We learned last week that the FDA was investigating Novartis for manipulating animal data related to its $2 million-a-dose gene therapy Zolgensma. The activity took place at AveXis, the firm Novartis bought for $8.7 billion in 2018, and Novartis reportedly knew but failed to report it to the FDA before the agency approved Zolgensma in May. There are still many unanswered questions, such as when the data-cooking took place. Before Novartis bought AveXis? After?
AveXis spun out of Nationwide Children’s Hospital in Columbus, OH, where the Zolgensma program originated. The manipulation didn’t take place there, according to hospital spokesperson Gina Berrichia.
“To our understanding, the reported data manipulation did not occur in preclinical studies or clinical trials conducted at Nationwide Children’s,” Berrichia wrote in an e-mail to Xconomy late Thursday. “We have not been contacted by the FDA, Novartis, or Avexis regarding this matter.”
The fallout first intensified late last week, when a group of Democratic senators wrote to FDA commissioner Ned Sharpless that “anything short of a forceful response would signal a green light to future pharmaceutical misbehavior.” This week, Novartis said it had taken action against brothers Brian and Allan Kaspar, the scientific founder and research chief of AveXis, respectively, while the FDA was still reviewing Zolgensma. The Kaspars “haven’t been involved in any operations at AveXis since early May,” were replaced by Novartis veteran Page Bouchard on Aug. 5, and are no longer with the company, Novartis said in a statement. Former FDA commissioner Robert Califf penned an op-ed in Stat about the FDA’s unusually harsh response.
Before he cofounded AveXis, Brian Kaspar led the development of Zolgensma at Nationwide, which—thanks in large part to Zolgensma’s success—has rapidly become a gene therapy research powerhouse, as Xconomy detailed back in April.
Oh, and Novartis also said US pharma chief Fabrice Chouraqui will step down at the end of the month, but it doesn’t seem Zolgensma was within Chouraqui’s purview.
There’s plenty more news to round up this week, so let’s get to it.
POLITICS & POLICY
—Angry about a Trump administration proposal to import cheaper drugs from Canada, Canadian officials are considering ways to thwart the plan. Stat has more.
—With the import issue in the spotlight, Sen. Chuck Grassley (R-IA), chairman of the Senate finance committee, called on the FDA to conduct more snap inspections of foreign drug-making facilities. The safety and quality of drug manufacturing in India and China has also recently been under scrutiny.
—A US judge last Friday rejected a challenge to Amgen (NASDAQ: AMGN) patents that protect its blockbuster autoimmune disease drug etanercept (Enbrel) from generic competition. Challenger Novartis, which wants to market a biosimilar in the US, will appeal the ruling.
—In the wake of last year’s revelation of the first babies to come from gene-edited embryos, scientists want to produce clearer rules about human embryo editing, Stat reported.
APPROVALS & DATA
—Two experimental drugs produced better survival rates among people infected with Ebola virus in the Democratic Republic of the Congo than previously available treatments, prompting study leaders to put everyone on the new drugs. People with low levels of the virus had survival rates of 94 percent and 89 percent with the two experimental drugs, REGN-EB3 from Regeneron Pharmaceuticals (NASDAQ: REGN) of Tarrytown, NY, and mAb114 from Ridgeback Biotherapeutics of Miami.
—The Global Alliance for TB Drug Development, a New York nonprofit, won FDA approval for Pretomanid, a tablet to treat drug-resistant forms of tuberculosis. It’s the second approval under a new regulatory pathway to encourage more antibacterial and antifungal products.
—By next week, the FDA could also give the nod to up to four new drugs for Duchenne muscular dystrophy, bacterial pneumonia, cancer, and rheumatoid arthritis. Xconomy previewed the approval decisions and the significance of each drug, including Roche’s entrectinib (Rozlytrek), which got the agency’s green light late Thursday.