AstraZeneca drug for rare disease NF1 approved by EU for children

·2-min read
Photo: AstraZeneca
AstraZeneca said it 'has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death'. Photo: AstraZeneca

A drug called Koselugo, created by AstraZeneca (AZN.L) and Merck & Co to treat children with neurofibromatosis type 1 (NF1), has been granted conditional approval by the European Union (EU).

NF1 has been described as a debilitating genetic condition affecting one in 3,000 individuals worldwide.

In 30% to 50% of patients, tumours develop on the nerve sheaths and can cause clinical issues such as disfigurement, motor dysfunction, pain, visual impairment and bladder or bowel dysfunction.

A trial has shown Koselugo reduces the size of inoperable tumours in children, reducing pain and improving quality of life.

This is the first approval of a medicine for NF1 PN in the EU and follows the positive recommendation by the Committee for Medicinal Products for Human Use of the European Medicines Agency in April 2021.

"Before this approval, surgery was the only treatment option for children in the EU with neurofibromatosis type 1 plexiform neurofibromas. This approval marks a significant step forward in addressing the debilitating impact of these tumours," said Roy Baynes, chief medical officer at Merck Research Laboratories. 

Read more: EU regulator recommends halting AstraZeneca vaccines for over-60s

“AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death,” the company said in a statement.

Shares in the company were down 1.5% on Tuesday morning.

AstraZeneca stock chart. Source: Yahoo Finance
AstraZeneca stock chart. Source: Yahoo Finance

Meanwhile the EU and AstraZeneca have had a tumultuous relationship when it comes to vaccines. In April the EU began legal action against the pharma giant, alleging it had failed to fulfil promises made in its contract to supply COVID-19 vaccines.

The EU believed AstraZeneca has been unfairly and illegally routing jabs to the UK at the expense of Europe and wanted at least 120 million doses by the end of June. However, a ruling said AstraZeneca must deliver only 80.2 million doses by September.

Earlier this month, a leading EU drugs regulator recommended that countries should avoid giving the vaccine to people over the age of 60.

Watch: Got both doses of AstraZeneca vaccine? Here's what you can and can't do

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