Vertex Pharmaceuticals Incorporated VRTX announced that its type II variation marketing authorization application (“MAA”) for its triple combo cystic fibrosis (“CF”) drug, Kaftrio, has been validated by the European Medicines Agency (“EMA”).

The MAA is seeking label expansion of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with the company’s another CF drug, Kalydeco (ivacaftor) as a potential treatment for CF patients aged 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (“CFTR”) gene

The regulatory application in Europe is supported by positive data from the phase III 445-104 study. Data from the study demonstrated that treatment with Kaftrio achieved a statistically significant 3.7% improvement in ppFEV1, a key measure for lung function, from baseline.

Last month, Kaftrio in combination with Kalydeco received approval 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 in Europe. A potential approval to the recent MAA will increase the eligible patient population as patients who have one copy of the F508del mutation along with another CFTR mutation, such as a gating (F/G) or residual function (F/RF) mutation, will also be eligible for treatment.

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 growth driver since then. A similar performance in Europe will be beneficial for the company. Vertex has already received reimbursement agreements for Kaftrio in EU countries, such as England, Denmark, the Republic of Ireland and Germany.

Shares of Vertex have rallied 20.9% so far this year against the industry’s decrease of 3%.

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 the potential to treat up to 90% of CF patients.

Vertex’s revenue growth in 2020 so far has primarily been 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 EU countries and approval for a younger patient population could bring additional Trikafta/Kaftrio revenues in 2021.

Zacks Rank & Stocks to Consider

Vertex currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the healthcare sector include Emergent BioSolutions Inc. EBS, Horizon Therapeutics HZNP and Alimera Sciences, Inc. ALIM. While Emergent BioSolutions and Horizon Therapeutics sport a Zacks Rank #1 (Strong Buy), Alimera Sciences carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Emergent’s earnings estimates have moved 65.5% and 21.7% north for 2020 and 2021, respectively, over the past 60 days. The stock has skyrocketed 93.1% year to date.

Horizon’s earnings estimates have been revised 59% and 55.6% upward for 2020 and 2021 each over the past 60 days. The stock has rallied 111.6% year to date.

Alimera’s loss estimates have narrowed 43.8% and 17.5% for 2020 and 2021, respectively, over the past 60 days.

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