The dust is beginning to settle after yesterday’s unanimous recommendation by the FDA’s Advisory Committee to fast track Kyprolis (carfilzomib) for FDA approval for use in refractory multiple myeloma patients.
Honestly, I think the FDA’s strong support of the drug caught the folks at Onyx a bit off-guard. I know they felt the drug had a real shot at approval this summer. But now, barring any unexpected data or off-the-wall controversy, approval seems all but certain.
I excitedly reached-out to Danielle Bertrand, Senior Manager of Public Affairs at Onyx Pharmaceuticals
, after I returned home from my monthly support group meeting. It was late and I’m sure she–and the entire Onyx team–were exhausted.
Still, I was disappointed by her response:
Danielle, on behalf of multiple myeloma patients everywhere, I would like to thank you and everyone at Onyx for their persistence and hard work developing Kyprolis. I know it hasn’t been easy!
My question is this: If and when Kyprolis gains fast-track approval sometime in July, how long do you expect it will take for the drug to be available for distribution? Will it only be available at larger cancer centers, or local oncology offices too?
Her answer was short and very vague, to say the least:
“If the FDA approves Kyprolis, we will be prepared for launch shortly afterwards and are committed to bringing it to patients as soon as possible.
That’s it? I emailed her one simple question, knowing I would be gone until late…
To be fair, I’m sure the Onyx team still doesn’t want to “jinx” their application. After all, this vote was only advisory. Official FDA Fast-Track approval won’t come until sometime in July.
But I have met a number of high ranking Onyx officials over the past three years. And I can tell you this with confidence: They will be ready to launch and distribute product FAST.
So let’s back-up and I’ll share the official press releases that Onyx released yesterday about the vote:
Company to Work with FDA toward PDUFA Date of July 27, 2012
SOUTH SAN FRANCISCO, Calif., June 20, 2012 /PRNewswire/ — Onyx Pharmaceuticals, Inc. (Nasdaq: ONXX) today announced that the U.S. Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) determined by a vote of 11-0 [with 1 abstention] that, in patients with relapsed and refractory multiple myeloma who have received at least two prior lines of therapy that included a proteasome inhibitor and an immunomodulatory agent (IMiD), the benefit-risk assessment is favorable for the use of Kyprolis™ (proposed brand name for carfilzomib). Onyx is developing Kyprolis for use in multiple myeloma across a variety of treatment lines.
“Today’s ODAC recommendation is an important regulatory milestone in the review of Kyprolis for relapsed and refractory multiple myeloma,” said Ted W. Love, M.D., Executive Vice President, Research and Development and Technical Operations at Onyx Pharmaceuticals. “Onyx is committed to bringing Kyprolis to patients as quickly as possible and looks forward to working closely with the FDA as the agency completes its review.”
The Prescription Drug User Fee Act (PDUFA) date for completion of FDA review of the Kyprolis NDA for accelerated approval is July 27, 2012. The ODAC provides FDA with independent expert advice and recommendations, however the final decision regarding approval is made by FDA.
The Kyprolis NDA is based on the 003-A1 study, an open-label, single-arm Phase 2b trial as well as supportive data from additional studies. The 003-A1 trial evaluated 266 heavily-pretreated patients with relapsed and refractory multiple myeloma who had received at least two prior therapies, including bortezomib and either thalidomide or lenalidomide.
So there you have it. For a company that has always been aggressive and pushed-the-envelope–For example, picking a trade name prior to FDA approval, a very confident move–I was a bit surprised that Danielle kept her comment so brief and close-to-the-vest.
Guess we are going to have to wait one more month for the answer to my question, which I will ask again!
This is good news for patients who are running out of treatment options–at least 25% of them.
Reviewing these exciting–yet disappointing–patient response numbers reminds me that I never ran the third installment of my three part series:
Immunotherapy: The good, the bad and the ugly. Part Three: The Ugly.
I will post that first thing tomorrow. Here’s a hint: It’s all about the money.
Feel good and keep smiling! Pat