The Unknown Patient Reports From Orlando
The Unknown Patient has been asked by many of his fellow myeloma fighters about when Revlimid (also known as Lenolidomide and CC-5013) will become available. Revlimid has been in the news recently because of successes in clinical trials for myeloma. It is said to work very well and have many fewer side effects than thalidomide. Revlimid could potentially offer options for people for whom other drugs, including thalidomide, have stopped working. It could also be an alternative for people who can’t tolerate the side effects of their current therapies.
Revlimid is a "thalidomide analog," meaning that its chemical formulation is based on thalidomide, but intended to remove the parts of the thalidomide molecule that cause that drug's most serious side effects (e.g., birth defects, neuropathy, and constipation) while retaining the parts that make it effective against myeloma.
Exciting results of randomized clinical trials of Revlimid conducted in the US and in Europe/Israel were presented at the 10th International Myeloma Workshop in Sydney, Australia and updated at the annual meeting of the American Society of Clinical Oncology this past weekend in Orlando. These trials were stopped because the results were conclusive that new therapy being tested (Revlimid plus dexamethasone) delivered better results than the control arm (dexamethasone alone.) These results will form the basis for submission of an NDA (new drug application) to the FDA (Food and Drug Administration) to seek approval of Revlimid for use in myeloma.
However, for those of you thinking that this sounds like Revlimid won’t be available for a long time, think again. There are a number of ways Revlimid could soon be available to those who need it.
First, the Unknown Patient spoke with a number of senior people from Celgene in Sydney and then in Orlando . They assured him that there is an "Expanded Access Program" (EAP) being put in place. The EAP protocol has been submitted to the FDA and has a 30 day turnaround time. Looking at what needs to happen if/when the FDA notifies Celegene about proceeding with the program, Celgene expects it will likely make Revlimid available on a limited basis via the EAP as early as mid-July. An EAP is a mechanism for making unapproved drugs with clear benefit available to patients who have a specific need before approval. EAP programs benefit patients by providing early access to drugs that have been shown to be safe and effective in clincical trials while the FDA is reviewing their application for approval. They are also used to gather more data on drug safety before the drug is introduced broadly into the market. Important details about this program (eligibility criteria, size of the program, firm roll out dates) remain Unknown but more information should be available soon.
Second, and more importantly, Celgene has already submitted its NDA to the FDA for use of Revlimid for treating Myelodysplastic Syndrome (MDS.) Revlimid had very positive results in trials for MDS and it seems to be headed for approval sometime late this year for treating this condition. In fact, there was more positive data on Revlimid for MDS presented at ASCO this past weekend. If Revlimid is approved for treating MDS, physicians then legally prescribed it for use in the US in myeloma. This is called "off label usage."
In reality, all use of thalidomide in the US today is "off label" as thalidomide has only been approved for treatment of leprosy. It has never been approved for myeloma. The application for thalidomide approval is currently being reviewed by the FDA. This past October, the FDA sent Celgene an “approvable letter” saying that the drug could be approved for myeloma if further data from a trial completed by the Eastern Cooperative Oncology Group were submitted to the FDA for review. The timing of this submission or the decision on thalidomide is unclear at this point.
So, it could be that Revlimid will follow a similar path of "off label" usage if it is approved for MDS. The difference is that Revlimid could be approved for myeloma within the year following the potential MDS approval date.
Bottom line, we can be hopeful that Revlimid will become available to myeloma patients on a limited basis this summer as part of an EAP, with broad availability sometime later this year if it is approved for MDS.
The Unknown Patient is sorry he had to use all that Unknown mumbo-jumbo to explain what's up with Revlimid. He hopes this report was understandable and helpful.