, severe active antineutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis. The FDA required a special safety study focused on liver risk as part of this approval.
So, 5 years on, what does the drug regulator do to fulfil its main function - to protect patients from a drug that causes patient harm, and is known to cause fatalities?
“In the new alert, the FDA recommends that patients talk to their healthcare professional about safety risks associated with avacopan and to discuss whether to continue therapy or switch to alternative treatments” especially if “they develop any signs or symptoms of liver injury, including nausea, vomiting, unusual itching, light-colored stools, yellowing of skin or eyes, dark urine, swelling in the stomach or abdomen, or pain in the right upper abdomen. The FDA also advised clinicians who have patients taking avacopan to conduct liver panel testing every 2 weeks in the first month of treatment, monthly for the next 5 months, and thereafter as clinically indicated”.
The FDA continues to state that avacopan should be discontinued promptly, and alternative treatments considered, in certain circumstances.
So there is a difference of opinion. The company is clearly confident that avacopan is both “effective and safe”, based, it says, on “robust clinical data and real-world evidence demonstrating the effectiveness and favorable benefit-risk profile”. And it is committed to continue marketing the drug.
However, the FDA says it has identified 76 cases of drug-induced liver injury “with reasonable evidence of a causal association with avacopan use … that comes from reviews of postmarketing data, medical literature, and the FDA Adverse Event Reporting System ... Of these 76 cases, 74 reported a serious outcome, including 54 hospitalizations and eight deaths”.
These clinical outcomes might be considered adequate grounds for taking more robust protective action for patients in addition to “discussing” the issues with a doctor. But no. One comment outlined in the Medscape article states that:
“The FDA’s Drug Safety Communication about avacopan highlighting the eight fatal cases of drug-induced liver injury is important, but insufficient”.
So, a drug that causes 8 deaths is not sufficient reason to withdraw, or ban it. How many more drugs are drug regulators approving, drug companies selling, and doctors prescribing, that are causing this level of patient harm?
Even Medscape raises this question. It asks why, if the FDA has requested the drug be voluntarily withdrawn from the US market, the agency not making this request publicly, and pushing back against the company for not having already withdrawn the drug? Why does the prescribing information for avacopan not include a boxed warning for the risk of fatal liver disease?
These are urgent questions for the FDA to answer. Yet the FDA has failed to do so, and so confirms that no pharmaceutical drug should be considered “safe and effective”, that drug regulators are failing to act decisively to protect patients, that doctors will therefore continue to prescribe harmful drugs to patients.
Avacopan is a small drug. But it represents a good example of how conventional medicine now treats the issue of patient safety. It demonstrates a medical system that is prepared to “discuss” how many patients it is allowed to kill, how much chronic disease it is allowed to cause, before any robust action is taken to protect us. It shows h
ow patient safety takes second place to pharmaceutical profits.
