While there are 7,000 orphan diseases globally, there are only 500 drugs available to treat all 7,000 diseases. In September of last year I wrote a blog post outlining how all new myeloma drugs classes in the last 16 years have had two FDA designations for each approved drug: one being an Orphan Drug Designation and the other being Fast Track, Priority Review, Breakthrough, etc. Having the Orphan Drug Designation truly matters in myeloma. On average, the time for FDA approval as an Orphan Drug is 5.3 years as compared to an 8-year average without the FDA Orphan Drug Designation!
Myeloma patients are blessed to have had 10 new drugs approved in the last 16 years. In addition, myeloma (considered an orphan disease) has seven new drugs which all have dual FDA designations, four of which are CAR T. Last year I called the American Society of Hematology (ASH) meeting a CAR show, and it continues at this year's 2020 ASH meeting.
These four include Jannsen’s JNJ-4528, CARsgen’s CT053, Bluebird’s bb2121, and Poseida’s P-BCMS-101. I attended a session which presented the last three and I will focus on two specifically: CT053 and P-BCMS-101.
Why was I impressed with this drug? In the phase I trial, the overall response rate (ORR) was very high at 79.2% and even higher at 100% in the Phase 1b. The duration of response was twice as long as other trials (21.8 months) and a new manufacturing process allows manufacting of 8 to 10 to reduce the need for bridging therapy. Many current methods take one to two months. It was also important to see that there was no Grade 3 or higher cytokine release syndrome (CRS).
Why do I like this drug? This is a VERY heavily pretreated patient population with an average of seven prior therapies, twice that of some other studies yet has a similar overall response rate of 67%. Cytokine Release Syndrome (CRS) was very low at 17% with an excellent safety profile. They have a new non-viral manufacturing process called piggyBack which provides a higher level of Stem Cell Memory T Cells which are felt to provide the best clinical response, better safety profile, and the potential for a response if reapplied.
The future looks very bright!
about the author
Gary is a myeloma survivor and patient advocate. His work centers around helping patients live longer by helping them to find facilities who are beating the average survival statistics. You can find Gary's site at www.myelomasurvival.com and follow him on Twitter at @grpetersen1