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Vertex Gets FDA Nod for Expanded Use of Casgevy in SCD & TDT
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Key Takeaways
Vertex gained FDA approval to expand Casgevy to patients aged two and older with SCD or TDT.
VRTX's Casgevy is the first genetic therapy approved for children as young as two with both disorders.
VRTX said label expansion applications remain under review in the United Kingdom and Saudi Arabia.
Vertex Pharmaceuticals (VRTX - Free Report) announced that the FDA has approved its one-shot gene therapy, Casgevy (exagamglogene autotemcel), for the treatment of individuals aged two years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT).
Following the latest nod, Casgevy became the first and only genetic therapy to be approved for treating children as young as two years for both severe SCD and TDT, both inherited blood disorders.
Regulatory filings seeking approval for the label expansion of Casgevy are currently under review in the United Kingdom and the Kingdom of Saudi Arabia for the given indication.
Casgevy was previously approved for treating SCD and TDT in patients aged 12 years and older.
VRTX’s Price Performance
Year to date, shares of Vertex have rallied 9.8% compared with the industry’s increase of 7%.
Image Source: Zacks Investment Research
More on VRTX’s Ongoing Activities With Casgevy
We remind investors that Vertex leads the global development and commercialization of Casgevy under the terms of the 2021 agreement, with support from CRISPR Therapeutics (CRSP - Free Report) .
Per the collaboration agreement, Vertex splits the program costs and profits in a 60:40 ratio with CRISPR Therapeutics.
In the first quarter of 2026, Casgevy’s sales were $42.9 million, down from $54.3 million recorded in the fourth quarter of 2025 due to quarter-to-quarter variability in Casgevy infusions.
Nonetheless, Casgevy’s launch metrics look positive with growing cell collections and product infusions. Vertex is also making rapid progress in the drug’s access and reimbursement.
In May 2026, Vertex signed a reimbursement agreement with Germany’s GKV-Spitzenverband for Casgevy, ensuring sustainable patient access for eligible individuals aged 12 years and older with severe SCD or TDT.
In 2026, Vertex expects continued quarter-to-quarter variability in Casgevy infusions, which the company expects will smooth out in 2027 and beyond. If commercialization of Casgevy ramps up successfully over the next few years, Vertex Pharmaceuticals believes the therapy has multibillion-dollar commercial potential.
Over the past 60 days, estimates for Immunocore’s 2026 bottom line have improved from a loss of 88 cents per share to earnings of 6 cents. Over the same period, EPS estimates for 2027 have risen from 24 cents to 87 cents. IMCR stock has lost 8.8% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 46.66%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $1.50 to $3.02, while estimates for 2027 have increased from $2.91 to $4.92 during the same time. LQDA shares have surged 130% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%.
View the Zacks #1 Rank List
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Vertex Gets FDA Nod for Expanded Use of Casgevy in SCD & TDT
Key Takeaways
Vertex Pharmaceuticals (VRTX - Free Report) announced that the FDA has approved its one-shot gene therapy, Casgevy (exagamglogene autotemcel), for the treatment of individuals aged two years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT).
Following the latest nod, Casgevy became the first and only genetic therapy to be approved for treating children as young as two years for both severe SCD and TDT, both inherited blood disorders.
Regulatory filings seeking approval for the label expansion of Casgevy are currently under review in the United Kingdom and the Kingdom of Saudi Arabia for the given indication.
Casgevy was previously approved for treating SCD and TDT in patients aged 12 years and older.
VRTX’s Price Performance
Year to date, shares of Vertex have rallied 9.8% compared with the industry’s increase of 7%.
Image Source: Zacks Investment Research
More on VRTX’s Ongoing Activities With Casgevy
We remind investors that Vertex leads the global development and commercialization of Casgevy under the terms of the 2021 agreement, with support from CRISPR Therapeutics (CRSP - Free Report) .
Per the collaboration agreement, Vertex splits the program costs and profits in a 60:40 ratio with CRISPR Therapeutics.
In the first quarter of 2026, Casgevy’s sales were $42.9 million, down from $54.3 million recorded in the fourth quarter of 2025 due to quarter-to-quarter variability in Casgevy infusions.
Nonetheless, Casgevy’s launch metrics look positive with growing cell collections and product infusions. Vertex is also making rapid progress in the drug’s access and reimbursement.
In May 2026, Vertex signed a reimbursement agreement with Germany’s GKV-Spitzenverband for Casgevy, ensuring sustainable patient access for eligible individuals aged 12 years and older with severe SCD or TDT.
In 2026, Vertex expects continued quarter-to-quarter variability in Casgevy infusions, which the company expects will smooth out in 2027 and beyond. If commercialization of Casgevy ramps up successfully over the next few years, Vertex Pharmaceuticals believes the therapy has multibillion-dollar commercial potential.
VRTX Zacks Rank & Stocks to Consider
Vertex currently has a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Immunocore (IMCR - Free Report) and Liquidia Corporation (LQDA - Free Report) , each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Immunocore’s 2026 bottom line have improved from a loss of 88 cents per share to earnings of 6 cents. Over the same period, EPS estimates for 2027 have risen from 24 cents to 87 cents. IMCR stock has lost 8.8% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 46.66%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $1.50 to $3.02, while estimates for 2027 have increased from $2.91 to $4.92 during the same time. LQDA shares have surged 130% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%.