A number of publications have appeared recently reporting on the efficacy and safety of cannabidiol (CBD) as an add-on therapy, as its clinical trials are concluding and its use is extending. This paper describes an “expanded access” program – that is, when the FDA allows access to a drug before approval. This is of interest because it is more like the normal, clinical use of a drug, as opposed to the way it is administered during a formal controlled trial.
CBD was utilized in conjunction with a variety of other drugs, with the most common partners being clobazam (ex. Onfi) (66%); valproic acid (ex. Depakote) (43%); and levetiracetam (ex. Keppra) (34%). At the first time-point of 12 weeks, the percentages of LGS/DS patients with ≥50%, ≥75%, and 100% reduction in major motor seizures were 53%, 23%, and 6%. Numbers for reductions of total seizures were actually similar (46%, 26%, and 5%). These levels of response remained quite stable throughout the study period (to week 96). The most common adverse effects were sleepiness (30%) (especially when in combination with clobazam) and diarrhea (24%). 41% experienced “serious AE’s”, including convulsion (14%), status epilepticus (9%), pneumonia (5%), and fever (4%).
All of these results are quite similar to those found in the controlled clinical trials, and those now being reported from formal extension trials focused on Dravet patients.