Drugs and biologics often end up with multiple names as they travel through the development and commercialisation. It can all get very confusing and even more so after the therapeutic is on the market but turns out to have multiple different names across multiple different jurisdictions. This article is designed to help investors understand drug naming conventions, the issues multiple names can create and the signals they might take from them.
Note, just to be clear, a biologic is a therapeutic, like a drug, that is produced from living organisms or contain components of living organisms. They are regulated by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in a similar, but separate, way to standard drugs. Here we just use the term drug for simplicity because naming conventions for drugs and biologics are essential the same.
Research & Development Stage Name.
Initially, a drug is usually given a research and development (R&D) stage name that is related to the group of molecules that the company is studying while it is looking for the right molecule to move into formal pre-clinical studies. For example, the lead drug for Kazia Therapeutics’ (ASX:KZA) brain cancer (glioblastoma multiforme) program, GDC-0084, was given its name to identify it from many similar molecules being put through studies to determine which one had the best characteristics for treating the particular brain cancer in question. This R&D stage name often begins with a few letters and ends in numbers. The letters may be derived from the company name, the laboratory name, the nature of the molecules, the disease they are looking to treat, a combination of those things, company policy or, simply, someone’s choice. The numbers are usually simply sequential.
International non-proprietary name.
Once the drug candidate looks like it might have real legs, the company will apply for an international non-proprietary name (INN). An INN is granted by the World Health Organisation (WHO), who coordinates drug naming worldwide to avoid confusion and create scientific uniformity. A name is granted upon application to the WHO. The WHO provides guides for how companies should go about deciding on which INNs might be suitable for their drug. Having digested those guidelines, the company will submit several names to the WHO. The WHO then runs its own checks of the names and sends a summary of the checks to WHO INN experts for comments. It is only when these experts all agree on one name, that the applicant is informed and the name published in WHO Drug Information, which indicates 4-month period for anybody to object to the name. If no objections are raised, the chosen name becomes the drug’s permanent INN. In Kazia’s case, the WHO approved the name, paxalisib. INN’s are always lower case unless they start a sentence. The end letters are given according to the active part of the drug, so the “lisib” in paxalisib indicates the drug is an inhibitor of the enzyme phosphatidylinositol 3-kinase. Drugs targeting this enzyme are fairly rare, but the enzyme is a proven cancer target. The ending “mab” means the drug is a monoclonal antibody and you will see a lot more of these types of drugs around than many others. You do not really need to know what each ending means. That is what Google is for.
The final name the drug will get is its commercial name, the one it is marketed under, like the anticoagulant Eliquis®. Normally, when you are writing formal material, the first time you mention a drug’s commercial name, you will put its INN and the makers in brackets afterwards. For example, Eliquis® (apixaban; Bristol-Myers Squibb, Pfizer). The xaban refers to the fact that the active part of this drug inhibits a molecule termed factor Xa, which when blocked, slows patients’ blood from clotting. The commercial name of a drug is derived in a similar fashion to the INN, but the considerations are different. The company will, again, several its marketing team/consultants have chosen. The names are chosen for their perceived marketing power and that they fit the FDA’s and/or EMA’s, guidance documents for choosing drug names. The FDA’s main guidance document of choosing a drug name in 37 pages long and refers to several other guidance about choosing a drug name, including a guidance document that explains how a drug name should be actually tested for suitability by the company. The FDA and EMA will then choose one of the names submitted, according to the selection criteria outlined in the guidance documents. More often than not, they seem to choose different ones (doing the drug marketers a real favour, not) and that is one reason why a drug might be called one thing in the US and another in the EU, and even others outside of those two jurisdictions (another is different companies owning different jurisdictional rights to a drug). The primary criteria that comes into play when the FDA or EMA chooses from the list the company submits, is how closely the name resembles existing commercial drug names, particularly, for drugs that, if confused, could harm the patient if a mistake in dispensing the medication were made. This is not an insignificant problem and, despite it probably having improved since, a 1999 report by the Institute of Medicine found that medication dispensing errors accounted for an estimated 7,000 deaths annually in the United States. Doctors write a lot of prescriptions and are notorious for their bad handwriting or scribbling, you might more accurately say. Regulators learned the hard way that they had to control commercial drug names. Imagine being given a drug to increase your blood pressure when you were supposed to receive one to lower it? Giving a stroke patient the wrong medication can be lethal.
There are also other rules for naming drugs. For example, a drug name cannot bring an air of superiority with it, like adding the prefix ‘super’ or ‘best’ to the name. The net result of all of the rules regarding drug names is that we get some pretty weird drug names, like Zytiga, Kineret, Imlygic, Praxbind and Xeljanz.
Drug Names Used in the Literature and at Conferences.
In research literature and at conferences, while a commercial name may be mentioned, it is the INN that is used throughout papers and presentations. There are a couple of reasons for this. The first is to avoid confusion, the INN is unique around the world no matter what other names the drug may be called and any academic (scientist or doctor) anywhere in the world with expertise in a particular area will understand exactly what drug is being talked about if you use the INN. A second reason INNs are used is that academics want their work to be seen as independent as possible and not promotional of a particular company or brand, even though the INN may well be specific to a particular brand name if it still has market exclusivity or no generic has been developed. The latter is, generally, unlikely, without exclusivity. If it was a company, like the maker of the drug that was studied, who funded the study or clinical trial, the researchers must still disclose this in a particular spot at near the end of the paper.