Kyprolis (Carfilzomib)

Kyprolis (carfilzomib) is approved by the U.S. Food and Drug Administration (FDA) to treat refractory myeloma. The intravenous infusion treatment was initially licensed to Proteolix, which was acquired by Onyx Pharmaceuticals. Onyx was subsequently bought by Amgen.

What is myeloma?

Myeloma is a cancer that affects certain types of immune cells. All immune cells are made in the bone marrow. In the case of myeloma, immune cells called B-cells start to grow and divide uncontrollably. These myeloma cells can appear in different bones and spread from one bone to another, which is why the disease is sometimes called multiple myeloma.

The myeloma cells produce abnormal antibodies, which interfere with immune function and cause many of the disease’s symptoms.

How does Kyprolis work?

In normal cells, proteins are recycled by cellular machines called proteasomes. These proteasomes digest misfolded or worn-out proteins, breaking them into their amino acids (the building blocks of proteins), which are subsequently used to make new proteins.

Kyprolis contains a small molecule (carfilzomib), which binds irreversibly to proteasomes, preventing them from breaking down proteins. This causes proteins to build up inside cells, poisoning them. Fast-growing cells like myeloma cells are more prone to the effects of carfilzomib.

Kyprolis in clinical research

The approval of Kyprolis was based on a Phase 3 clinical trial (NCT02412878) in 478 people with relapsed or refractory myeloma. These are patients who failed to respond to at least two, but no more than three, other therapies, including Velcade (bortezomib) and an immunomodulatory treatment. They were randomly assigned to either Kyprolis once or twice a week in combination with dexamethasone (to reduce side effects).

Interim trial data were presented during an oral session at the 54th Annual Meeting of the American Society of Clinical Oncology, and simultaneously published in The Lancet Oncology.

The study’s primary goal was improvements in progression-free survival — the time from the beginning of treatment to disease progression or death. Secondary endpoints were overall response rate, safety, and tolerability of the treatment.

Patients treated once-weekly with Kyprolis showed a significant improvement in progression-free survival: 11.2 months compared to 7.6 months with twice-weekly Kyprolis treatment. In addition, 7.1% of patients had complete responses in the once-weekly treatment group compared to 1.7% in the twice-weekly group.

Kyprolis is still being studied in a number of ongoing clinical trials.

Other information

Kyprolis can cause a number of side effects, including fatigue, anemia, nausea, thrombocytopenia (low platelet counts), difficulty breathing, and fever.


Last updated: Nov. 12, 2019


Myeloma Research News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.