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Vertex's Kalydeco Gets EU Approval to Treat CF in Infants

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Vertex Pharmaceuticals Inc. VRTX announced that the European Commission has granted marketing authorization to its cystic fibrosis (CF) drug Kalydeco (ivacaftor) for expanded use in infants aged from six to less than 12 months with at least one of specified nine mutations in the CFTR gene.

Following this nod, CF patients as young as six months in Europe can now have access to Kalydeco to treat the underlying cause of CF.

Kalydeco was approved by the FDA to address the same patient population in April 2019.

The approval was based on data from an ongoing open-label phase III study, ARRIVAL, which evaluated children aged less than 24 months with a CFTR gating mutation.

The safety profile of the study was similar to the observations in earlier phase III studies of older children and adult CF patients, which demonstrated improvements in sweat chloride, indicating a secondary endpoint.

Notably, in Europe, Kalydeco was until now approved to treat CF in children aged 12 months and older with at least one of specified nine mutations in the CFTR gene. The drug is also approved for CF patients aged 18 years and above with R117H mutation in the CFTR gene.

Shares of Vertex have surged 32.9% so far this year compared with the industry’s increase of 6.1%.

We would like to remind investors that Kalydeco along with Vertex’s two other CF medicines, namely Orkambi and Symdeko (known as Symkevi in Europe) are collectively approved for treating a good number of CF patients in North America, Europe and Australia. Total CF product revenues in the first nine months of 2019 were $2.7 billion, reflecting a rise of approximately 23% year over year.

Meanwhile, in October 2019, Vertex gained FDA approval for its fourth medicine Trikafta to treat CF. The drug is a triple combination regimen (elexacaftor/tezacaftor/ivacaftor and ivacaftor). With this approval, the company can address a significantly larger CF patient population — almost 90% patients — ahead. With the nod to Trikafta, approximately 45,000 patients worldwide are now eligible to be treated with one of Vertex’s four CF medicines.

Several companies like AbbVie (ABBV - Free Report) , Eloxx Pharmaceuticals, ProQR Therapeutics PRQR and Proteostasis Therapeutics are developing medicines to treat CF. Even though Vertex enjoys a strong position in this market, stiff competition due to entry of additional products would erode its revenues.

Zacks Rank & Other Key Pick

Vertex currently carries a Zacks Rank #2 (Buy). Another top-ranked stock in the biotech sector is Anika Therapeutics Inc. ANIK, sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Anika’s earnings estimates have moved 16% north for 2019 and 17.4% for 2020 over the past 60 days. The stock has soared 64.9% so far this year.

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