Evomela Approved as Pre-transplant Conditioning Therapy for Multiple Myeloma Patients in China

Evomela, an injectable formulation of melphalan, is now available in China as a high-dose conditioning therapy for people with multiple myeloma who will undergo a stem cell transplant, CASI Pharmaceuticals, its maker, announced.

The product is also approved as a palliative treatment for patients who cannot take oral medicines. Palliative care is meant to provide relief from disease symptoms and stress, improving quality of life.

CASI submitted a priority review application to the China National Medical Products Administration (formerly known as the China Food and Drug Administration) for Evomela in multiple myeloma in April 2018. Evomela is now the only formulation of melphalan approved for use in China.

Autologous stem cell transplant can improve remission rates and prolong overall survival for people with multiple myeloma. The procedure consists of extracting blood or bone marrow stem cells from a patient, then treating the person with high doses of chemotherapy or radiotherapy to kill the cancer cells, and injecting the extracted cells to serve as a founding, cancer-free cell population.

Melphalan — a chemotherapeutic agent used in several types of cancers — is the preferred agent for multiple myeloma patients before an autologous stem cell transplant.

Evomela does not contain propylene glycol, a chemical compound with significant side effects, unlike melphalan formulations previously approved in the U.S.

The therapy uses Ligand Pharmaceuticals’ Captisol technology. Captisol is a drug-delivery system based on modified sugars, called cyclodextrins, that optimizes the solubility and stability of different medicines. Through Captisol, Evomela is stable for up to four hours at room temperature.

Evomela’s approval could help meet the demand for autologous stem cell transplants among multiple myeloma patients in China. Around 16,900 multiple myeloma patients there are thought to be eligible for autologous stem cell transplants each year, and about 800 undergo the procedure every year.

Evomela, distributed by Spectrum Pharmaceuticals, has been commercially available in the U.S since 2016, when the U.S. Food and Drug Administration (FDA) approved it as a conditioning treatment for patients undergoing a stem cell transplant and as a palliative treatment for those who cannot use oral therapies. The FDA also designated it an orphan drug.