Novartis’s data scandal is over
Last year, when the FDA first chastised Novartis for submitting falsified data on its gene therapy, the agency threatened “civil or criminal charges.” As of this week, the investigation is over, and there will be no penalties.
As STAT’s Ed Silverman reports, the FDA instructed Novartis to correct the problem, which arose from its AveXis division, but chose not to issue any punishment. The problem, you may recall, involved preclinical data for Novartis’s Zolgensma, approved to treat spinal muscular atrophy.
At the time of the controversy, the company fired two AveXis leaders who “either manipulated or pressured laboratory personnel to manipulate data,” but said the false information had no bearing on the drug's safety or efficacy. What’s striking about the FDA’s final decision is its contrast from the agency’s initial hard line.
Read more.
As STAT’s Ed Silverman reports, the FDA instructed Novartis to correct the problem, which arose from its AveXis division, but chose not to issue any punishment. The problem, you may recall, involved preclinical data for Novartis’s Zolgensma, approved to treat spinal muscular atrophy.
At the time of the controversy, the company fired two AveXis leaders who “either manipulated or pressured laboratory personnel to manipulate data,” but said the false information had no bearing on the drug's safety or efficacy. What’s striking about the FDA’s final decision is its contrast from the agency’s initial hard line.
Read more.
Rudy Giuliani has a plan to treat Covid-19
And the FDA is standing in his way. Or at least that’s how Giuliani sees it.
As STAT’s Adam Feuerstein reports, President Trump’s personal attorney has been using his his Twitter account and podcast to promote a stem cell treatment being developed by a New Jersey biotech company called Celularity.
The thing is, there is no evidence Celularity’s treatment, which has been used in early-stage studies of some cancer patients, has any benefit for Covid-19. And Giuliani, in a video interview with Celularity CEO Bob Hariri, urged the FDA to quickly clear the treatment for clinical trials in patients infected with the novel coronavirus.
To Paul Knoepfler, a stem cell researcher at the University of California, Davis, Giuliani’s words run the risk of politicizing what is meant to be a scientific process. “You could imagine Trump retweeting Giuliani about the stem cell treatment or even mentioning it verbally at a briefing,” Knoepfler said. “That could do a lot of harm.”
Read more.
As STAT’s Adam Feuerstein reports, President Trump’s personal attorney has been using his his Twitter account and podcast to promote a stem cell treatment being developed by a New Jersey biotech company called Celularity.
The thing is, there is no evidence Celularity’s treatment, which has been used in early-stage studies of some cancer patients, has any benefit for Covid-19. And Giuliani, in a video interview with Celularity CEO Bob Hariri, urged the FDA to quickly clear the treatment for clinical trials in patients infected with the novel coronavirus.
To Paul Knoepfler, a stem cell researcher at the University of California, Davis, Giuliani’s words run the risk of politicizing what is meant to be a scientific process. “You could imagine Trump retweeting Giuliani about the stem cell treatment or even mentioning it verbally at a briefing,” Knoepfler said. “That could do a lot of harm.”
Read more.
Today begins a new era at Vertex
As of this morning, Vertex Pharmaceuticals has a new CEO, as Reshma Kewalramani takes over for Jeffrey Leiden, concluding his seven-year tenure.
And it is, to put it lightly, an interesting time to lead a global biopharmaceutical company. Like every other business in the U.S., the drug industry is grappling with the coronavirus outbreak and a corresponding economic shutdown. Vertex said last week that the outbreak hasn't imperiled the company’s ability to supply its approved medicines for cystic fibrosis, but “to reduce the burden on the healthcare system at a time of critical need,” it will pause enrollment in some clinical trials and consider delaying new ones.
Beyond Covid-19, Kewalramani is inheriting a company at an inflection point. Vertex’s cystic fibrosis franchise is on pace to become a $10 billion enterprise, but what comes next remains to be seen. This year, the world will see mid-stage data on VX-814, a treatment for the rare lung disease AAT deficiency, and early results on VX-147, meant to treat a rare kidney disease abbreviated FSGS. Just how well each drug pans out will have a meaningful impact on Kewalramani’s early tenure.
And it is, to put it lightly, an interesting time to lead a global biopharmaceutical company. Like every other business in the U.S., the drug industry is grappling with the coronavirus outbreak and a corresponding economic shutdown. Vertex said last week that the outbreak hasn't imperiled the company’s ability to supply its approved medicines for cystic fibrosis, but “to reduce the burden on the healthcare system at a time of critical need,” it will pause enrollment in some clinical trials and consider delaying new ones.
Beyond Covid-19, Kewalramani is inheriting a company at an inflection point. Vertex’s cystic fibrosis franchise is on pace to become a $10 billion enterprise, but what comes next remains to be seen. This year, the world will see mid-stage data on VX-814, a treatment for the rare lung disease AAT deficiency, and early results on VX-147, meant to treat a rare kidney disease abbreviated FSGS. Just how well each drug pans out will have a meaningful impact on Kewalramani’s early tenure.
If the ACA gets overturned, biosimilars could disappear
Woven into the famously unwieldy Affordable Care Act is the legislation that brought biosimilars, cheaper versions of biotech medicines, to the U.S. That means the pending Supreme Court decision over whether the ACA is constitutional has existential implications for the growing biosimilar market.
Writing in STAT, Sandoz U.S. President Carol Lynch argues that biosimilar medicines are finally beginning to show real benefits to the health care system. For every treatment with an approved biosimilar, the average selling price of reference medicines declines each year by about 9%. That suggests that more biosimilars mean lower drug costs, provided the law doesn’t change.
“If the Supreme Court takes the unthinkable step of overturning most — or all — of the ACA, the regulatory pathway for biosimilars would essentially disappear overnight,” Lynch writes. “That would have ramifications for millions of patients who would no longer see more affordable biosimilar versions of high-cost biologic treatments reach the market.”
Read more.
Writing in STAT, Sandoz U.S. President Carol Lynch argues that biosimilar medicines are finally beginning to show real benefits to the health care system. For every treatment with an approved biosimilar, the average selling price of reference medicines declines each year by about 9%. That suggests that more biosimilars mean lower drug costs, provided the law doesn’t change.
“If the Supreme Court takes the unthinkable step of overturning most — or all — of the ACA, the regulatory pathway for biosimilars would essentially disappear overnight,” Lynch writes. “That would have ramifications for millions of patients who would no longer see more affordable biosimilar versions of high-cost biologic treatments reach the market.”
Read more.
More reads
- White House pressures FDA on unproven Japanese drug. (Politico)
- We shouldn’t rush to use an unproven malaria drug to treat the coronavirus. (STAT)
- China’s venture capitalists start making deals, amid signs of recovery. (The Information)
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