Miracle drug for rare diseases: How effective is ixazomib?

Ixazomib was investigated in a major study involving 722 adults with multiple myeloma (MM) whose disease did not improve or relapsed after prior treatment. The study compared ixazomib to placebo (a dummy treatment), both taken with lenalidomide and dexamethasone. The first analysis of the data showed that ixazomib was effective in extending the time patients lived without their disease getting worse (progression-free survival): patients treated with ixazomib lived an average of 21 months, compared with an average of 15 months for those treated with placebo. However, there is uncertainty about the extent of the improvement, as further analysis of the data showed a reduced effect.

In a subsequent similar study involving 115 patients, many of whom had advanced disease, patients who received ixazomib and lenalidomide and dexamethasone lived an average of 6.7 months without disease progression, compared with an average of 4 months for patients who received placebo and lenalidomide and dexamethasone.

The original drug of Ixazomib has been launched in China and is included in Class B medical insurance. The price of common capsule preparations may be more than 10,000 yuan per box, which is relatively expensive. The price of the original drug of ixazomib sold overseas is more than 10,000 US dollars per box (the price may fluctuate due to the exchange rate). Ixazomib Generic drugs are also produced and sold overseas, and their drug ingredients are basically the same as those of the original drugs sold domestically and abroad. For example, the price of 4mg*3 tablets produced by a Laos pharmaceutical factory may be less than 1,000 yuan per box (the price may fluctuate due to the exchange rate), and the price is relatively cheap.