Cystic fibrosis is a progressive disease that causes persistent lung infections and can lead to breathing difficulties. For many years, scientists have been looking for new treatments for this genetic illness, and now, two promising new drugs have been developed.
The two developments have been announced this week, and could bring hope to patients suffering from both cystic fibrosis and Alzheimers. The drugmaker responsible for them, Biogen, said earlier this week that it will now be seeking FDA approval for the drug, aducanumab.
The company first announced the drug in 2015. After this, Biogen said it had cleared the final stage of clinical trials, which showed it was safe for humans. The drug works by reducing the amount of amyloid plaque in the brain – this is believed to be responsible for the cognitive decline that happens in Alzheimer’s patients.
However, several months ago, Biogen said it was unlikely to be approved by the FDA due to further testing, showing that it wouldn’t be effective in treating Alzheimer’s. But, according to the drugmaker, new data has shown that these results were incorrect and that increasing the dosage resulted in “reduced clinical decline”. It will now be seeking approval for the drug.
In a statement, the Alzheimer’s Association welcomed the news, saying: “Alzheimer’s and other dementias are complex, and their effective treatment and prevention will likely also be a complex – but achievable – task. All currently pursued treatments that are considered safe should be continued to determine their efficacy.”
If cleared, it will also be suitable for patients over the age of 12 with cystic fibrosis who has at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It’s estimated that this represents around 90% of cystic fibrosis sufferers.
The FDA said in a press release: “At the FDA, we’re consistently looking for ways to help speed the development of new therapies for complex diseases, while maintaining our high standards of review. Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy.”