- Priority Review (PR)
- Breakthrough Therapy (BT)
- Accelerated Approval (AA)
- Fast Track (FT)
A more in depth description of these additional designations and the advantages of getting them is available if you CLICK HERE.
Those drugs approved for use in mutiple myeloma in the last 16 years and the date of FDA approval for clinical use is as follows:
Velcade 2003 Pomalyst 2013 Ninlaro 2015
Revlimid 2006 Faradak 2015 Darzalex 2015
Doxil 2007 Empliciti 2015 Selinexor 2019
How can history repeat itself? First if we find what drugs which have at least two FDA designations but are not as yet approved by the FDA for clinical use, this just may be a very good list to see what the next FDA approvals likely will be. I count 7 drugs which have an orphan drug and one other FDA designation. For all of the drugs above which have been approved the average time from orphan drug designation to FDA clinical approval is 4 years, as compared to an average of 8 years without these two designations. A remarkable feat of twice as fast to market. Those 7 new drugs are with dual FDA designations are:
Drug Company Designations Description
AMG420 Amgen OD&FT (BCMA) Bispecific T-Cell Engager
(BiTE®) Antibody Construct
CLR131 Celectar OD&FT Radiotherapeutic phospholipid drug
GSK2857916 GlaxoSmithKline OD&BT (BCMA) antibody-drug
bb2121 Celgene OD&BT BCMA chimeric antigen receptor
Galinpepimut-S Sellas OD&FT Immunotherapy vaccine to elicit a
strong response against WT1
P-BCMA-101 Poseida OD,FT&BT Autologous chimeric antigen receptor
(CAR) T-cell therapy
Melflufen Oncopeptides OD&AA Drug activated by aminopeptidases,
overexpressed in myeloma
Many more drugs have an OD designation, but as yet have not received the second designation. So additional likely candidates could be added if and when they receive an additional designation by the FDA. This is remarkable when you consider the average orphan disease has less than one approved drug for each of the 6000 orphan diseases! If history repeats itself, I would argue we have a high probability of having 7 new drugs approved for the treatment of myeloma within 4 years or a rate almost 3 times that of the historic rate of myeloma drug development. This would be absolutely miraculous! I must thank the FDA with government support for putting in place the Orphan Drug Act, but again there is a synergistic magical sauce in the myeloma care community which has made myeloma a template for rare disease drug development. All the players in this community which includes the researchers, academics, myeloma specialists, patients, caregivers, patient advocates, large and small drug companies, supportive care teams, NCI, FDA, IMF, MMRF, LLS, Myeloma Crowd, and all those I may have left out, work in a talented cooperative team environment!
In this group of drugs are 6 new drug classes, and for each new class of drugs approved by the FDA we have had a 1 year increase in life expectancy. If history repeats itself, 6 new drugs would mean 6 more years of life expectancy for all myeloma patients, and 6 more steps towards THE CURE.