Highly unusual move of an ODE director leading clinical review of a drug had nothing to do with public sentiment regarding Epidiolex's derivation from marijuana, US FDA says; Unger instead wanted to use the review as an example for future reviews to rely more on the agency's analyses rather than those of applicants.
GW Pharmaceuticals PLC's epilepsy treatment Epidiolex (cannabidiol) has been regarded as highly unusual in that it is now a US FDA-approved drug that is derived from marijuana. Just as unusual, however, is a factor that helped it clear the agency approval hurdle: a review of the safety data led by Office of Drug Evaluation I (ODE-1) Director Ellis Unger.
Reviews of clinical data are almost always conducted by medical officers
The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.
Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.
The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.
The Pink Sheet drug review profile investigates the long and rigorous process behind the FDA’s recent relaxation of the cardiovascular safety warning for testosterone products.
Some advisory committee members said adding quantitative data on misuse, abuse, opioid use disorder and overdose to labeling would be helpful, but others worried the two epidemiological studies were not sufficiently generalizable to a broader population.
With review and development uncertainly increasing, "it’s going to take longer for certain targets … to progress to a place where they’ve been derisked enough that big pharma is ready to write a big check," said Andrew Goodman of Paul Hastings.
Two US FDA advisory committees will discuss results from epidemiological studies that attempted to quantify the prevalence, incidence and risk factors for misuse, abuse, overdose and death with opioid analgesics.
