ALPHABET SOUP: NEW DRUG APPROVALS
Acronyms abound in the world of new drug approvals. Check out our cheat sheet below for help with some of the most common:
BLA: Biologic Licensing Application
Planning on developing a biologic drug? Would you like to bring it to market? You will need to submit one of these to the FDA which gives them the details on your manufacturing process, chemistry, pharmacology, clinical pharmacology, and the medical effects of your biologic.
EMA: European Medicines Agency
Roughly parallel to the FDA, the EMA is the European Union’s agency for the evaluation of new medicinal products. An EMA approval means market access to its 27 member countries without having to seek approval in each individually.
IND: Investigational New Drug
Congratulations! There is a molecule you consider a viable candidate for commercial development, understand its pharmacological properties, and have already run animal studies to determine it will not be acutely toxic to human beings. Now it is time to contact the FDA and fill out an IND, which upon approval, grants you the ability to begin limited human trials.
IRB: Institutional Review Board
Once you are in clinical trials, an IRB—sometimes known as an independent ethics committee or ethical review board—is formally designated to approve, monitor, and review biomedical and behavioral research involving humans. The chief priority of IRBs is to protect human subjects from physical or psychological harm.
NDA: New Drug Application
The NDA application is the vehicle through which drug sponsors formally propose that the FDA has approved a new pharmaceutical for sale and marketing in the U.S. The data gathered during the animal studies and human clinical trials of an IND become part of the NDA.
TERM OF THE WEEK: RARE DISEASE
In the United States, the FDA considers disorders that affect fewer than 200,000 people a rare disease.
The term “rare disease” is often interchanged and synonymous with orphan diseases. The latter term may encompass both rare diseases and disorders that affect more than 200,000 people, but for which there is no reasonable expectation for the cost of developing and marketing such a drug would be recovered.
EASILY CONFUSED: BREAKTHROUGH VS. FAST TRACK DESIGNATION
Both designations are intended to expedite the development and review of drugs for serious or life-threatening conditions; however, there are differences in what needs to be demonstrated for each designation.
To qualify for breakthrough therapy designation, the drug sponsor must provide preliminary clinical evidence indicating that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies.
In contrast, a drug may qualify for fast track designation by submitting either nonclinical or clinical data that demonstrates the potential to address unmet medical need.
COCKTAIL FODDER: FIRST BREAKTHROUGH DRUG
Gazyva is a treatment for chronic lymphocytic leukemia. It was approved by the FDA on November 1, 2013 and has the distinction of the first drug with breakthrough therapy designation to reach market.
Emily Burke, PhD has worked in biopharma for 20 years, gaining science writing experience at The Scripps Research Institute and Ionis Pharmaceuticals. As a Ph.D. molecular biologist, she is passionate about advancing the public’s understanding of science. In addition to being a self-proclaimed “science geek,” she is regularly asked to speak at international scientific meetings. When not teaching and writing the WEEKLY for Biotech Primer, Dr. Burke swims with her swim club and performs regularly on the improv circuit in San Diego.