The US Food and Drug Administration (FDA) has given its approval to Vertex’s combination treatment for cystic fibrosis (CF), marking the biotech’s third product for the lung condition alongside Kalydeco and Orkambi.
Dubbed Symdeko, the treatment uses existing drug Kalydeco (ivacafto) alongside tezacaftor in a combination that has been shown to improve lung function, reduce pulmonary exacerbations and increase BMI (though the latter was not improved in a statistically significant manner).
In a Phase III trial, patients also reported fewer symptoms such as coughing and difficulty breathing. Symdeko will be used in patients from the age of 12 who have two copies of the F508del mutation, or who have at least one mutation that is responsive to tezacaftor and ivacafto.
The treatment comes with a listed price of $292,000 per year, with sales of the new therapy expected to range between $100 million and $500 million through to 2020. It is anticipated to eventually form the backbone of triple combinations targeting the condition.
“Today is an exciting day for the CF community,” Vertex chairman Jeffrey Leiden said.
“This approval is an important milestone in our journey to treat every person with CF, and we remain committed to urgently advancing our efforts to develop new medicines that treat the underlying cause of CF for the many people still waiting.”
Vertex expects the new therapy to receive approval from the European Medicines Agency (EMA) in the latter half of 2018.
Treatments for CF work to reduce and control mucus in the lungs, replace digestive enzymes and prevent chest infections. Bronchodilators are taken to widen airways and ease breathing difficulties, while steroid medication is used to treat nasal polyps. Mucus-thinning medicines include dornase alfa, hypertonic saline and mannitol dry powder, and the drug ivacaftor is taken to reduce mucus levels in the body. However, the latter is only suitable for around 4% of CF patients. Currently medicines for the condition can help around 45% of CF sufferers, while there is hope that Vertex’s new combination will treat up to 90% of patients.
The company has two ongoing Phase III trials examining triple therapy for CF. A Phase II trial has already shown positive results testing tezacaftor and Kalydeco alongside investigational CFTR protein corrector VX-659, improving lung function by 13.3%. The two drugs will be tested alongside another candidate, VX-445, giving the company two options for further trials.