EU approves AstraZeneca Drug Koselugo for Treating NF1 in children


  • AstraZeneca and Merck drug Koselugo has been approved by the EU.
  • Koselugo is the first medicine approved to treat children with NF1, which is a rare disease.
  • The drug was approved as per the SPRINT Stratum 1 Phase II trial, which showed positive results for NF1 treatment.

The European Union has approved the drug Koselugo, manufactured by AstraZeneca (LON: AZN) and Merck & Co, to treat children above 3 years of age with neurofibromatosis type 1 (NF1), a rare genetic disease. The drug was approved as per the SPRINT Stratum 1 Phase II trial, which showed positive results for NF1 treatment.  

NF1 is a genetic condition affecting one in 3,000 people worldwide. NF1 is a complex multi-system human disorder that happens due to mutation of a gene on chromosome 17, leading to the production of protein called neurofibromin. NF1 causes tumours along with the nervous system, which can grow anywhere in the body.

Children affected with this disease develop tumours and can cause issues such as bowel dysfunction, bladder disorder, disfigurement, pain, visual impairment, and others. The symptoms start showing from early childhood, with different levels of severity, which can also shorten the life span of the patient.

First of a kind

The European Commission approval was based on positive results from the SPRINT Stratum 1 Phase II trial, which was sponsored by the National Institute of Health's National Cancer Institute Cancer Therapy Evaluation Program.

The drug Koselugo showed positive results such as reducing the size of inoperable tumours in children, reducing pain, and improving quality of life.

Koselugo demonstrated an objective response rate (ORR) of 66% in paediatric patients with NF1 PN. When treated with this drug as twice-daily oral monotherapy.

In April 2021, the drug Koselugo got a positive recommendation by the Committee for Medicinal Products for Human Use of the European Medicines Agency.

Experts opinion

The drug approval marks a significant step in the treatment of NF1 and the debilitating impact of these tumours. Before the medicine, surgery was the only treatment option for children affected by the rare disease, said Roy Baynes, chief medical officer at Merck Research Laboratories.

Dave Fredrickson, Executive Vice President of Oncology Business Unit, said AstraZeneca drug Koselugo had transformed the way this rare disease plexiform neurofibromas can be treated. “Clinical trials of Koselugo in adult patients with NF1 PN along with an alternative age-appropriate formulation for paediatric patients are scheduled to begin this year.”

AstraZeneca is a research-based biotechnology and pharmaceutical company that aims to cure every form of cancer by developing and delivering life-changing medicines to treat patients.

The shares of AstraZeneca were trading at GBX 8308.00, down by 0.5%, on 23 June at 08:025 GMT+1. AstraZeneca’s Year-to-Date returns stood at 13.39%.

Headquartered in New Jersey, Merck & Co. is an American multinational pharmaceutical company. In the 2021 Fortune500 magazine, the company is ranked 69th. The company develops medicines, vaccines, biological therapies, and animal health products.