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FDA gives Vertex the go-ahead for Symdeko in CF

By Randy Osborne, Staff Writer

Well ahead of the February 28 PDUFA date, Vertex Pharmaceuticals Inc. scored an approval of the combo drug Symdeko (tezacaftor/ivacaftor and ivacaftor) for cystic fibrosis (CF), specifically to treat the disease in people age 12 and older with two copies of the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or who have at least one mutation that is responsive to tezacaftor/ivacaftor. Ivacaftor alone was previously approved as Kalydeco.

Symdeko is Boston-based Vertex’s third drug cleared to treat the underlying cause of CF, the company noted, and shipping to pharmacies will start this week. The wholesale acquisition cost for the treatment is $22,400 per 28-day pack (about $292,000), a price that’s about 25 percent higher than modeled by Raymond James Analyst Laura Chico.

Vertex had withheld fiscal year 2018 guidance until Symdeko’s approval, so those numbers rolled out, too. The company estimated total CF product revenues of $2.65 billion to $2.80 billion, in line with pre-consensus guesses of $2.77 billion and implying a 10 percent year-over-year growth from the mid-point of the range. Vertex reiterated the non-GAAP combined R&D and selling, general and administrative (SG&A) expenses of $1.50 billion to $1.55 billion, along with GAAP combined R&D and SG&A expenses of $1.80 billion to $1.95 billion.

James’ Chico said in a report that she “continue[s] to like the setup” at Vertex and raised her Symdeko estimates slightly for fiscal year 2018, “which increases our revenue [target] to $2.74 billion [previously $2.63 billion]. Our non-GAAP R&D and SG&A remain unchanged, and with the higher revenue, our non-GAAP EPS increases to $3.05 from $2.70,” as compared to consensus of $3.02). “We see [Symdeko’s] approval as an important gating step to help advance triple-combo regimen efforts,” she said, holding onto her outperform rating on the shares.

In November 2017, The New England Journal of Medicine published the Symdeko-bolstering results of two phase III studies called Evolve and Expand. They enrolled about 750 people with CF ages 12 and older with two copies of the F508del mutation or with one F508del mutation and one mutation that results in residual CFTR function. Across both studies, patients treated with the compound turned up statistically significant and clinically meaningful improvements in lung function and other measures of disease, with a favorable safety profile. The most common adverse events, regardless of treatment group, included infective pulmonary exacerbation and cough.

The first data from the ongoing Extend rollover study with Symdeko, also made public in November, show that the lung function improvements and the safety and tolerability profiles seen in Evolve and Expand were sustained for up to 48 weeks.

Approval of Symdeko is expected in the EU during the second half of this year, as the EMA has validated the marketing authorization application for the tezacaftor/ivacaftor combination, Vertex said. Shares of the company (NASDAQ:VRTX) were trading premarket at $155, up $2.99.