A health ministry panel approved Wednesday the production and sale in
The drug, Zolgensma, to be produced and sold by
The medication carries a price tag of
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
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
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
© Kyodo News International, Inc., source