Business-in-Brief

Advanced medicine

• The FDA’s Center for Biologics Evaluation and Research (CBER) unveiled six new draft guidance documents covering gene therapy. The guidance documents focus on i) rare disease therapies, ii) retinal disorder therapies, iii) haemophilia therapies, iv) chemistry, manufacturing and control (CMC) information, v) long-term follow-up observational studies that collect data on adverse events, and vi) retroviral vector-based therapies testing.
• Japanese scientists will use induced pluripotent stem (iPS) cells in a Parkinson’s trial for the first time, following successful animal studies. In an attempt to replace the lost dopaminergic cells responsible for Parkinson’s, the researchers, led by Jun Takahashi at Kyoto University’s Center for iPS Cell Research and Application (CiRA), will derive dopaminergic progenitors from iPS cells and inject roughly five million of them into the forebrain. Sumitomo Dainippon Pharma hopes to manufacture and start selling cellular medicine based on the data from the clinical trials by March 2023.

Politics

• In reaction to Brexit, Sanofi will begin to increase the amount of medicine stocks held in the UK from around 10 weeks’ to 14 weeks’ worth, as of April 2019, based on internal assumptions of potential delays following a “no deal” scenario, according to a statement from the company. Hugo Fry, managing director of Sanofi UK, said, “Patient safety is our main priority and we have made arrangements for additional warehouse capacity in order to stockpile our products, where global supply allows, in the UK and increase UK-based resource to prepare for any changes to customs or regulatory processes.”
• In the US, Senators Dick Durbin and Chuck Grassley have proposed funding-bill amendments forcing pharma companies to put drug pricing in their ads – as proposed by President Trump in his American Patients First blueprint for reducing drug prices. The proposal has bipartisan backing, including from the Democrats. PhRMA remains opposed, arguing that including list prices may deter patients from talking to a doctor about treatment.

Scandals

• The Chinese government has promised tough penalties for those involved in the sale of 252,600 faulty vaccines, after widespread public anger. A special cabinet investigation team found that Changchun Changsheng Bio-technology, the vaccine maker involved, had manufactured inferior vaccines, falsified data, sold ineffective vaccines, and fabricated production and inspection records relating to a rabies vaccine used for infants.
• A Republican congressman in the US has been charged with insider trading after allegedly telling his son the results of a clinical trial so that they could sell stock. Chris Collins was heavily invested in Innate Immunotherapeutics, a small drugmaker based in Australia. The company has no approved drugs and the failed multiple sclerosis trial sent its stock tumbling once the news went public. Federal prosecutors are accusing Collins of using his knowledge of the failed trial to sell stock before the news went public – avoiding losses of more than $570,000.