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Vertex (VRTX), CRSP's Casgevy Gets FDA Nod for Thalassemia
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Vertex Pharmaceuticals (VRTX - Free Report) , along with CRISPR Therapeutics (CRSP - Free Report) , announced that the FDA expanded the label of their one-shot gene therapy Casgevy to treat transfusion-dependent beta thalassemia (TDT) in patients aged 12 years and older.
The approval comes just a month after the FDA approved the therapy for sickle cell disease (SCD) in the same age group. Following this initial approval, Casgevy became the first gene therapy utilizing the Nobel prize-winning CRISPR technology. This technology can selectively delete, modify or correct a disease-causing abnormality in a specific DNA segment.
This decision also came two months early, as the agency had previously set the PDUFA date of Mar 30. CRISPR/Vertex claim that the approval of Casgevy provides a curative treatment option for TDT patients.
Vertex and CRISPR will charge $2.2 million for the one-time treatment, which is the same amount charged by the companies for SCD treatment. The companies believe that this pricing is justified as a TDT patient living in the United States spends more than $5 million in his lifetime in healthcare costs.
In the past year, shares of Vertex and CRISPR have risen 41.2% and 25.5%, respectively, against the industry’s decline of 12.2%.
Image Source: Zacks Investment Research
An inherited blood disorder, TDT is marked by life-long anemia wherein the body does not have enough healthy red blood cells (RBCs). The lack of RBCs leads to reduced oxygen flow in the blood, with patients likely to experience multiple complications, including fatigue and shortness of breath. TDT patients require life-long treatment with frequent blood transfusions.
The Vertex/CRISPR therapy utilizes a patient’s blood stem cells, which are subsequently modified and then infused as a one-time single-shot treatment as part of a hematopoietic (blood) stem cell transplant. However, the process of collecting stem cells requires the use of high-dose chemotherapy, and modifying stem cells can take up to six months alone.
Currently, the only options available to TDT patients are stem cell transplants from a matched donor and the FDA-approved gene therapy Zyntelgo marketed by bluebird bio (BLUE - Free Report) . We remind investors that bluebird’s Zyntelgo was approved by the FDA in 2022 as the first gene therapy for TDT.
Apart from Zyntelgo, Vertex/CRISPR also faces stiff competition from bluebird in the SCD space. Alongside Casgevy’s approval in SCD, the FDA also approved bluebird’s Lyfgenia gene therapy for SCD indication.
The Vertex/CRISPR announced list price of $2.2 million is likely to give Casgevy an edge over bluebird bio, whose therapies are priced at around $3 million. However, bluebird bio’s experience with marketing gene therapies gives it a significant advantage over Vertex/CRISPR. Apart from Zyntelgo and Lyfgenia, bluebird also markets Skysona for active cerebral adrenoleukodystrophy.
In the past 60 days, Sarepta’s loss estimates for 2023 have improved from a loss of $6.95 per share to $6.55 per share. During the same period, earnings estimates per share for 2024 have risen from 96 cents to $2.01. Sarepta’s shares have lost 10.4% in the past year.
Sarepta’s earnings beat estimates in each of the last four quarters, delivering an average surprise of 48.67%. In the last reported quarter, Sarepta’s earnings beat estimates by 72.29%.
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Vertex (VRTX), CRSP's Casgevy Gets FDA Nod for Thalassemia
Vertex Pharmaceuticals (VRTX - Free Report) , along with CRISPR Therapeutics (CRSP - Free Report) , announced that the FDA expanded the label of their one-shot gene therapy Casgevy to treat transfusion-dependent beta thalassemia (TDT) in patients aged 12 years and older.
The approval comes just a month after the FDA approved the therapy for sickle cell disease (SCD) in the same age group. Following this initial approval, Casgevy became the first gene therapy utilizing the Nobel prize-winning CRISPR technology. This technology can selectively delete, modify or correct a disease-causing abnormality in a specific DNA segment.
This decision also came two months early, as the agency had previously set the PDUFA date of Mar 30. CRISPR/Vertex claim that the approval of Casgevy provides a curative treatment option for TDT patients.
Vertex and CRISPR will charge $2.2 million for the one-time treatment, which is the same amount charged by the companies for SCD treatment. The companies believe that this pricing is justified as a TDT patient living in the United States spends more than $5 million in his lifetime in healthcare costs.
In the past year, shares of Vertex and CRISPR have risen 41.2% and 25.5%, respectively, against the industry’s decline of 12.2%.
Image Source: Zacks Investment Research
An inherited blood disorder, TDT is marked by life-long anemia wherein the body does not have enough healthy red blood cells (RBCs). The lack of RBCs leads to reduced oxygen flow in the blood, with patients likely to experience multiple complications, including fatigue and shortness of breath. TDT patients require life-long treatment with frequent blood transfusions.
The Vertex/CRISPR therapy utilizes a patient’s blood stem cells, which are subsequently modified and then infused as a one-time single-shot treatment as part of a hematopoietic (blood) stem cell transplant. However, the process of collecting stem cells requires the use of high-dose chemotherapy, and modifying stem cells can take up to six months alone.
Currently, the only options available to TDT patients are stem cell transplants from a matched donor and the FDA-approved gene therapy Zyntelgo marketed by bluebird bio (BLUE - Free Report) . We remind investors that bluebird’s Zyntelgo was approved by the FDA in 2022 as the first gene therapy for TDT.
Apart from Zyntelgo, Vertex/CRISPR also faces stiff competition from bluebird in the SCD space. Alongside Casgevy’s approval in SCD, the FDA also approved bluebird’s Lyfgenia gene therapy for SCD indication.
The Vertex/CRISPR announced list price of $2.2 million is likely to give Casgevy an edge over bluebird bio, whose therapies are priced at around $3 million. However, bluebird bio’s experience with marketing gene therapies gives it a significant advantage over Vertex/CRISPR. Apart from Zyntelgo and Lyfgenia, bluebird also markets Skysona for active cerebral adrenoleukodystrophy.
Vertex Pharmaceuticals Incorporated Price
Vertex Pharmaceuticals Incorporated price | Vertex Pharmaceuticals Incorporated Quote
CRISPR Therapeutics AG Price
CRISPR Therapeutics AG price | CRISPR Therapeutics AG Quote
Zacks Rank & A Key Pick
While Vertex carries a Zacks Rank #2 (Buy), CRISPR carries a Zacks Rank #3 (Hold). A better-ranked stock in the overall healthcare sector is Sarepta Therapeutics (SRPT - Free Report) , which also carries a Zacks Rank #2. You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.
In the past 60 days, Sarepta’s loss estimates for 2023 have improved from a loss of $6.95 per share to $6.55 per share. During the same period, earnings estimates per share for 2024 have risen from 96 cents to $2.01. Sarepta’s shares have lost 10.4% in the past year.
Sarepta’s earnings beat estimates in each of the last four quarters, delivering an average surprise of 48.67%. In the last reported quarter, Sarepta’s earnings beat estimates by 72.29%.