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08.08.19 8 FOREIGN NEWS MORE than two months after the Food and Drug Administration approved an expensive new gene therapy drug for a rare disease in children, the agency said it has major questions about the accu- racy of some of the testing data. e FDA said Tuesday that the drug maker — AveXis, which is owned by Novartis — was aware of problems with the data and didn't inform the agency until after the drug was approved. e drug Zolgensma was approved in May to treat spinal muscular atrophy in children two and under. Children with the genetic mutation, which kills nerve cells in the spine, lose their ability to walk, eat and breathe. It affects about one in every 11,000 babies, and many die before turning two. e new drug, a type of gene therapy that fixes the mutation, has given hope to parents who often have few other op- tions. With a price tag of $2.1 million, it is the most expensive drug in the world — the cost has been a major roadblock for patients who can't get insurance to cover it. Dr Peter Marks, director of the FDA's Center for Biologics Evaluation and Re- search, said Tuesday in a statement that AveXis manipulated some of the early data from testing in animals. at data was submitted as part of the drug's ap- plication for approval. "Ensuring truthful, complete and ac- curate data in product applications is a critical component of industry's respon- sibility as they work to demonstrate the safety, purity and potency of biological products," Marks said. "e submission of such truthful, complete and accurate data is also critical for the FDA to be able to protect the public health, and the law requires it." e FDA stopped short of pulling ap- proval for the drug, saying that the agen- cy "remains confident that Zolgensma should remain on the market," and that all testing done in humans showed that the drug was safe and effective. e agency hasn't ruled out possible crim- inal or civil action against AveXis, ac- cording to the FDA statement. "e idea that Novartis would go for approval of the most expensive drug in the history of the world and do it with data it knew to be inaccurate is an out- rage and it could put patients in jeop- ardy," David Mitchell, a cancer patient and founder of the advocacy group Patients For Affordable Drugs said via email. One drug development expert said the case is unusual, but there should be no cause for alarm for patients. "If I'm a parent, my major concern is the health of my child, and the FDA is reassuring me," Kenneth Kaitin, direc- tor of the Tufts Center for the Study of Drug Development, said. "e FDA is being transparent about it, and it has no impact on the value of that drug in treating a child." FDA raises accuracy questions over trial data of gene therapy drug Mastercard buys a £2.6bn stake in Danish payments firm Nets in its biggest ever aquisition PAYMENTS giant Mas- tercard has made its big- gest ever acquisition, buying a stake in a Danish rival for £2.6billion. e firm said it is snap- ping up part of payments firm Nets in a push to of- fer faster payments. e deal will see Mas- tercard absorb Nets' clearing, electronic billing and instant payment busi- nesses, which are part of the Denmark-based com- pany's corporate arm. It comes after the American giant acquired UK-based Vocalink, formerly owned by a group of 18 banks, for £700million in 2017. Michael Miebach, chief product and innovation officer at Mastercard, said: 'is deal strength- ens our unique position as the one-stop partner for any bank, merchant or government's payment needs.' Bo Nilsson, group chief executive of Nets, said: 'With its resources and global reach, Mastercard is uniquely positioned to unlock the potential of Nets' account-to-account business.' Mastercard admitted paying for the deal will hit profits for up to two years after it completes. Nets employs 3,500 staff across the group. e FDA stopped short of pulling approval of Zolgensma, which treats children with spinal muscular atrophy

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