An oral drug combination by Swiss pharmaceuticals company Novartis showed promise in treating a subgroup of patients suffering from a common childhood brain cancer in a trial.
In the mid-stage trial, 47% of the patients that were given the two drugs Tafinlar and Mekinist saw their tumours shrink, far above a rate of 11% in a comparative group of participants on standard chemotherapy, the drugmaker said on Monday.
The participants, aged 1 to 17 years, were suffering from low-grade gliomas (LGG), the most common childhood brain cancer, which is diagnosed in more than 1,000 U.S. children per year.
The trial only included those who were found to have a mutation known as BRAF V600, a genetic contributor in about 15% to 20% of pediatric LGG cases.
Among further results of the trial with 110 participants, the median time without disease progression was 20.1 months for those given the Novartis drug combo, compared to 7.4 months on chemotherapy.
The new oral treatment candidate also caused less severe side effects than burdensome chemotherapy.
Tafinlar and Mekinist, a pill combination with $1.7 billion in 2021 sales, up 10%, had previously won approvals to treat certain skin, lung and thyroid cancer patients who have the BRAF V600 genetic change.
It is an important growth driver in Novartis's oncology business, a key therapeutic area for the Swiss group, which relies on drug development as it considers the sale of its Sandoz business with off-patent generic drugs.
For the childhood brain cancer trial, a more convenient liquid form was used for the first time.
The data, presented at the annual meeting of the American Society of Clinical Oncology (ASCO) on Monday will be submitted to regulators as part of a request for approval, Novartis added.
(Reporting by Ludwig Burger, Editing by Louise Heavens)
