What does “off-label” medication mean?

“Off-label” medication refers to the use of a drug outside the scope of its formal approval. The English word“off” means “outside,” while“label” can be translated as “designation” or “approval.”

More than 100 different medications are used to treat the symptoms and signs of ALS. Some of these drugs are used off-label. They were originally developed and approved for other conditions, without any initial intention to use them for ALS.

An example of off-label use is the use of the drug pirenzepine, which was originally developed to treat gastritis and peptic ulcers. The drug inhibits secretion in the stomach as well as in the salivary glands. In ALS, pirenzepine is used off-label (for gastritis) to inhibit saliva production and thereby treat sialorrhea. This deliberately exploits a side effect (dry mouth) of the drug to reduce a distressing symptom of ALS (sialorrhea). This principle of off-label use is applied to several drugs in the symptomatic treatment of ALS.