Many people remember the 1972 children’s book “Alexander and the Terrible, Horrible, No Good, Very Bad Day,” a comical story of an eponymous boy whose day goes wrong at almost every turn. While it hasn’t been all bad news, the book’s overall theme captures how this week played out for biopharma.
Although they occurred separately, events caused several drugmakers’ shares to plummet this week. They included a public rebuke by the Food and Drug Administration over allegations of manipulated preclinical data; an erroneous adverse event report submission to the agency; the scheduling of an advisory committee meeting that delayed an approval date; and a product manufacturing issue.
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First, on Tuesday, the Food and Drug Administration took the unusual step of publicly criticizing Swiss drugmaker Novartis for the alleged inclusion of some manipulated animal data in its regulatory approval application for the gene therapy Zolgensma (onasemnogene abeparvovec-xioi), but didn’t inform the agency until months later, when it the product was already approved. The company’s shares fell by as much as 4 percent, though the therapy will remain on the market.
In a conference call with investment bank analysts Wednesday morning, Novartis CEO Vas Narasimhan tried to reassure them that the data manipulation was limited to a small number of scientists using a long-disused mouse assay, and that the delay was so that the company could complete an internal investigation. Novartis ended up getting some respite Wednesday evening, as the Centers for Medicare and Medicaid Services issued a long-awaited Medicare coverage decision for CAR-T cell therapies, including Novartis’ Kymriah (tisagenlecleucel).
But the biggest concentration of bad news came on Thursday.
Cambridge, Massachusetts-based Sarepta Therapeutics’ shares fell more than 12 percent on the Nasdaq following what it called the erroneous submission of an adverse event report to the Food and Drug Administration’s Adverse Event Reporting System in connection with its Phase II study of the gene therapy SRP-9001 in Duchenne muscular dystrophy. The company said that event – of rhabdomyolysis, muscle breakdown releasing damaging proteins in to the blood – was not reported by the company or a study investigator, and rhabdomyolysis is a common risk of DMD. Moreover, in addition to the patient having recovered, the trial is blinded, making it unclear if the event was drug-related.
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Another company, Dublin-based Amarin, said the FDA informed it that there would be an advisory committee meeting concerning its application to have the label for its triglyceride-lowering drug, Vascepa (icosapent ethyl), expanded based on results of a cardiovascular outcomes study. The agency had previously been silent about whether it would convene an AdCom meeting, but had told the company it would decide whether to grant the approval by Sept. 28. However, because the meeting will take place on the soonest possible date, Nov. 14, Amarin said it anticipates the action date will be sometime in late December. Shares of Amarin fell nearly 18 percent Friday morning following the news.
Meanwhile, CEO Howard Robin of San Francisco-based Nektar Therapeutics revealed during the company’s earnings call Thursday evening that a test of 22 lots of its experimental cancer drug NKTR-214 (bempegaldesleukin, or bempeg) used in a Phase I/II study were different from the other 20. Product from the two lots – whose differences from the rest stemmed from a suboptimal batch of in-process pharmaceutical intermediates – were administered to patients. Subsequent clinical testing found that patients who received bempeg from those two lots experienced lower clinical benefit than those who received the drug from the unaffected lots. Shares of Nektar fell 39 percent on the Nasdaq Friday morning.
Photo: Creative_Touch, Getty Images
