Regulation & Policy

Gene therapy for bladder cancer

Country
United States

The US Food and Drug Administration has approved a gene therapy for bladder cancer, the second authorisation of a novel gene treatment within a month. The new drug, Adstiladrin (nadofaragene firadenovec), is indicated for the treatment of patients with bladder cancer which hasn’t yet invaded the muscle and is unresponsive to the Bacillus Calmette-Guérin (BCG) immunotherapy. The decision comes not long after the agency approved a gene therapy for haemophilia B.

FDA approves first faecal microbiota product

Country
United States

The first faecal transplant therapy derived from the gut microbiome of healthy donors was approved by the US Food and Drug Administration on 30 November, paving the way for regulatory reviews of other products in the sector. The therapy, Rebyota, was developed by Ferring Pharmaceuticals Inc and approved for the prevention of recurrent Clostridioides difficile infection, a potentially life-threatening disease.

Gene therapy for haemophilia B approved

Country
United States

The first gene therapy for adults with haemophilia B has been approved by the US Food and Drug Administration after showing that a single dose of the treatment was able to significantly reduce abnormal bleeding.

Haemophilia B is a genetic disease arising from missing or insufficient levels of blood clotting factor IX, a protein that is needed to produce blood clots to stop bleeding. Symptoms can include prolonged or heavy bleeding after an injury or surgery, or spontaneous bleeding without a clear cause. Most people affected by the disorder are men.

Antibiotic drug use drops

Country
Netherlands

The use of antibiotic medicines in animals has dropped sharply in Europe following multiple public policy initiatives and national campaigns, according to the European Medicines Agency. In a report issued on 18 November, the agency said that sales of veterinary antibiotics in animals decreased by 47% in the ten years to 2021 – the lowest level ever reported. The report is based on data from 25 European countries.

JAK inhibitor label update

Country
Netherlands

A safety review of the Janus kinase (JAK) inhibitor group of medicines for chronic inflammatory disorders has concluded that the drugs should only be used in patients with certain risk factors if no suitable alternatives exist. The review was conducted by the pharmacovigilance risk assessment committee of European Medicine Agency and its conclusions were endorsed on 11 November by the agency’s main scientific committee, the CHMP. The recommendations now go to the European Commission for formal approval.

Regulatory support for academic medicine

Country
Netherlands

The European Medicines Agency has launched a new project to help academic scientists and those working for non-profit organisations understand the regulatory requirements for developing and eventually commercialising cell, gene and tissue engineered therapies. The project complements the agency’s priority medicines scheme, PRIME, which was launched in 2016 to provide support to developers of medicines that meet a major public health need.

New drug for ALS

Country
United States

The US Food and Drug Administration has approved a new drug for amyotrophic lateral sclerosis, a rare disease that attacks and kills the nerve cells that control voluntary muscles. In a Phase 2 trial, the drug Relyvrio (sodium phenylbutyrate and /taurursodiol), showed an ability to slow the rate of neuromuscular decline. Moreover in a follow-up study, it extended patient survival compared with a placebo.

New RSV treatment reviewed

Country
Netherlands

A new treatment designed to protect infants from infection from the respiratory syncytial virus (RSV) has received a positive opinion from the European Medicines Agency. The accelerated review was in response to evidence that infections from RSV are on the rise following a relaxation of restrictions that have been in place to prevent Covid-19.

EMA recommends bivalent Covid-19 vaccine

Country
Netherlands

The European Medicines Agency has recommended approval of a bivalent vaccine to protect against two sub-variants of the SARS-CoV-2 virus. The authorisation is for an updated booster shot developed by Pfizer Inc and BioNTech SE to protect people against the omicron subvariants BA.4 and BA.5 in addition to the original strain of SARS-CoV-2. The new vaccine is to be used in people aged 12 years and older who have received at least a primary course of vaccination to prevent Covid-19.

Enzyme replacement for genetic disease

Country
France

An enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD), a rare genetic disease that can cause premature death, has cleared the US Food and Drug Administration making it the first medicine to treat symptoms of the disease that are not related to the central nervous system. The FDA approval of Xenpozyme (olipudase alfa) was granted to Genzyme, a division of Sanofi SA.