A health ministry panel approved Wednesday the production and sale in Japan of a medication thought to be the most expensive in the world that is used to treat a rare childhood genetic disorder.

The drug, Zolgensma, to be produced and sold by Novartis Pharma K.K., the Tokyo-based unit of Swiss pharmaceutical giant Novartis AG, will get the official green light from the Ministry of Health, Labor and Welfare in March.

The medication carries a price tag of $2.1 million in the United States, where it was approved by the Food and Drug Administration in May last year.

It is a one-time therapy for spinal muscular atrophy, a genetic disorder that causes motor neuron loss and muscle wasting. The disorder affects about two infants out of every 100,000 and can lead to severe respiratory problems and early death.

It will be the second gene therapy drug approved in Japan. Once officially approved, the medication would be covered by national health insurance as early as May.

The drug will be used to treat children less than two years of age and Novartis calculates it will be administered to 15 to 20 individuals per year in Japan.

With side effects including heart damage and liver problems reported during clinical testing, the company will observe patients' health and check the drug's safety and effectiveness.

Following the revelation last year of data manipulation during animal testing, the health ministry said it had examined other experimental data to confirm the safety of the medication.

==Kyodo

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