Regulation & Policy

A wave of clinical trial applications for new cell and gene therapies has reached the US Food and Drug Administration triggering a review of clinical guidance documents for developers. In an announcement on 15 January, Scott Gottlieb, the FDA commissioner, said new guidances are being drafted for gene replacement therapies as well as for cell-based gene therapies such as the chimeric antigen receptor (CAR) T cell drugs.

The US Food and Drug Administration has approved a new indication for Lynparza (olaparib) for the maintenance treatment of adults with suspected germline BRCA-mutated advanced ovarian cancer.

The European Medicines Agency’s main scientific committee has recommended approval of a new hybrid medicine for managing chronic pulmonary infection due to the bacteria Pseudomonas aeruginosa in patients with cystic fibrosis.

Novartis has agreed to offer Kymriah (tisagenlecleucel) the cell-based gene therapy, for an undisclosed discount to patients under the age of 25 years following a negotiation with UK authorities, including the National Institute for Health and Care Excellence (NICE). Supplies of Kymriah will be financed through the Cancer Drugs Fund, a special fund for promising treatments, NICE announced on 16 November.

A Chinese scientist’s reported use of the Crispr gene editing tool to alter a gene in two  embryos which were implanted into a mother’s womb has created an uproar in the scientific community. The edit was performed when the embryos were just a day old and involved alterations to the CCR5 gene in order to prevent the offspring from contracting HIV. They were reported on YouTube videos, rather in a scientific journal, after twin girls bearing the DNA changes were born.

A new precision medicine for patients whose cancers have a specific genetic feature has been approved by the US Food and Drug Administration – the second time the agency has based a decision on a common biomarker rather than the location of a tumour in the body.

The US Food and Drug Administration has approved a new oral treatment for acute myeloid leukaemia (AML) in adults who are 75 years or older and who have other chronic conditions that may preclude the use of intensive chemotherapy. The drug, Daurismo (glasdegib), is an inhibitor of the Hedgehog pathway which when abnormal, is thought to contribute to the development of cancer stem cells. Daurismo was developed by Pfizer Inc.

The European Commission has cleared Takeda Pharmaceutical Company Ltd’s proposed $62 billion takeover of Shire Plc, subject to its disposal of an investigational drug in Shire’s portfolio for the treatment of inflammatory bowel disease (IBD). The Shire compound overlaps with Entyvio, a drug marketed by Takeda for the same disease.

A new treatment for sleeping sickness, a life-threatening tropical disease, has received a positive review by the European Medicines Agency, paving the way for its approval in sub-Saharan Africa where the illness is endemic. The medicine, Fexinidazole Winthrop (fexinidazole), was developed through a collaboration between Sanofi SA and the non-profit organisation, Drugs for Neglected Diseases initiative (DNDi).

The antibody-drug conjugate Adcetris (brentuximab vedotin) has received a new lymphoma indication from the US Food and Drug Administration under a procedure that allows the agency to study key clinical data for the compound prior to its formal regulatory application.