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Vertex (VRTX), CRISPR Get Positive CHMP Opinion for Gene-Therapy
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Vertex Pharmaceuticals Incorporated (VRTX - Free Report) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (“EMA”) had adopted a positive opinion, recommending the conditional approval of Casgevy to treat severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
The CHMP’s recommendation, although not legally binding, will be considered by the EMA in its decision-making process for the approval of Casgevy in the EU for SCD and TDT. A final decision from the regulatory body is expected in February 2024.
Conditional marketing authorizations (CMAs) are usually granted to medicines that fulfill a significant unmet medical need with no satisfactory treatment methods or when a particular medicine offers a major therapeutic advantage over currently available treatments.
A CMA is granted when comprehensive clinical data is not yet complete, but the clinical benefit of the medicine outweighs the temporarily incomplete clinical data, which is poised to become available in the future. CMAs are valid for one year and renewable annually with ongoing regulatory review of data.
Casgevy, a one-time CRISPR/Cas9 gene-edited therapy, is being developed by Vertex in collaboration with CRISPR Therapeutics (CRSP - Free Report) for the SCD and TDT indication.
Subject to approval, Casgevy will become the only gene therapy for EU patients aged 12 years and older, suffering from either severe SCD with recurrent vaso-occlusive crises (VOCs) or TDT, who are eligible for hematopoietic stem cell (HSC) transplantation when an HSC donor is not available.
Year to date, shares of Vertex have gained 42.2% against the industry’s 17.7% decline.
Image Source: Zacks Investment Research
SCD is a group of inherited blood disorders. It is a complex and progressive genetic disease that generally leads to unpredictable and debilitating VOCs. High concentrations of sickle hemoglobin in red blood cells cause them to become misshapen, sticky and rigid, with a shorter life span, which manifests acutely as hemolytic anemia, vasculopathy and vaso-occlusion.
TDT is also a life-threatening genetic disease that takes a severe toll on the quality of life of patients living with it. TDT patients require frequent blood transfusions and iron chelation therapy throughout their life. In addition, the complications associated with TDT result in reduced life expectancy.
We would like to remind the investors that in November 2023, Vertex and CRISPR first received conditional approval for Casgevy in the UK to treat both SCD and TDT. Casgevy is Vertex’s first approved therapy outside of its marketed portfolio of cystic fibrosis drugs and its first-ever FDA-approved therapy for CRISPR.
This month, Casgevy was also approved by the FDA to be marketed in the United States for the treatment of SCD. CRISPR and Vertex are also developing Casgevy for the treatment of TDT in the United States. A regulatory filing for the use of Casgevy for this indication is currently under review. A decision from the FDA is expected on Mar 30, 2024.
Vertex Pharmaceuticals Incorporated Price and Consensus
The FDA had simultaneously approved another gene therapy for the treatment of SCD, namely Lyfgenia (lovo-cel), developed by bluebird bio (BLUE - Free Report) .
Among the two gene therapies, the approval of Vertex/CRISPR Casgevy was more impressive since it marks the first time that the FDA approved a gene therapy utilizing the Nobel prize-winning CRISPR technology.
The gene therapy drugs, however, come with a hefty price tag, which may limit patient access. Vertex and CRISPR disclosed that they would commercially launch Casgevy at $2.1 million. In a separate press release, bluebird bio announced that it will launch Lyfgenia at $3.1 million.
Furthermore, the investors were also worried by the long and arduous treatment process of the gene therapy drugs, along with several side effects, including low levels of platelets, white blood cells and fertility problems.
bluebird bio‘s Lyfgenia label includes a black box warning for hematologic malignancy (blood cancer). Patients infused with bluebird bio’s therapy are required to be monitored for this malignancy for life.
Notably, last week, Vertex announced entering into a licensing agreement with Editas Medicine, Inc. (EDIT - Free Report) for further development of Casgevy. The agreement grants Vertex a non-exclusive license to utilize Editas’ Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of SCD and TDT, including Casgevy.
The Cas9 gene editing tool will provide Vertex access to a broad range of genetic mutations, which makes it possible to develop innovative gene-editing medicines with a novel mechanism of action.
Editas Medicine is currently developing its gene therapy candidate, EDIT 301, now known as renizgamglogene autogedtemcel (reni-cel), as a potential one-time, durable gene editing medicine to treat SCD and TDT. The candidate is currently being developed in two separate early-mid-stage studies, RUBY and EdiTHAL, to treat SCD and TDT, respectively.
Image: Bigstock
Vertex (VRTX), CRISPR Get Positive CHMP Opinion for Gene-Therapy
Vertex Pharmaceuticals Incorporated (VRTX - Free Report) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (“EMA”) had adopted a positive opinion, recommending the conditional approval of Casgevy to treat severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
The CHMP’s recommendation, although not legally binding, will be considered by the EMA in its decision-making process for the approval of Casgevy in the EU for SCD and TDT. A final decision from the regulatory body is expected in February 2024.
Conditional marketing authorizations (CMAs) are usually granted to medicines that fulfill a significant unmet medical need with no satisfactory treatment methods or when a particular medicine offers a major therapeutic advantage over currently available treatments.
A CMA is granted when comprehensive clinical data is not yet complete, but the clinical benefit of the medicine outweighs the temporarily incomplete clinical data, which is poised to become available in the future. CMAs are valid for one year and renewable annually with ongoing regulatory review of data.
Casgevy, a one-time CRISPR/Cas9 gene-edited therapy, is being developed by Vertex in collaboration with CRISPR Therapeutics (CRSP - Free Report) for the SCD and TDT indication.
Subject to approval, Casgevy will become the only gene therapy for EU patients aged 12 years and older, suffering from either severe SCD with recurrent vaso-occlusive crises (VOCs) or TDT, who are eligible for hematopoietic stem cell (HSC) transplantation when an HSC donor is not available.
Year to date, shares of Vertex have gained 42.2% against the industry’s 17.7% decline.
Image Source: Zacks Investment Research
SCD is a group of inherited blood disorders. It is a complex and progressive genetic disease that generally leads to unpredictable and debilitating VOCs. High concentrations of sickle hemoglobin in red blood cells cause them to become misshapen, sticky and rigid, with a shorter life span, which manifests acutely as hemolytic anemia, vasculopathy and vaso-occlusion.
TDT is also a life-threatening genetic disease that takes a severe toll on the quality of life of patients living with it. TDT patients require frequent blood transfusions and iron chelation therapy throughout their life. In addition, the complications associated with TDT result in reduced life expectancy.
We would like to remind the investors that in November 2023, Vertex and CRISPR first received conditional approval for Casgevy in the UK to treat both SCD and TDT. Casgevy is Vertex’s first approved therapy outside of its marketed portfolio of cystic fibrosis drugs and its first-ever FDA-approved therapy for CRISPR.
This month, Casgevy was also approved by the FDA to be marketed in the United States for the treatment of SCD. CRISPR and Vertex are also developing Casgevy for the treatment of TDT in the United States. A regulatory filing for the use of Casgevy for this indication is currently under review. A decision from the FDA is expected on Mar 30, 2024.
Vertex Pharmaceuticals Incorporated Price and Consensus
Vertex Pharmaceuticals Incorporated price-consensus-chart | Vertex Pharmaceuticals Incorporated Quote
The FDA had simultaneously approved another gene therapy for the treatment of SCD, namely Lyfgenia (lovo-cel), developed by bluebird bio (BLUE - Free Report) .
Among the two gene therapies, the approval of Vertex/CRISPR Casgevy was more impressive since it marks the first time that the FDA approved a gene therapy utilizing the Nobel prize-winning CRISPR technology.
The gene therapy drugs, however, come with a hefty price tag, which may limit patient access. Vertex and CRISPR disclosed that they would commercially launch Casgevy at $2.1 million. In a separate press release, bluebird bio announced that it will launch Lyfgenia at $3.1 million.
Furthermore, the investors were also worried by the long and arduous treatment process of the gene therapy drugs, along with several side effects, including low levels of platelets, white blood cells and fertility problems.
bluebird bio‘s Lyfgenia label includes a black box warning for hematologic malignancy (blood cancer). Patients infused with bluebird bio’s therapy are required to be monitored for this malignancy for life.
Notably, last week, Vertex announced entering into a licensing agreement with Editas Medicine, Inc. (EDIT - Free Report) for further development of Casgevy. The agreement grants Vertex a non-exclusive license to utilize Editas’ Cas9 gene editing technology for ex vivo gene editing medicines targeting the BCL11A gene in the fields of SCD and TDT, including Casgevy.
The Cas9 gene editing tool will provide Vertex access to a broad range of genetic mutations, which makes it possible to develop innovative gene-editing medicines with a novel mechanism of action.
Editas Medicine is currently developing its gene therapy candidate, EDIT 301, now known as renizgamglogene autogedtemcel (reni-cel), as a potential one-time, durable gene editing medicine to treat SCD and TDT. The candidate is currently being developed in two separate early-mid-stage studies, RUBY and EdiTHAL, to treat SCD and TDT, respectively.
Zacks Rank
Vertex currently has a Zacks Rank #3 (Hold).
You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.