US and Japanese regulators have reached different conclusions on a rare kidney disease drug produced by Otsuka Pharmaceutical Co. Otsuka is based out of Tokushima, Japan. US regulators rejected the drug for treatment, while it was approved by Japanese regulators.
The drug, Samsca, was developed in order to treat autosomal dominant polycystic kidney disease, or ADPKD. The disease affects patients by causing a swelling of the kidneys and which is extremely painful. In addition, the disease causes renal failure in about half of patients by age 70. The disease is genetic, and Otsuka claims that it affects a significant number of people in countries across the globe. Over 30,000 people in Japan, 200,000 in Europe, and 120,000 in the US are afflicted by ADPKD.
Otsuka claims that Samsca is the only drug which has shown potential at being able to slow the disease progression in patients. Otsuka has run clinical trials in 15 different countries. The trials were extensive, including over 1,400 patients. The studies concluded that Samsca significantly reduced the progression of the disease by slowing the development of cysts inside of the kidneys.
The FDA rejected the drug this past year, claiming that the company's clinical trials were inconclusive. Japan's Ministry of Health, Labor, and Welfare approved the drug earlier this week for treatment in Japan. The FDA had concerns about the results of the study, as well as the potential for a harmful buildup of enzymes in the liver. Samsca has already been approved in other countries and is currently sold in the US for the treatment of heart failure.