FDA guides special access to retatrutide - before approval for use?

Well this is a first in the obesity world. In USA, the FDA has provided guidance to drugmaker Eli Lilly to make weight loss medication retatrutide available, before regulatory approval, through a special expanded access program.

As blogged previously, retatrutide is a GLP1, glucagon, and GIP (‘triple G’) agonist that has demonstrated previously unsurpassed weight loss, well into the range of weight loss achieved with bariatric surgery. It is currently completing phase 3 trials and is therefore not yet approved by any regulatory agency for use, anywhere in the world.

The American expanded access program for retatrutide is structured to review requests, for specific patients, from healthcare professionals, who:

are age 18 or older
have BMI ≥35 despite adherence to, and tolerance of, treatment with highest available dose of weight management medication
have at least two serious or life-threatening obesity-related complications which are being treated by standards of care
are unable to participate in clinical trials of retatrutide or similar agent (because they don’t meet criteria for the available studies, or don’t have access to an enrolling site)
have had all standard treatment options discussed, including bariatric surgery

The FDA has permitted special access in the past for medications to treat certain cancers, rare genetic disorders like cystic fibrosis, or infectious diseases like HIV. I’d say that special access for retatrutide stands apart in that there are so many people who would fit the above criteria.

I am guessing that one reason for this expanded access program is because of the rampant use of products claiming to be retatrutide, sourced by people from online/overseas companies. Medications that have not yet been approved for use are not legal to distribute for human use; these online/overseas companies get away with selling retatrutide by labeling it as being ‘for research purposes only’. I shudder to contemplate what is in these products: is it actually retatrutide, is the dosing correct, are there other drugs mixed in, are there contaminants, and so on. (More on sketchy compounding practices blogged here; example of fake Ozempic actually containing insulin and winding people up in hospital with severe low blood sugars on BBC here). Not to mention that people ordering reta online from these companies are not getting the support and care from a health care professional to guide in safe titration of medication, how to manage side effects, what to do to monitor other medications/health issues etc. Much of this is being driven by social media and influencers who are promoting retatrutide without understanding these potentially dangerous downfalls.

I have mixed feelings about this special access program for retatrutide. On the positive side, obesity, and the recognition that many people need medication to treat it, is finally being taken seriously as a chronic disease – this program is a signal that regulators and society are finally moving past the ‘just eat less and move more’ narrative. I would also MUCH rather see people getting actual retatrutide from the company who makes it (Eli Lilly), rather than risk the dangerous approach of ordering a product purported to be retatrutide from goodness knows where, not having any guarantee of what is (or isn’t) in it, plus having to manage treatment without health care professional support.

On the downside, we haven’t finished studying retatrutide for safety and efficacy yet (though some of the trials have been completed, but not yet peer reviewed or published, as blogged here and here and here) (disclaimer: I am an investigator in some of these studies, including serving as the Canadian National Lead Investigator on the ongoing TRIUMPH OUTCOMES cardiovascular outcome trial). So, we don’t yet have the full picture of efficacy or safety. Also, the special access program is apparently available to a ‘limited’ number of people – how will they choose who will get access, from the tsunami of applications they are likely to receive?

BOTTOM LINE: The FDA special access program (USA only) for retatrutide reflects that obesity is finally being considered a chronic disease that can benefit from weight management medication, and reflects the tremedous benefit that many people enjoy with retatrutide (based on topline results from trials just completed). Getting actual retatrutide from the makers of the product (Eli Lilly) pre-approval is better than ordering it from online sources with no guarantee of what is (or isn’t) in those products. Caution is important in use of a medication pre-approval, as it hasn’t yet been through the careful safety and regulatory review of data that protects patients and guides clinicians in safe and effective use of medicines.

Disclaimer: I am an investigator in clinical trials of retatrutide. I receive honoraria as a continuing medical education speaker and consultant from the maker of retatrutide (Eli Lilly).

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