Experts in the health ministry issued preliminary approvals Wednesday for Collategene, a regenerative treatment for damaged arteries, as well as the anti-leukemia therapy Kymriah. The candidates next move to a formal certification, with drug prices to be set in May at the earliest.
AnGes, the Osaka-based developer that has worked on Collategene since its founding in 1999, would become the first Japanese company to receive approval for a treatment that regenerates blood vessels.
Collategene treats critical limb ischemia, which entails the severe blockage of arteries in the feet or legs. An estimated 150,000 Japanese annually are afflicted with this disease, which affects many diabetes patients and can lead to amputation. With Collategene, DNA injected into the patient forms new blood vessels to improve circulation.
Kymriah is a chimeric antigen receptor T-cell therapy developed by Swiss drugmaker Novartis. The immune-related T cells are removed from a patient, genetically modified to target cancer cells, then reintroduced to the body. The treatment was found to be effective in about 80% of young leukemia patients.
The global gene therapy market will reach $15 billion in 2024, British research firm Evaluate forecasts, amounting to 1% of the entire pharmaceutical market. So far, only about 10 gene therapy products have been approved worldwide.
But gene therapy is anticipated to grow 100% annually, with the U.S. expected to approve 10 new products yearly. Japan likely will approve more than one product each year. Governments worldwide are streamlining the approval process. Though novel drugs typically take 15 years before they are available for sale, this period would be truncated to just a few years for gene therapy.
Collategene and Kymriah are expected to qualify for coverage under Japan's national health insurance system. But like other revolutionary treatments, both will fetch exorbitant price tags.
In the U.S., Novartis has set a price of $475,000 for the one-time infusion of Kymriah, and the price in Japan likely will approach that sum. Collategene therapy will cost $18,000 to $27,000 per patient, multiple sources say.
The blockbuster cancer treatment Opdivo would cost the Japanese government 1.75 trillion yen ($15.8 billion at current rates) if used to treat 50,000 people in a year, according to a 2016 estimate. That figure prompted lawmakers to modify the drug pricing system, which resulted in Opdivo costing less than half as much as the original price tag.
But a rush of novel, expensive biopharmaceuticals could spark a more drastic overhaul of Japan's public health insurance system, including the general provision of covering all newly approved pharmaceutical products. For example, Britain rejected public coverage for cancer treatment Yescarta in August due to its expense. Japan likely will debate whether a public health system is rendered meaningless if certain drugs become out of reach for those of lower income.
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