Vertex's Triple Combo Cystic Fibrosis Pill Kaftrio Gets EU Nod

Vertex Pharmaceuticals Incorporated VRTX announced that the European Commission has approved its triple combination cystic fibrosis (CF) regimen Kaftrio (ivacaftor/tezacaftor/elexacaftor). The drug is now approved in combination with Kalydeco (ivacaftor) for treating CF in patients aged 12 years and above with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in their CFTR gene.

Kaftrio is already marketed in the United States as Trikafta, which was approved by the FDA in October 2019. Trikafta’s early approval and launch was a significant milestone for Vertex. The drug generated sales worth $1.8 billion in the first half of 2020. It saw a solid uptake in the country since its early unveiling and has been a key driver for the company ever since.

Following the nod, about 10,000 new patients with one minimal function mutation and one F508del mutation are eligible to be treated with Kaftrio in Europe to address the underlying cause of CF. Moreover, people aged 12 years and above with two F508del mutations will also be eligible for the new triple combination regimen.

The marketing authorization in Europe was based on data from two global phase III studies, which showed that treatment with Kaftrio led to statistically significant and clinically meaningful improvements in lung function, which was the primary endpoint as well as all key secondary endpoints. The triple combination regimen was generally well tolerated in both studies.

Vertex already received reimbursement agreements for Kaftrio in the EU countries, such as England, Denmark, the Republic of Ireland and Germany. With the approval of Kaftrio, eligible patients in these countries will now have access to the triple combination regimen in the upcoming weeks.

Shares of Vertex have rallied 23.9% so far this year compared with the industry’s increase of 1.9%.

Meanwhile, Trikafta is also being evaluated in phase III studies for treating children aged from six years to 11 years. A supplemental new drug application (sNDA) seeking an approval for the pediatric patient population is expected to be filed in the fourth quarter of 2020. Trikafta/Kaftrio is crucial for Vertex’s long-term growth as it comes with a potential to treat up to 90% of CF patients.

Vertex’s revenue growth in 2020 is primarily recognized from the uptake of Trikafta as well as higher international revenues owing to additional ex-U.S. reimbursement arrangements. Notably, a high percentage of the currently eligible patients is already on Trikafta. The approval of Kaftrio in the EU, reimbursement deals for the product in the EU countries and an approval for younger patient populations could bring additional Trikafta/Kaftrio revenues in 2021.

Zacks Rank & Stocks to Consider

Vertex currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the healthcare sector include Emergent BioSolutions Inc. EBS, Horizon Therapeutics Public Limited Company HZNP and QIAGEN N.V. QGEN, all sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

Emergent’s earnings estimates have moved 62.3% and 78.8% north for 2020 and 2021, respectively, over the past 60 days. The stock has skyrocketed 137.3% year to date.

Horizon’s earnings estimates have been revised 61.5% and 57.7% upward for 2020 and 2021 each over the past 60 days. The stock has rallied 108.1% year to date.

QIAGEN’s earnings estimates have moved 23.2% and 34.7% north for 2020 and 2021, respectively, over the past 60 days. The stock has soared 54.9% year to date.

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