Zolgensma is the world’s most expensive drug priced at more than $2 million. AveXis, the gene therapy subsidiary of Swiss pharma giant Novartis, was aware of “data manipulation” involving its Zolgensma gene therapy for spinal muscular atrophy before it was approved in May, but did not inform the Food and Drug Administration until later, the agency said Tuesday. “With the creation of Novartis Gene Therapies, we will continue to advance our gene therapy pipeline for rare genetic diseases, to accelerate the delivery of transformative innovation in areas of high unmet need, and to reimagine medicine for patients all around the world,” Narasimhan said in a Friday news release. The company acknowledged it had the information earlier, but said it was completing its own internal investigation before reporting to the FDA. Since 2013, Novartis Gene Therapies (formerly AveXis) has had one focus: bringing change to those devastated by genetic diseases. All rights reserved. The AveXis controversy began shortly after the FDA approved Zolgensma in May 2018. (Novartis). But it wasn't the data problem itself that spun the incident into scandal. Want an ad-free experience?Subscribe to Independent Premium. Two months after that green light, Novartis reported that some of the data in its FDA application were inaccurate. Novartis says it is the most widely used gene therapy in the world. In other businesses, data manipulation gets people fired, and opens the can of worms for very stiff fines / lawsuits / jail-terms / de-licensing, etc. Brian Kaspar, previously chief scientific officer of AveXis, the biotech company that developed Novartis's gene therapy, Zolgensma, "is a respected member of … An AveXis scientist had told Novartis about the faked data before the drug’s approval, but the company didn't notify the FDA until weeks later. Reproduction in whole or part is prohibited. AveXis receives positive CHMP opinion for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA) ... AveXis headquarters is located at 2275 Half Day Rd #160, Bannockburn. But is the deal bad news for Biogen (NASDAQ:BIIB)? RELATED: Novartis' AveXis opens Zolgensma plant in Colorado with 350 employees. exclude terms. This decision was based partly on the success of Zolgensma, originally developed by AveXis, as a gene therapy for spinal muscular atrophy (SMA) that is approved in the U.S., Japan, Europe, and … Call it a two for one: By changing the name of its AveXis unit to Novartis Gene Therapies, the Swiss drugmaker highlights the importance of gene therapies to its future hopes while nixing a moniker linked to last year's Zolgensma data scandal. With new AveXis name, Novartis spotlights marquee role for gene therapy business, FDA lets Novartis off the hook in Zolgensma data manipulation, Novartis' AveXis opens Zolgensma plant in Colorado with 350 employees, Cytiva joins COVID-19 effort with $31M US government grant. A great fit. Two years after acquiring the gene therapy company AveXis, Novartis has renamed it Novartis Gene Therapies to underscore the potential value of developing such treatments for genetic diseases.. Zolgensma, Novartis' signature gene therapy treatment, is the cornerstone for the newly re-named Novartis Gene Therapies. To 90 % off Computer and Console games ( NASDAQ: BIIB?... Children via lottery that some of the data problem itself that spun the incident into scandal buyout was great! National biotech columnist, reporting on the intersection of biotech and Wall Street its new owner ). Change to those devastated by genetic diseases in Zolgensma data manipulation vaccine manufacturing partner BioSolutions. 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