Ever since Carfilzomib, made by ONYX Pharmaceuticals, began progressing through the clinical trials process from Phase I forward, it has been a regular topic of interest on the IMF Hotline. The recent announcement that Carfilzomib is being made available to eligible patients through an early-access program has attracted a lot of people's attention. Until a few days ago, Carfilzomib was a drug in Phase III clinical trials not expected to be approved for at least another six months to a year.
For those who are unsure what Carfilzomib is, it's a late-stage, selective next-generation proteasome inhibitor. It is being studied as a single-agent treatment and in conjunction with REVLIMID. At the recent International Myeloma Workshop in Paris, we noted that in multiple clinical studies carfilzomib has a "positive efficacy signal" with an encouraging safety profile, including low rates of neuropathy.
The results obtained with Carfilzomib during this expanded access program will be available to support the approval of the drug for prescription use. The data will be compared to findings in a new, recently published, IMF study, which provides a benchmark for patients with relapse/ refractory myeloma. This study clearly demonstrates the need for more new drugs to treat myeloma.If you have any questions about this new development, the IMF's toll-free Hotline (800-452-2873) is available to help you understand the eligibility requirements and provide contact information for the early access program. This program may have opened a new door for patients that have exhausted all other options, but it isn't necessarily the best course of treatment for everyone. I urge any patient who is interested in getting into this early access program to discuss it with their own doctor before making any decisions.