We use cookies to understand how you use our site and to improve your experience. This includes personalizing content and advertising. To learn more, click here. By continuing to use our site, you accept our use of cookies, revised Privacy Policy and Terms of Service.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
Vertex's Kalydeco Wins Approval in EU for CF in Children
Read MoreHide Full Article
Vertex Pharmaceuticals Incorporated (VRTX - Free Report) announced that the European Commission has granted marketing authorization to its cystic fibrosis (CF) drug Kalydeco (ivacaftor) for expanded use in children and adolescents aged from six months to 18 years with the R117H mutation in the CFTR gene, the most common residual function mutation.
Following this nod, CF patients aged between six months and 18 years with the R117H mutation in Europe can now access Kalydeco to treat the underlying cause of CF.
Per the press release, Kalydeco will be immediately made available to additional eligible patients in Germany and shortly across countries where respective long-term reimbursement contracts were previously secured.
Notably, In May 2020, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended Kalydeco’s label expansion for the same patient population.
In Europe, Kalydeco was until now approved to treat CF in children aged six months and older with at least one of the specified nine mutations in the CFTR gene. The drug also got a regulatory nod for CF patients aged 18 years and above with R117H mutation in the CFTR gene.
Shares of Vertex have rallied 23.7% so far this year compared with the industry’s increase of 7.9%.
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.
Trikafta is crucial for Vertex’s long-term growth as it has the potential to treat up to 90% of CF patients. The drug is under review in Europe with an approval expected later in 2020.
Meanwhile, phase III studies are also ongoing to evaluate Trikafta in children aged from six to 11 years. An sNDA seeking approval for the pediatric patient population is expected to be filed in 2020. A potential approval of Trikafta in the EU and for the pediatric patients in the United States could bring additional revenues for Vertex in 2020.
Several companies like AbbVie (ABBV - Free Report) , Eloxx Pharmaceuticals and Proteostasis Therapeutics are also developing medicines to treat CF. Even though Vertex enjoys a strong position in the said market, stiff competition due to entry of additional products may erode its revenues.
Zacks Rank & Another Key Pick
Vertex currently sports a Zacks Rank #1 (Strong Buy). Another top-ranked stock in the biotech sector is Ligand Pharmaceuticals Incorporated , which flaunts the same Zacks Rank as Vertex. You can see the complete list of today’s Zacks #1 Rank stocks here.
Ligand’s earnings estimates have been revised 7.2% upward for 2020 and 10% for 2021 over the past 60 days. The stock has rallied 18.6% year to date.
Breakout Biotech Stocks with Triple-Digit Profit Potential
The biotech sector is projected to surge beyond $775 billion by 2024 as scientists develop treatments for thousands of diseases. They’re also finding ways to edit the human genome to literally erase our vulnerability to these diseases.
Zacks has just released Century of Biology: 7 Biotech Stocks to Buy Right Now to help investors profit from 7 stocks poised for outperformance. Our recent biotech recommendations have produced gains of +50%, +83% and +164% in as little as 2 months. The stocks in this report could perform even better.
Image: Bigstock
Vertex's Kalydeco Wins Approval in EU for CF in Children
Vertex Pharmaceuticals Incorporated (VRTX - Free Report) announced that the European Commission has granted marketing authorization to its cystic fibrosis (CF) drug Kalydeco (ivacaftor) for expanded use in children and adolescents aged from six months to 18 years with the R117H mutation in the CFTR gene, the most common residual function mutation.
Following this nod, CF patients aged between six months and 18 years with the R117H mutation in Europe can now access Kalydeco to treat the underlying cause of CF.
Per the press release, Kalydeco will be immediately made available to additional eligible patients in Germany and shortly across countries where respective long-term reimbursement contracts were previously secured.
Notably, In May 2020, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended Kalydeco’s label expansion for the same patient population.
In Europe, Kalydeco was until now approved to treat CF in children aged six months and older with at least one of the specified nine mutations in the CFTR gene. The drug also got a regulatory nod for CF patients aged 18 years and above with R117H mutation in the CFTR gene.
Shares of Vertex have rallied 23.7% so far this year compared with the industry’s increase of 7.9%.
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.
Trikafta is crucial for Vertex’s long-term growth as it has the potential to treat up to 90% of CF patients. The drug is under review in Europe with an approval expected later in 2020.
Meanwhile, phase III studies are also ongoing to evaluate Trikafta in children aged from six to 11 years. An sNDA seeking approval for the pediatric patient population is expected to be filed in 2020. A potential approval of Trikafta in the EU and for the pediatric patients in the United States could bring additional revenues for Vertex in 2020.
Several companies like AbbVie (ABBV - Free Report) , Eloxx Pharmaceuticals and Proteostasis Therapeutics are also developing medicines to treat CF. Even though Vertex enjoys a strong position in the said market, stiff competition due to entry of additional products may erode its revenues.
Zacks Rank & Another Key Pick
Vertex currently sports a Zacks Rank #1 (Strong Buy). Another top-ranked stock in the biotech sector is Ligand Pharmaceuticals Incorporated , which flaunts the same Zacks Rank as Vertex. You can see the complete list of today’s Zacks #1 Rank stocks here.
Ligand’s earnings estimates have been revised 7.2% upward for 2020 and 10% for 2021 over the past 60 days. The stock has rallied 18.6% year to date.
Breakout Biotech Stocks with Triple-Digit Profit Potential
The biotech sector is projected to surge beyond $775 billion by 2024 as scientists develop treatments for thousands of diseases. They’re also finding ways to edit the human genome to literally erase our vulnerability to these diseases.
Zacks has just released Century of Biology: 7 Biotech Stocks to Buy Right Now to help investors profit from 7 stocks poised for outperformance. Our recent biotech recommendations have produced gains of +50%, +83% and +164% in as little as 2 months. The stocks in this report could perform even better.
See these 7 breakthrough stocks now>>