If History Repeats Itself, The State of Myeloma New Drug Development Is STRONG! The Myeloma Miracle – Part 3


In the last 16 years multiple myeloma has had 10 drugs approved. All of these drugs have had two things in common, they all were awarded an FDA Orphan Drug(OD) designation, and secondly they all had one of the additional FDA designations of Fast Track, Breakthrough Therapy, Accelerated Approval, or Priority ReviewL.  NO new drug has ever been approved for myeloma in 16 years without at least two of these FDA drug approvals.  To be an orphan drug it must first affect no more than 200,000 patients in the US. The FDA’s Office of Orphan Products Development is tasked to evaluate scientific and clinical data from companies to identify which therapies can improve the lives of people suffering from rare diseases. Orphan status is given to drugs and biologics defined as “those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders”. The other destinations are designed to speed the availability of drugs that treat serious diseases, especially when the drugs are the first available treatment or if the drug has advantages over existing treatments. The Food and Drug Administration has developed four distinct and successful approaches to making such drugs available as rapidly as possible:

  • 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 

Kyprolis       2012     
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

                                                                                                 conjugate (PDC™)
GSK2857916               GlaxoSmithKline           OD&BT          (BCMA) antibody-drug   
                                                                                                 conjugate (ADC)   
bb2121                          Celgene                       OD&BT          BCMA chimeric antigen receptor
                                                                                                 CAR T

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.

Good luck and God Bless your Cancer Journey/ editor@myelomasurvival.com
For more information on multiple myeloma CLICK HERE and you can follow me on twitter at: https://twitter.com/grpetersen1