The US Food and Drug Administration has approved a new indication for Casgevy, a cell-based gene therapy for two blood disorders, making it available to individuals as young as two years. The disorders are sickle cell disease, a rare inherited blood disorder, and transfusion-dependent beta thalassaemia. Casgevy (exagamglogene autotemcel) was first approved by the FDA in 2023 for children with sickle cell disease from the age of 12 years.
uniQure NV has raised $259 million from a public share offering on Nasdaq a little more than a week after receiving positive feedback from the US Food and Drug Administration for a marketing authorisation application for the gene therapy AMT-130. Proceeds from the offering were announced on 25 June. They will be used to advance the therapy, which has been developed to treat Huntington’s disease, towards commercialisation. The company is expected to file an application for an accelerated approval of the therapy during the third quarter.
TRIMTECH Therapeutics Ltd, which is developing small molecule drugs that target and degrade protein aggregates associated with neurodegenerative diseases, has raised an additional $14 million in seed funding, bringing the total for the round up to $47 million. The financing was led by Johnson & Johnson Innovation and the Business Growth Fund Ltd (BGF), which joined existing investors.
Eli Lilly and Company has broadened an existing partnership with Shanghai, China-based Abbisko Therapeutics Co Ltd to collaborate on the discovery and development of new, but still unidentified medicinal products. In a statement released on 23 June, the US company said the upgraded collaboration is valued at about $1.9 billion.
California, US-based Frazier Life Sciences has led a $115 million Series A financing for RQ Biotechnology Ltd, a UK company developing engineered antibody therapies for influenza in order to provide durable protection against serious viral infections. The investor syndicate also includes the venture capital groups Forbion and EQT Life Sciences, alongside founding investor LifeArc Ventures.
AbbVie Inc, which for years marketed Humira, a former blockbuster medicine, is to replenish its portfolio with the acquisition of Apogee Therapeutics, a clinical stage biotech company with candidate products for inflammatory and immunological diseases. Announced on 22 June, the all-cash transaction values Apogee at $135.11 per share or approximately $10.9 billion. This represents a premium of about 49.5% over Apogee’s closing price on Nasdaq the day before the announcement.
A new study on the economic impact of pharmaceutical innovation in Europe shows sustained benefits across society, according to EFPIA, the European industry federation. Data from the study, released on 22 June, shows that between 2014 and 2024 Europe’s investment of €11.67 billion in new medicines returned more than five times the amount in social, economic and hospital cost savings – equating to €66 billion in total. This figure includes more than €9 billion in direct hospital savings. Key findings include:
The US Food and Drug Administration issued new draft guidance on 29 May about how companies can reduce the use of animals in drug testing – this time for cancer medicines. Entitled, Oncology Pharmaceuticals: Streamlined Nonclinical Safety Studies for Biologics and Conjugated Products, the guidance recommends new criteria for general toxicology studies, including when animal testing may be unnecessary because there is no binding or pharmacologic activity.
The European Medicines Agency, in coordination with the African Medicines Agency and national regulators in Africa, started talks in early June about organising clinical trials to evaluate possible vaccines and therapies against Ebola disease caused by the Bundibugyo virus. The virus is currently circulating in the Democratic Republic of Congo and Uganda and was declared a public health emergency of international concern by the World Health Organization on 17 May. As of 18 June, the WHO had reported just over 900 cases of the disease and approximately 235 deaths.
The US Food and Drug Administration has approved a new carbapenem antibiotic from GSK Plc to treat complicated urinary tract infections – the first oral treatment of its kind for the disease. The authorisation, announced on 17 June, is for Utebzi (tebipenem pivoxil), a broad-spectrum carbapenem antibiotic of the beta-lactam class. The treatment will be available as a tablet, in addition to its availability as an in-hospital intravenous therapy.