The first quarter of 2018 for Vertex Pharmaceuticals was highlighted by the U.S. Food and Drug Administration’s February approval of Symdeko, a combination treatment for cystic fibrosis.
The first quarter of 2018 for Vertex Pharmaceuticals was highlighted by the U.S. Food and Drug Administration’s February approval of Symdeko, a combination treatment for cystic fibrosis. This morning the company said the drug’s launch is underway and patients are receiving treatment.
In only a few months’ time Symdeko has generated $34 million in new revenue for Vertex, the company said in its quarterly report. That number will certainly expand in future quarters. Also, the company said it anticipates European approval of the combination treatment later this year.
Symdeko is the third cystic fibrosis treatment that Vertex has had approved. Vertex already markets CF drugs Kalydeco and Orkambi, which allows the company to already treat about 30,000 CF patients. There are an estimated 75,000 CF patients people in North America, Europe and Australia who have cystic fibrosis and Vertex hopes to develop treatments that can benefit more people. Today Vertex said it has initiated Phase III studies of VX-445, tezacaftor and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis (CF). Vertex is keeping its promise of advancing triple combination treatments for CF patients. Vertex believes the combination treatment will target the underlying cause of cystic fibrosis in patients who have one F508del mutation and one minimal function mutation (F508del/Min) – one of the most difficult types of CF to treat.
In March the company announced a Phase III trial for VX-659, a triple combination therapy for people with cystic fibrosis (CF) who have two copies of the F508del mutation, which is the most common genetic form of the disease. That’s the second Phase III trial for this particular combo. In February the company initiated a Phase III trial of VX-659 for patients with one F508del mutation and one minimal function mutation.
While the company is advancing VX-445 and VX-659, Vertex indicated that one mid-stage treatment could be held up by the FDA due to lack of data. The FDA is seeking dose-ranging data for VX-561, which is being evaluated as a once-daily regimen in F508del/Min and F508del/F508del patients. The company said it will comply with the FDA’s request to support the potential late-stage development of the therapy. Jeffrey Leiden, Vertex’s chief executive officer, told investors during a conference call today that the company will develop a bridging study to address the FDA’s concerns.
While that bit of news was not pleasing to investors, shares are down about 1.5 percent as of 11 a.m., the company reported strong growth from its CF pipeline this quarter. Sales of CF drugs came in at $638 million for the quarter, which was an increase of about 33 percent over the same quarter one year ago. With those strong sales from the quarter, Vertex reiterated its financial guidance of $2.65 to $2.8 billion for its CF franchise.
“During the quarter, the number of patients eligible for and being treated with our CF medicines continued to increase and drive revenue and earnings growth. We continued to make significant progress toward our goal of developing new and better medicines for the treatment of CF and other serious diseases,” Leiden said.
