Regulation & Policy

Scott Gottlieb, commissioner of the US Food and Drug Administration, has resigned his position reportedly in order to spend more time with his family in Connecticut. The FDA job involved a weekly commute to Washington DC, a distance of about 277 miles.

Announced on 5 March, the resignation is expected to take effect in about a month. It was confirmed in a tweet by President Trump who praised Dr Gottlieb for his work leading the drug regulatory authority, a job that he has held since May 2017.

An approach to drug payments, where the price of a new medicine is linked to its effectiveness, may enable patients suffering from cancer to get new treatments faster, according to Cancer Research UK.

The European Medicines Agency (EMA) has been told it will have to continue servicing the lease covering its former London headquarters even as it completes plans for a move to Amsterdam. The move to Amsterdam follows the UK’s 2016 vote to leave the EU.

The UK High Court ruled on 20 February that the move “was not required as a matter of law.” Therefore the agency’s rental contract remains in force along with the landlord’s right to collect rent. The case was brought by the property company Canary Wharf Ltd.

The EMA is reportedly studying its options for an appeal.

The US Food and Drug Administration has given ‘fast track’ status to a prospective antibody treatment wholly-owned by Innate Pharma SA for the treatment of Sézary syndrome, an aggressive form of cutaneous T cell lymphoma.

A fast track designation enables the developers of promising drugs the opportunity for an expedited review. In this case the drug, IPH4102, produced favourable Phase 1 data in adult patients who had relapsed after receiving at least two prior therapies.

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.