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Vertex Seeks Nod for Cystic Fibrosis Drugs for Rare Mutations

Vertex Pharmaceuticals Incorporated VRTX announced that the FDA has accepted its three supplemental new drug applications (sNDA) seeking approval of its cystic fibrosis (CF) drugs, Trikafta, Symdeko and Kalydeco for additional rare CFTR mutations. If approved for this expanded use, these drugs will be eligible to treat additional 600 CF patients.

Meanwhile, these sNDAs are also seeking approval for certain people with CF who are currently eligible for Kalydeco to become eligible for Symdeko or Trikafta as well as people currently eligible for Symdeko to become eligible for Trikafta.

The FDA is expected to give its decision on the sNDA on Dec 30, 2020.

The sNDAs are based on in-vitro data from a validated cell assay model. Data from the model showed that some rare mutations in the CFTR gene are responsive to one of more of the three medicines.

Vertex’s stock has risen 25.8% this year so far compared with an increase of 2.7% for the industry.



Other than Trikafta, Symdeko and Kalydeco, Vertex also markets Orkambi. These four CF medicines are collectively approved to treat around approximately 45,000 patients worldwide

Consistent positive regulatory approvals have led to an increase in the eligible patient population for Vertex’s approved medicines in the past 2-3 years. In 2019 alone, Vertex received nine new regulatory approvals or label expansions for its CF medicines globally. Also, in 2019, Vertex reached a number of key reimbursement agreements in important ex-U.S. countries like England and France, which expanded access to its CF medicines. With consistent expansion in the patient population, Vertex’s CF product revenues rose 29% in 2017, 40% in 2018 and 32% in 2019. Vertex’s revenue growth in 2020 is primarily being driven by the uptake of Trikafta as well as higher international revenues due to additional ex-U.S. reimbursement arrangements.

While Vertex’s main focus is on the development and strengthening of its CF franchise, the company also has a rapidly advancing early-stage portfolio in five other specialty disease areas like pain, alpha-1 antitrypsin deficiency (AAT), sickle cell disease, beta-thalassemia and APOL1-mediated kidney diseases. Vertex is co-developing a gene editing treatment, CTX001 in partnership with CRISPR Therapeutics CRSP in two devastating diseases — sickle cell disease and thalassemia.

Zacks Rank and Stocks to Consider

Vertex currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Some better-ranked stocks from the biotech sector include Emergent Biosolutions EBS and Horizon Therapeutics HZNP, both sporting a Zacks Rank #1.

Emergent Biosolutions’ earnings per share estimates have moved up from $4.30 to $6.32 for 2020 and from $4.97 to $7.01 for 2021 in the past 30 days. The stock has risen 105.4% so far this year.

Horizon Therapeutics’ earnings per share estimates have increased from $2.00 to $2.86 for 2020 and from $3.05 to $4.29 per share in the past 30 days. The stock has surged 108.5% so far this year.

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.

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Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report
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