FDA Sets Date For Pomalidomide Advisory Committee Meeting
Celgene announced this morning that the U.S. Food and Drug Administration has set a date for an advisory committee meeting to review the company’s application for approval of pomalidomide.
Celgene (NASDAQ: CELG) has applied to the Food and Drug Administration (FDA) to have pomalidomide (Pomalyst) approved, in combination with dexamethasone(Decadron), for the treatment of patients with relapsed and refractory multiple myeloma that has progressed after at least two prior therapies.
The Oncologic Drugs Advisory Committee (ODAC), which advises the FDA regarding the potential approval of new cancer drugs, will meet on November 8 to review the pomalidomide application.
The FDA is not legally bound to follow the advice of its advisory committees regarding new drug applications, but it usually does. The agency is required to make a decision about pomalidomide’s application by February 10.
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Until the FDA reaches a decision regarding pomalidomide’s application, the drug is available through Celgene’s expanded access program, called PEXIUS, for relapsed and refractory myeloma patients in the U.S. who do not have any other treatment options.
Pomalidomide belongs to the same class of drugs as thalidomide (Thalomid) and Revlimid (lenalidomide) – two drugs that Celgene already markets as myeloma treatments in the U.S. and internationally. Celgene is developing pomalidomide as a potential new treatment for multiple myeloma and for myelofibrosis, a disorder of the bone marrow in which the marrow is replaced by scar (fibrous) tissue.
Celgene completed the FDA submission of pomalidomide’s new drug application in April (see related Beacon news).
In June, the FDA granted “standard review designation” to the application (see related Beacon news).
Although Celgene may have hoped that pomalidomide would be granted a so-called “priority review” by the FDA, the agency’s decision to do a standard review instead did not come as a surprise, given that Kyprolis (carfilzomib), another drug that the FDA recently approved for multiple myeloma, also was granted standard review (see related Beacon news).
Under the FDA’s standard review process, the agency must decide whether to approve a drug within 10 months of the time the drug is submitted for review.
If the FDA had granted priority review to pomalidomide, the agency would have needed to complete its review within six months. The FDA can grant priority review to drugs that offer significant advances in treatment, particularly for diseases in which there are inadequate treatment options.
Results from the latest clinical trials involving pomalidomide in relapsed/refractory multiple myeloma were presented at the American Society of Clinical Oncology annual meeting this summer (see related Beacon news).
Physicians as well as industry analysts have responded positively to the drug’s performance, particularly among patients previously treated with both Revlimid and Velcade (bortezomib).
If the FDA makes a positive decision regarding pomalidomide’s new drug application, Celgene will be allowed to start marketing the drug in the United States as a new treatment for relapsed and refractory myeloma.
A negative decision by the FDA would delay the launch of pomalidomide until Celgene is able to address any questions or concerns raised by the FDA.
If pomalidomide is approved by February, it will be the second new myeloma drug to be approved by the FDA within a year’s time. In July, the agency approved Kyprolis for the treatment of people with multiple myeloma who have received at least two prior therapies (see related Beacon news).
Celgene also submitted a marketing authorization application for pomalidomide to the European Medicines Agency this past June, meaning the drug could be approved for use in Europe by the middle of 2013.
For more information regarding the pomalidomide FDA advisory meeting in November, see the Celgene press release.
Related Articles:
- FDA Approves Once-Weekly Dosing And Revised Safety Information For Kyprolis
- ASCO 2018 Update – Expert Perspectives On The Key Multiple Myeloma-Related Oral Presentations
- Dr. Christoph Driessen On Nelfinavir In The Treatment Of Multiple Myeloma
- Latest Myeloma Research To Be Presented At The American Society Of Clinical Oncology Annual Meeting (ASCO 2018)
- ECT-001 Granted Regenerative Medicine Advanced Therapy (RMAT) Designation By U.S. FDA
To Myeloma Beacon staff;
do you know anything about approval of Kyprolis in Europe?
Hi Asaryden,
The only thing I found was from 2008:
Granted orphan designation in the EU (EU/3/08/548) June 2008
I do not know the rules for orphan drug status in the EU. That designation in the USA would mean it is available for those without any other therapeutic option.
I suspect the EU is waiting for results of other clinical trials in larger patient populations for greater approval.
Onyx expanded their 86 patient trial to 300 patients in Europe. The trial is called FOCUS.
Perhaps the Beacon staff has more recent data/changes for EU since 2008.
Suzie:
IMO the more exciting drug, although further away from FDA consideration, is Daratumumab. Have you looked at the results of the phase I/II trial? Ridiculous response rates at the higher doses for people that are very heavily pre-treated (from what I remember, median 5+ lines of therapy including Velcade and an IMiD). I was excited to hear that J&J purchased it as they have the resources to move it along ASAP.
Hi Ron!
I was going to respond to you on the daratumumab thread, but it looks like comments are closed.
I agree with you the dose ranging trial sounds impressive in terms of the response rates in RR patients.
These numbers are remarkable:
"Of the three patients who received 2 mg/kg of daratumumab, one patient experienced a 67 percent reduction of monoclonal proteins in the blood and a 55 percent reduction of monoclonal proteins in the urine.
All three of the patients who received 4 mg/kg of daratumumab experienced reductions in their blood monoclonal protein levels by 49 percent, 64 percent, and 100 percent, respectively. These patients also experienced a significant reduction in the percentage of plasma cells in the bone marrow (80 percent, 89 percent, and 97 percent).
Similarly, all three of the patients who received 8 mg/kg of daratumumab also experienced reductions in their blood monoclonal protein levels by 4 percent, 39 percent, and 100 percent, respectively.
Two out of three patients who received 16 mg/kg of daratumumab experienced reductions in their monoclonal protein levels. One patient had a 50 percent reduction of blood monoclonal proteins, and the other patient had a 33 percent reduction of urine monoclonal proteins."
The thing that is concerning though is are these events:
"Fourteen percent of patients experienced severe side effects, including low red blood cell and platelet counts, abnormal liver function tests, spasms of the airways making breathing difficult, and an inflammatory syndrome."
Given that there were only 29 patients the 14% sounds high for serious grade 3/4 events. The airway spasms is very concerning..we know how to manage the hematological effects well, but not so confident about the breathing difficulty and inflammatory syndrome. I wonder if those patients had to discontinue therapy. I would be very apprehensive if I experienced the difficulty breathing. I wonder if higher doses of dex might have diminished the inflammatory response and if the grade 3/4 events were seen at all doses or just the higher doses.
I am not sure that the benefits of therapy outweigh the risks given the type of AE's that have been seen thus far with daratumumab in this dose-ranging trial.
It is great though that we are seeing more and more therapeutic choices for myeloma, especially for RR patients.
asaryden,
Onyx intends to file for approval of Kyprolis in Europe based on results of the Phase 3 ASPIRE trial. Initial results are expected in early 2013 and full results in 2014.
More information about this can be found in the last section of the following article:
Questions And Answers About The FDA’s Approval Of Kyprolis
http://www.myelomabeacon.com/news/2012/07/25/questions-and-answers-about-the-fdas-approval-of-kyprolis-carfilzomib/
suzierose,
You can read more about pomalidomide's efficacy and safety in the following article:
Updated Pomalidomide Clinical Trial Results Presented (ASCO 2012)
http://www.myelomabeacon.com/news/2012/06/25/updated-pomalidomide-clinical-trial-results-presented-asco-2012/
The following information may answer some of your questions:
“Pomalidomide has been tested in multiple clinical trials … and responses were between 30 percent to up to 65 percent when patients had one to three prior lines of therapy. Most interestingly, this agent is showing responses even in patients who are [resistant to] Velcade or Revlimid,” said Dr. Irene Ghobrial from the Dana Farber Cancer Institute in Boston, who presented a review of some of the pomalidomide results at ASCO.
“Now we have a new immunomodulatory agent that will be added to [our available treatments] for myeloma. This is important for patients who are Velcade and Revlimid refractory. As myeloma patients live longer and longer… they need new agents of therapy, and pomalidomide may be one of those agents,” added Dr. Ghobrial.
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