A health ministry panel decided Friday to designate the sedative thalidomide as a priority research drug for bone marrow cancer treatment, more than 40 years after it was banned for causing severe birth defects.
Osaka-based Fujimoto Pharmaceutical Corp. filed a request last year for the ministry to designate thalidomide as what is technically known as an “orphan drug.”
Under the ministry’s definition, an orphan drug is a product used to treat rare but serious diseases that affect fewer than 50,000 patients. Any manufacturer of such a drug receives official backing in research and development, extended market monopoly status and tax breaks.
Fujimoto Pharmaceutical hopes to receive official approval to produce and sell the drug, which doctors have been importing in greater numbers to treat multiple myeloma and other ailments. The trend started rising in the 1990s after other countries approved limited use of the drug.
The Health, Labor and Welfare Ministry is likely to designate thalidomide as an orphan drug for treatment of multiple myeloma about a month after its pharmaceutical affairs panel issues an advisory on the matter, ministry sources said. Friday’s decision was made by a subcommittee of the panel.
The drug remains controversial. While groups representing those afflicted by multiple myeloma are pushing for approval, other groups supporting people who have suffered side effects are calling for regulations to prevent health problems caused by medicines.
Thalidomide was developed in what was then West Germany in 1954 and sold worldwide as a sedative and medicine to help digestion. Its use was stopped after a large number of deformed babies were born to pregnant women who had used the drug.
It was marketed in Japan from 1958 to 1962, when it was banned after more than 300 babies with attenuated limbs were born to women who took thalidomide in the early stages of pregnancy.
Since the late 1990s, some doctors have been importing the drug individually. Some 530,000 thalidomide tablets were imported in fiscal 2003 through March 31, 2004, according to industry insiders.
In December, the health ministry issued a guideline on thalidomide use — an unusual move for a banned medicine.
Midori Horinouchi, the head of a nationwide group of myeloma patients, welcomed Friday’s development and called on the ministry to promptly deal with the matter.
“We hope to see thalidomide reach those patients who have pinned their last hopes on it as swiftly as possible,” she said.
Kiyoshi Mamiya, head of the secretariat for a group of thalidomide side effect sufferers, acknowledged that ministry action was a step forward compared to the unrestricted flood of individual imports. However, he urged the government to draw up strict rules for using the substance.
In the United States, where thalidomide has been approved since 1998 to treat Hansen’s disease, distributors have developed a rigid system of prescription and distribution control under the guidance of the U.S. Food and Drug Administration.
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