The FDA made the data problem public earlier this week. It appears to affect only a small amount of data, and the FDA said it "remains confident" the drug, Zolgensma, should still be sold. In the pharmaceutical industry, where data is the basis for approval of life-or-death drugs, data manipulation is serious business. In addition to Warren and Sanders, both presidential hopefuls, the letter was also signed by Sens.
The timeline of Novartis' disclosure to the FDA also inspired ire from the senators. Novartis heard allegations of data manipulation in March, before Zolgensma was approved in May. In a conference call with investors this week, Novartis CEO Vas Narasimhan said the company had been investigating the allegations itself and denied that it had been influenced by the timing of Zolgensma's approval.
The data manipulation was carried out by only a few people, and Novartis would "exit" them, he said. The drugmaker "tried to do all of the right things," Narasimhan said.
But the senators disagreed, calling Novartis's actions "unconscionable" and urging the FDA to take action. The regulator has previously said it could impose either civil or criminal penalties. The senators also asked the FDA why it had scrapped a regulation proposed last fall, which would have required healthcare companies to quickly report falsified dataand whether that regulation would be reissued because of the AveXis news.
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Emma Court. Elizabeth Warren and Bernie Sanders are among a group of five senators who slammed Novartis' AveXis for submitting manipulated data to the FDA ahead of the approval of its gene-therapy drug. The FDA revealed the data-manipulation issue earlier this week, saying that it affected only a small portion of product-testing data and that the regulator was confident the drug, Zolgensma, should keep being sold. Novartis said it first learned about the data problem in March.
The five senators encouraged the FDA to "use your full authorities to hold AveXis accountable for its malfeasance," adding that "anything short of a forceful response would signal a green light to future pharmaceutical misbehavior.
Novartis AveXis gene therapy Politics Strategy pharma.The pandemic may have altered the course of life as we know it, for awhile, but the FDA wants you to know that it is still raring to go when it comes to stamping accelerated approvals on rare disease therapies headed to patients with no existing meds to pick from.
By compiling resources, leveraging existing networks, lobbying regulators and setting up a steering committee to prioritize promising candidates, the goal is to generate more comprehensive data quicker. Thursday night saw perhaps the most vivid and dramatic illustration to date.
Of patients recruited at the University of Chicago Medicine, had severe disease and most of them have been discharged. Two deaths were recorded. Without any public demands for a change. Coronavirus changed all that — perhaps forever. And that is playing a major role in accelerating the work of a small group of vaccine revolutionaries that includes Moderna — even though they have yet to prove in a pivotal study that their technology is both safe and effective.
Charles Goetz, who studied entrepreneurship, suggested GoFundMe. What appeared online three months later had the smoothness of a VC pitch and the commanding lilt of a call to arms. That was the week the coronavirus moved from far-flung threat to looming menace for many Americans, when Vice President Pence was named to head the White House taskforce, the stock market suffered its worst fall since and the first cases of unknown origin appeared on the West Coast — a sign the virus was spreading within the US.
They promised a tantalizing antidote. Five years after turning his stake in Pharmacyclics into a personal fortune worth more than a billion dollars, Bob Duggan is back at the helm of a biotech.
I assure you that I did not plan to do this. I wanted to write a little story about proteins and cancer, nuclear magnetic imaging and a scientist turned entrepeneur of billion-dollar biotechs. Maybe a touch on communist East Germany. A sprinkle on her what I assumed had been past life as a world-class athlete.
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Please note this link is one-time use only and is valid for only 24 hours. Jason Mast. Jason Mast Associate Editor jason endpointsnews. Jason Mast on LinkedIn. John Carroll.Last month, the U. Food and Drug Administration said Novartis could face civil or criminal penalties because of the data manipulation and the possibility the company had waited to notify regulators.
They were written by the company in late August and released by the regulator on Tuesday. Kaspar cooperated with the company investigation.
Then and now, Dr. Kaspar has appropriately and categorically denied all wrongdoing. The FDA said in August that Novartis had flagged the manipulation to regulators in June, a month after Zolgensma had been approved and several months after a whistleblower told the company about the data manipulation.
The company has also committed to letting the FDA know within five business days if it receives any credible allegation related to data integrity impacting any of its pending drug applications. The disease often leads to paralysis, breathing difficulty and death within months for babies born with the most serious Type I form. Discover Thomson Reuters.
Directory of sites. United States. Business News. Michael Erman.Investors should be aware of risks posed by competitive pressures, generic erosion, and FDA recalls. Novartis is a big pharma company increasingly focused on high growth areas such as rare diseases, gene therapy, and oncology. The company is also targeting the large underserved markets of psoriasis and multiple sclerosis. With new management at the helm, the company is aggressively trying to become more focused and nimbler like a biotech company.
Novartis completed five commercial launches inwhich is a feat even for a biotech company and much more so for a big pharma company.
Finally, the company pays healthy dividends to its shareholders. Novartis can thus offer an appealing mix of value, growth, and income to retail investors. The company's pipeline includes 16 advanced platform therapies. Novartis' Innovative Medicines segment is its biggest growth driver.
The company is guiding for mid 30s core margin for this segment in the short term. In the medium term, the company expects mid to high 30s core margin for this segment. To that effect, the company is consolidating its manufacturing footprint by focusing on high-end technologies. The company is also optimizing its employee count as well as retail footprint across geographies.
Forthe company has guided for mid to high single digits revenue growth, excluding Sandoz US oral solids and dermatology business. The company expects core operating income to grow YoY by a high single to a low double-digit percentage. The company has also guided for mid to high single-digit revenue growth for the Innovative Medicines segment and low single-digit revenue growth for Sandoz division in This is mainly driven by robust uptake in dermatology and rheumatology indications.
The company expects penetration in China and Japan in Lutathera, Kymriah, and Kisqali have also emerged as potent growth drivers. InNovartis reported five NME new medical entity approvals of potential blockbuster therapies. Mayzent is approved as the first oral drug to treat secondary progressive MS with active disease.
Adakveo is approved by the FDA for the reduction of frequency of vaso-occlusive crises or VOCs in sickle cell disease. There are more than 54, patients suffering from sickle cell disease and having more than one VOC per year in the U. The commercial product became available within two days of approval in November Novartis is now actively engaging with payers working through reimbursement.
The company has already secured initial orders for the product from community centers and anticipates permanent J-Code.In fact, with certain exceptions, the FDA is the only regulatory agency for drugs and devices that independently evaluates both the science and the source data from relevant studies. In many ways, the FDA serves the world as an arbiter of quality in the global development of medicines.
But based on those reports it seems that Novartis discovered an issue relating to data integrity while preparing its Biologics License Application BLA submission for marketing approval for Zolgensma.
According to public statements from both Novartis and the FDANovartis followed its usual internal procedure and the FDA is confident that the data integrity issues did not concern clinical trials conducted with human volunteers.
The overwhelming majority of scientists, clinical investigators, and employees of corporate and academic research organizations are highly motivated by the mission of improving health and conduct impeccable research.
That said, preclinical and clinical research are complex. Mistakes, sometimes quite subtle ones, are common in study design and conduct, as well as in the analysis and reporting of findings. Purposeful manipulation or falsification of data and analyses, on the other hand, is much less common. Nevertheless, the complexity and level of detail needed makes it relatively easy for inaccurate work to pass muster among non-experts, and for the smaller fraction of purposeful data manipulators or fabricators, detecting such malfeasance requires expertise and dedicated effort.
Working to create a cure for a frequently lethal disease in children is a noble undertaking. What then could motivate people involved in such an effort to produce faulty data and reports? Obviously, the enormous financial rewards that accompany a successful therapy can affect behavior. But within the industry, more complex incentives are in play. The capacity to monitor costs at every step of medical product development is much more refined than the capacity to monitor the quality and design of the development program.
Project teams are also under unremitting pressure to cut timelines, with significant rewards handed out for reaching a milestone early, and punishments for delays. Medical product development is an expensive, high-risk undertaking, and adhering to timelines and milestones is essential.
Yet monitoring quality is also critically important. The most pervasive risk in drug development is not so much outright falsification as it is the acceptance of inadequate data or inserting bias into the analysis. For example, it is well known that if experiments with contrary results are not included in the overall assessment or outlying data points are excluded from analysis, the interpretation can be biased. Duke behavioral scientist Dan Ariely has made the argument that while we tend to focus on egregious cases, the much bigger issue is the less sensational but far more common acceptance of sloppiness, bias, or manipulation in data collection and analysis.The therapy, Zolgensma, is a one-time treatment for spinal muscular atrophy, a muscle-wasting disease and leading genetic cause of infant mortality that affects one in every 11, births.
The company said it's "working closely with insurers to create 5-year agreements based on success of the treatment as well as other novel pay-over-time options. This marks a new era in medicine where new therapies can cure patients in a single treatment — but at a high price.
Insurers and governments will need to figure out how to pay for these therapies and society will need to decide whether any drug, even lifesaving ones, are worth millions of dollars. Acting FDA Commissioner Ned Sharpless lauded the approval, saying in a statement that it marked "another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases. With each new approval, we see this exciting area of science continue to move beyond the concept phase into reality.
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We want to hear from you. Get In Touch. CNBC Newsletters.One of two top scientists said to be ousted from Swiss drug giant Novartis in connection with a data manipulation scandal is denying any wrongdoing. Brian Kaspar, previously chief scientific officer of AveXis, the biotech company that developed Novartis's gene therapy, Zolgensma, "is a respected member of the scientific community with an impeccable record," his attorney, John Hueston of Hueston Hennigan, said in an e-mailed statement.
Novartis said last week that Kaspar, along with his brother, Allan Kaspar, who led research and development at AveXis, hadn't been involved with the company since May. Though the company didn't say so explicitly, a person familiar with the matter said last week their departure was related to the issue of manipulated data that supported Zolgensma's approval by the Food and Drug Administration.
The report by the FDA earlier this month that Novartis not only relied on manipulated data in the drug's application, but did so knowingly, has put pressure on its chief executive, Vas Narasimhan. In a conference call with analysts, Narasimhan said Novartis waited to alert the agency about the data manipulation until it had completed its own investigation — about a month after the drug was approved. Both the FDA and Novartis said the data in question didn't concern the drug's safety and efficacy, and that Zolgensma should remain on the market.
The Kaspars were among a handful of scientists who were fired in connection with the data manipulation, said the person, who declined to be named because an investigation into the situation is ongoing. It's considered a breakthrough in the treatment of spinal muscular atrophy, a rare and devastating childhood disease. The FDA has threatened potential civil or criminal penalties against the company. Kaspar "stands proudly behind the safety and efficacy of the drug that he and his team worked so hard to develop," the statement from his attorney said.
He is prepared to assert his rights and defend his conduct accordingly. Kaspar continues to wish the clinicians and the company continued success in the treatment of children with this deadly and devastating disease. Hueston was also lead prosecutor in the case against Enron executives Kenneth Lay and Jeffrey Skilling, in which both men were convicted of securities and wire fraud.
Hueston said he does not represent Kaspar's brother, Allan, who has not responded to a message seeking comment. Sign up for free newsletters and get more CNBC delivered to your inbox.
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Novartis Reports Q4 2019 Financial Results, Zolgensma Sales Fall Short
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