Melphalan flufenamide (trade name Alkeran) is a chemotherapy drug used to treat multiple myeloma and certain other cancers, including ovarian cancer and brain tumors. It is a combination of two drugs: melphalan, an alkylating agent, and flufenamide, an antimetabolite. Melphalan flufenamide works by interfering with the growth of cancer cells, which eventually die. Common side effects of the drug include nausea, vomiting, hair loss, and fatigue.
Melphalan flufenamide, also known by brand names Pepaxto and Pepaxti, was a medication intended to treat multiple myeloma, but it was withdrawn from the market in October 2021. Here's a breakdown of its history and uses:
Intended Use:
- Targeted for multiple myeloma: Melphalan flufenamide was designed to treat adults with relapsed or refractory multiple myeloma. This refers to multiple myeloma that has come back after treatment (relapsed) or doesn't respond well to initial treatment (refractory).
Mechanism of Action:
- Combining effects: Melphalan flufenamide was a prodrug, meaning it needed conversion into its active form inside the body. It combined two components:
- Melphalan: An alkylating agent that disrupts cancer cell DNA replication.
- Flufenamic acid: Aided in delivering melphalan into cancer cells.
Withdrawal from Market:
- Phase 3 trial results: Melphalan flufenamide's withdrawal stemmed from the results of a key clinical trial called OCEAN. This trial compared melphalan flufenamide with another standard treatment for multiple myeloma. The trial showed that melphalan flufenamide did not improve overall survival compared to the control group.
Current Status:
- No longer available: Due to the lack of improvement in overall survival and following discussions with the FDA, the manufacturer withdrew melphalan flufenamide from the market in October 2021.
Alternative Treatments:
- If you have been diagnosed with multiple myeloma, consult your doctor about alternative treatment options. There are various established treatment approaches for this condition.