Citing the urgent need for new drugs to treat neurological diseases, the US Food and Drug Administration has shaken up its review teams to encourage experts to work across disciplines and identify new opportunities for treatment.
After one volunteer died and four others were seriously injured during a clinical study in France in 2016, the European Medicines Agency revised its rules for first-in-human studies to tighten the requirements for the dosing of experimental drugs in humans. Introducing the changes in 2017, the agency said that clinical trial protocols are now more complex than ever before and sponsors will need to work harder to exercise oversight.
AstraZeneca Plc has won a new indication for its checkpoint antibody Imfinzi (durvalumab) from the US Food and Drug Administration to reduce the risk of non-small cell lung cancer progressing. Imfinzi targets the programmed death ligand-1 (PD-L1).
The US Food and Drug Administration has used a novel endpoint to approve a new drug for prostate cancer. Measuring metastasis-free survival, the agency decided that the Janssen Pharmaceutical drug Erleada (apalutamide) is effective.
After Amazon, Berkshire Hathaway Inc and JP Morgan Chase & Co announced on 30 January that they will take steps to reduce the rising cost of US health insurance, shares of health insurance companies and pharmacies declined.
A decades’ long discussion in the EU about how to establish common rules for assessing the value of new medicines has come to a conclusion with a proposal from the European Commission on 31 January to regulate health technology assessment (HTA).
The European Medicines Agency is recommending approval of a new medicine for haemophilia A developed by Roche that is intended to prevent bleeding or reduce the frequency of bleeding episodes in patients who have developed resistance to existing medicines.
A cancer drug that was dropped from development in 2011 but put back into clinical testing two years later with a different group of patients, has received approval for a third indication from the US Food and Drug Administration. The newest approval is to treat patients with certain types of breast cancer whose tumours have a BRCA gene mutation.
The first gene therapy for patients with an inherited retinal disease was authorised by the US Food and Drug Administration on 19 December, bringing the number of approved gene therapies in the US up to three. Luxturna (voretigene neparvovec-rzyl), was developed by Spark Therapeutics Inc.
The European Medicines Agency has issued a positive opinion for an allogeneic stem cell therapy to treat complex perianal fistulas in patients with Crohn’s disease. The product, Alofisel (darvadstrocel), was developed by TiGenix NV of Belgium.