The internet is flooded with excitement following the FDA’s limited approval of celgene’s newest IMiD, pomalidomide (trade name, POMALYST). POM is an analogue of thalidomide (trade name, Thalomid). Researchers actually developed POM before Revlimid, but sat on it for a decade–choosing to intensify clinical study activity recently, culminating in yesterday’s FDA approval.
I’m going to delay reporting on the moral indignation some patients feel toward Celgene, arguing that the company could have brought this drug to market more quickly. Just because POM was developed way back in 2002 doesn’t necessarily support their conspiracy theory. But I must admit it does look bad. If the company delayed final development of pomilidomide–waiting for better timing in order to maximize profits–that would be unfortunate; even immoral.
Could POM have reached the market more quickly if Celgene had pushed harder like Onyx did getting Kyprolis approved? I will make a few calls and see if I can’t get the company to release a plausible timeline. In the meantime, let’s set all that aside and focus on the potential of POM and what yesterday’s approval means for us now.
Yesterday’s Celgene press release proudly touts:
POMALYST® oral therapy comprises pomalidomide, an IMiDs® compound. POMALYST and other IMiDs compounds continue to be evaluated in over 100 clinical trials.
Some of those are trials designed to FINALLY get Revlimid approved for front-line use in newly diagnosed patients. Didn’t know it wasn’t? Once insurance companies and Medicare start to pay for specialists’ off-label use of a drug like Revlimid, expanded FDA approval becomes practically irrelevant–in this country. The only reason they are doing it is to gain approval by tougher international committees that control European, Japanese and Canadian markets.
There I go regressing again. Focus on POM, Pat, focus!
Several readers have already emailed me about POM, asking why the approval is such a big deal. After all, the primary study results sited by the FDA isn’t even a Phase III clinical trial. Fair enough! I was a bit puzzled by that too, since I have friends that have been taking part in a study at Mayo Clinic for well over four years–and from what I hear “on the inside,” the results are fantastic!
Another question is about the underwhelming study results in the Phase II trial Celgene did use. A 29% partial response rate with a progression free survival (PFS) rate less than 4 months? Previous study results showed percentages of 50% or higher when pomalidomide was used by patients that had previously used thalidomide or lenalidomide. What gives?
This approval is a big deal because outside of Kyprolis, POM is the first new myeloma drug to gain FDA approval of any kind since 2006. But let’s not start there. In an effort to keep my post at a reasonable length, let me sum things up and give my educated opinion about what’s really going on here.
The overall response rate in that study cited by the FDA was actually 45% in heavily pre- treated patients. And yes, the PFS was achingly short. But none of that is what’s important. Like many other myeloma drugs, POM works much better in combination than alone. And I’m not talking about simply with dex. Check out this table showing results of an international combo study from December’s ASH:
These percentages are from a European study featuring POM, Cytoxan and prednisone. And check-out those numbers! 67% partial response in patients that relapsed on Revlimid? Impressive!
Look, there are dozens of ongoing studies with better numbers than the one sited by the FDA. The best I can tell, this study must have met specific criteria the FDA wanted to see.
I have heard such prominent myeloma specialists as Dr. Richardson, Dr. Durie, Dr. Orlowski, Dr. Palumbo, Dr. Jagannath, Dr. Lacy, Dr. Dispenzieri and Dr. Rajkumar all share their feelings that POM is an improvement and a better drug than Thalomid or Revlimid. And everyone, including experts at the FDA, realize POM won’t be used alone. Combinations are king! And POM may well turn-out to be a key component in some of the most effective combination therapies ever developed very soon.
When Kyprolis was approved last year, that drug’s numbers in patient’s refractory to Velcade was only around 24%. POM’s numbers are better than that. But what have clinicians learned since? Giving Kyprolis at higher doses increases those numbers into the 40’s.
I will take the word of many of the World’s top hematologists when they express excitement over pomalidomide’s approval. Representatives from the IMF and MMRF seem ecstatic over the news. So are several patients I know who have successfully used the drug over the years, including marathoner Don Wright. I understand that POM isn’t a cure. But myeloma experts seem to concur its the best anti-myeloma drug we have so far.
Feel good and keep smiling! Pat