FDA staff say Vertex CF combo works, unclear how
Vertex Pharmaceuticals Inc’s combination of an experimental compound and an approved drug significantly improved lung function in cystic fibrosis patients with the most common genetic mutation underlying the disease, FDA staff said.
The U.S. Food and Drug Administration staff reviewers, however, were not sure whether Vertex’s already approved therapy, Kalydeco, had a positive effect alone.
They also questioned if the experimental compound, lumacafotor, added any benefit.
Shares of the drugmaker, which is seeking approval for the combination to treat cystic fibrosis (CF) patients with the particular mutation, rose as much as 5 percent in early morning trading on Friday.
The FDA is trying to ask the panel if the evidence is enough to show that the combination’s benefit is significantly better than that of a single component, RBC Capital analyst Michael Yee said. “We think this is unlikely to block approval.”
CF is caused by a missing or defective gene that makes the body produce a thick, sticky mucus resulting in inflammation and recurrent bacterial infections. Most patients die in their mid-twenties.
Those afflicted by the incurable disease — 70,000 worldwide and 30,000 in the United States — need to take a cocktail of vitamins, painkillers and antibiotics to maintain daily functioning.
The FDA staff was satisfied with the safety profile of the combination, to be called Orkambi, according to documents released on Friday.
Orkambi is being evaluated for CF patients with a type of the F508del mutation, which accounts for half of all CF patients.
An FDA approval on July 5 will mean Vertex would be eligible to target about 8,500 patients in the United States, said Zachry Barber, Vertex’s senior director of corporate communications.
Kalydeco, approved to treat 10 different gene mutations, addresses about 2,000 people in the country. Its current label states that it is not effective in patients with F508del mutation.
The drug, which had sales of about $464 million in 2014, is expected to generate peak sales of $1.1 billion in 2020, according to Thomson Reuters Cortellis.
A panel of independent advisers to the FDA will make a recommendation on Orkambi on Tuesday. The agency is not obliged to follow the recommendations, but typically does.
Vertex shares were up 3.3 percent at $129.13 on the Nasdaq, after falling 5 percent in premarket trading.
(Reporting by Vidya L Nathan and Natalie Grover in Bengaluru; Editing by Joyjeet Das)