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Vertex's Kalydeco Gets FDA Nod for Cystic Fibrosis in Infants

Zacks Equity Research
·3 min read

Vertex Pharmaceuticals Incorporated VRTX announced that the FDA has approved its cystic fibrosis (CF) drug Kalydeco (ivacaftor) for an expanded use in infants aged between four and less than six months with at least one mutation in their CFTR gene that is responsive to Kalydeco.

Following this nod, Kalydeco became the first and the only CFTR modulator approved to treat eligible infants with CF as early as four months of age as they can now access the medicine to treat the underlying cause of CF.

The FDA approval was based on data from a cohort in the 24-week phase III open-label safety cohort named (ARRIVAL), which evaluated six eligible infants with CF, aged from four months to less than six months, having one of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). Per the company, the safety profile demonstrated in this cohort was similar to the response observed in older children and adults.

Kalydeco is already marketed for the treatment of CF in patients aged between six months and above in the United States and Europe.

Shares of Vertex have rallied 22.4% so far this year compared with the industry’s increase of 1.4%.


We note that Vertex markets four CF medicines, namely Kalydeco, Orkambi, Symdeko/Symkevi and Trikafta. Approximately 45,000 patients worldwide are now eligible to be treated with one of Vertex’s four CF medicines.

Notably, Trikafta is crucial for Vertex’s long-term growth as it has the potential to treat up to 90% of CF patients. Trikafta was approved under the trade name of Kaftrio in the EU during August. The drug generated sales worth $1.8 billion in the first half of 2020. It has seen a solid uptake since its early unveiling and has been a key driver for the company ever since.

Meanwhile, phase III studies are ongoing to evaluate Trikafta in children aged six to 11 years. An sNDA seeking approval for the product among this pediatric patient population is expected to be filed in the fourth quarter of 2020. The recent nod in Europe and a potential label expansion of the drug for younger patient population could bring additional revenues for Vertex this year as well as 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 presently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Emergent’s earnings estimates have been revised 55.5% upward for 2020 and 37.3% for 2021 over the past 60 days. The stock has surged 88.4% year to date.

Horizon Therapeutics’ earnings estimates have moved 46.7% north for 2020 and 49.1% for 2021 over the past 60 days. The stock has skyrocketed 116.2% year to date.

QIAGEN’s earnings estimates have been revised 10.6% upward for 2020 and 22.5% for 2021 over the past 60 days. The stock has soared 53.6% year to date.

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QIAGEN N.V. (QGEN) : Free Stock Analysis Report
 
Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report
 
Emergent Biosolutions Inc. (EBS) : Free Stock Analysis Report
 
Horizon Therapeutics Public Limited Company (HZNP) : Free Stock Analysis Report
 
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