News

The US Food and Drug Administration has reorganised its information technology work into a new office and appointed British educated Vid Desai to lead it. The new Office of Digital Transformation will combine all of the functions of the agency’s current IT, data management, and cybersecurity functions in a single unit, reporting directly to the FDA commissioner. It is part of a modernisation drive started in 2019 to operate more efficiently and increase the use of new technologies in the review of medicines and the monitoring food safety.

Jyseleca (filgotinib), a small molecule drug that blocks enzymes contributing to inflammatory diseases, is being recommended by the European Medicines Agency for the treatment of ulcerative colitis. Jyseleca has already been approved in the EU for rheumatoid arthritis. A new indication would broaden the drug’s coverage to include the treatment of adults with moderate to severely active ulcerative colitis who have not responded to earlier treatments.

A novel form of insulin that is intended to accelerate absorption of the hormone by patients with Type 1 diabetes has met the primary endpoint of non-inferiority in a Phase 1 trial compared with NovoRapid, a standard of care. Arecor Therapeutics Plc, the developer, announced the results on 20 September. AT278 is an ultra-concentrated formulation that has been designed to speed absorption of insulin post injection even when delivered at a high concentration.

A bid by Philip Morris International Inc for Vectura Group Plc became unconditional on 15 September following acceptance by investors representing 74.77% of Vectura’s shares, the two companies announced. In August, the Vectura board of directors accepted the offer and this has now been approved by shareholders.

A new Dutch biotech company was launched on 16 September to discover and develop antibody-based drugs for diseases caused by oxidative stress. Oxitope Pharma BV was founded and seed funded by Forbion Capital Partners and uses technology in-licensed from the University of California San Diego, US.

Anjarium Biosciences AG, a Swiss company with technology for delivering gene therapies to patients without the use of a viral vector, has raised CHF 55.5 million (€50.96 million) from a group of investors co-led by Gimv and Abingworth LLP. The money will be used to expand the company’s staff and advance several of its programmes towards the clinic.

France-based Coave Therapeutics (formerly Horama SAS) has entered into a licensing deal, giving Théa Open Innovation, an ophthalmology company, rights to co-develop and commercialise a gene therapy for retinitis pigmentosa.

Astellas Pharma Inc has reported the death of a participant in a clinical trial of a gene therapy for the treatment X-linked myotubular myopathy, a rare neuromuscular disease caused by mutations in the MTM1 gene. The participant, who developed an adverse event during the trial, passed away on 9 September. “The cause of death is still pending,” the company announced on 14 September.

The US Food and Drug Administration has placed a clinical hold on a Phase 1/2 trial of a gene therapy for adults with phenylketonuria, a genetic disorder caused by the deficiency of an enzyme needed to break down an amino acid in certain foods. The clinical hold was based on findings from a preclinical study showing that six out of seven mice receiving the highest dose of the therapy developed liver tumours. One of the tumours was cancerous. BioMarin Pharmaceutical Inc, the developer, announced the clinical hold on 6 September.

AbbVie Inc has acquired rights to a gene therapy in development by Regenxbio Inc for wet age-related macular degeneration (AMD) which the companies say has potential as a one-time treatment for the disease. The therapy’s viral vector encodes an antibody fragment which is expressed inside the body to inhibit vascular endothelial growth factor (VEGF).