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Recent popular reports from Yimaik★Review of the regulatory policy of cell therapy in 2020, cell therapy is on the right trackNew observations from Yimaik★Hair loss? Don’t get hair transplants, let’s take a look at the magic of stem cell therapyMedClub New Observation March 10, 2021/MedClub News/--March 8, 2021, BioMarin announced that the US Food and Drug Administration (FDA) awarded the company valoctocogene roxaparvovec (hereinafter referred to as: valrox) Regenerative Medicine Advanced Therapy Designation (RMAT), a gene therapy candidate product for hemophilia A.
RMAT is a supplement to the breakthrough therapy name obtained by the company in 2017.
RMAT aims to accelerate the development of new regenerative therapies.
Cell therapies that have been certified by RMAT have also obtained the benefits of fast track qualification and breakthrough therapy certification.
These advantages include the FDA’s early guidance for manufacturing and clinical development, such as identifying intermediate endpoints that support accelerated approval.
In the United States, treatments for rare diseases that affect less than 200,000 people will be granted ODD designation.
If a drug granted ODD obtains the first FDA approval under the specified conditions, the drug is eligible for 7 years of market exclusivity.
It is worth mentioning that valrox was awarded the RMAT title, just in the "Bleeding Disorders Awareness Month" (Bleeding Disorders Awareness Month), which was initiated by the National Hemophilia Foundation to celebrate and commemorate the bleeding disorder community .
In August 2020, the FDA rejected BioMarin’s application for the marketing of hemophilia A gene therapy because of the fluctuations in the average annualized bleeding rate (ABR) in the Phase 1/2 study data published by BioMarin and the patients The level of factor VIII in the body continues to decline, and the FDA believes that this requires more complete follow-up data.
The FDA hopes that BioMarin can complete the Phase 3 study of valrox and read the complete two-year follow-up data.
However, according to the press release, the last patient of the valrox phase 3 study was enrolled in November 2019, and the final data cannot be read until November 2021.
▲ Image source: 3-year ABR of two cohorts of patients in the Phase 1/2 study (Image source: BioMarin official website) This news caused BioMarin's stock to fall by 35.
28% on the same day, and its market value evaporated by 7 billion US dollars.
Although based on the disclosure, the reason for the FDA's refusal is not to question the safety and effectiveness of valoctocogene roxaparvovec (hereinafter referred to as: valrox), but its tolerability.
According to the requirements of the US FDA, the launch schedule of valrox is expected to be postponed to 2022.
Subsequently, BioMarin withdrew valrox's European listing application.
In January 2021, BioMarin announced the 1-year follow-up data of the global phase 3 clinical study of valrox.
The data showed that the expression level of endogenous FVIII decreased slowly compared with the phase 1/2 study and remained within a certain range.
Provides hemostatic effect inside.
It also shows that valrox is well tolerated.
Hemophilia A gene therapy track, you catch up with me.
In recent years, thanks to advances in gene therapy and protein engineering, gene therapy has achieved significant breakthroughs in the clinical application of hemophilia, and the drug market for hemophilia is also growing.
Significant changes have taken place.
According to the latest report published by Coherent Market Insights, the hemophilia gene therapy market will exceed US$780 million in 2026, with a compound annual growth rate (CAGR) of 56.
0% from 2018 to 2026. At present, many companies in the world are deploying hemophilia A gene therapy products.
The following examples are some candidate products that are progressing quickly: Pfizer/Sangamo: Pfizer/Sangamo's hemophilia A candidate gene therapy SB-525 ( (Or PF-07055480) has entered the phase 3 clinical stage, and will evaluate the efficacy and safety of SB-525 treatment for patients with moderate to severe hemophilia A.
The observation period of the study is 5 years to further evaluate the durability and effectiveness of this gene therapy.
Roche/Spark: At the 14th European Association for Hemophilia and Related Diseases (EAHAD 2020) Annual Meeting in 2021, Spark Therapeutics, a subsidiary of Roche, submitted a preliminary clinical trial of its candidate gene therapy SPK-8016 for hemophilia A data.
SPK-8016 is a gene therapy based on the AAV vector expressing coagulation factor VIII developed by Spark.
The data released this time comes from the ongoing phase 1/2 study of an open-label, non-random, dose-escalation study, which aims to To evaluate the safety and effectiveness of SPK-8016 in adult male patients with severe hemophilia A and no history of anti-factor VIII inhibitors.
Novo Nordisk/bluebird: In October 2019, Novo Nordisk and bluebird jointly announced a three-year cooperation agreement to develop next-generation genome editing therapies for the treatment of genetic diseases.
The focus of the cooperation will be to use bluebird's mRNA-based homing endonuclease gene editing technology-megaTAL technology to develop gene therapy for hemophilia A.
Recommended reading: Novo Nordisk and bluebird have reached a cooperation on the development of hemophilia gene therapy, and major pharmaceutical companies continue to inject fresh blood | Yimai Meng broke the news Bayer: Bayer's hemophilia A gene therapy DTX201 The phase 1/2 clinical trial of (AAVhu37 FVIII) also achieved positive results.
The preliminary data published on SAH 2020 showed that DTX201 can safely and effectively increase the level of coagulation factor VIII (FVIII), and prevent or reduce the level of coagulation factor VIII (FVIII) in clinical trials.
The bleeding status of the first two patients with severe hemophilia A. Bayer has also established a joint venture called Casebia Therapeutics with the Swiss company CRISPR Therapeutics to try new treatments for hemophilia based on CRISPR gene editing technology.
Recommended reading: ASH2019: Bayer's AAV Gene Therapy has positive results in Phase 1/2 clinical trials in hemophilia ARegeneron reveals that Regeneron: Regeneron announces that it will deliver a prepayment of US$70 million to Intellia Therapeutics Funds and 30 million US dollars of equity investment, totaling 100 million US dollars, to expand the cooperation between the two parties since 2016.
The main focus this time is to develop potential CRISPR/Cas9 editing gene therapy products for the treatment of hemophilia A and hemophilia B.
In preclinical research, the company demonstrated the first CRISPR/Cas9-mediated targeted transgene insertion in the liver of non-human primates, which can produce normal or higher levels of circulating human factor IX.
Factor IX is a missing or defective coagulation protein in patients with hemophilia B.
These results indicate that the transgene insertion can provide the functional factor 9 gene, which encodes an important protein.
▲ Picture source: Intellia official website.
At present, gene therapy has achieved relatively positive results in blood diseases, and gene therapy products have been successfully approved for marketing.
In 2022, both Pfizer/Sangamo and Roche/Spark will turn their attention to gene therapy.
In this regard, the development prospects are also worth looking forward to.
And at the upcoming 2021 BPIT Biopharmaceutical Innovation Technology Conference in Shanghai in April, Dong Biao, Chairman and General Manager of Zhishan Weixin, will give a speech on the process development strategy of rAAV gene medicine for hemophilia A.
Let us Focus on hot topics in the field of biomedicine together, and focus on new trends and developments in the industry together.
The Biopharmaceutical Innovation Technology Conference (BPIT) will kick off in Shanghai from April 9th to 10th, 2021.
The four major conference venues are exciting (click to view the focus topics). Participants who successfully registered for the Shanghai 2021 BPIT conference will receive a limited edition of "CAR-T Cell Therapy Industry Research Report" 2021.
V3.
1 produced by Xingyao Research Institute under Medical Media.
RMAT is a supplement to the breakthrough therapy name obtained by the company in 2017.
RMAT aims to accelerate the development of new regenerative therapies.
Cell therapies that have been certified by RMAT have also obtained the benefits of fast track qualification and breakthrough therapy certification.
These advantages include the FDA’s early guidance for manufacturing and clinical development, such as identifying intermediate endpoints that support accelerated approval.
In the United States, treatments for rare diseases that affect less than 200,000 people will be granted ODD designation.
If a drug granted ODD obtains the first FDA approval under the specified conditions, the drug is eligible for 7 years of market exclusivity.
It is worth mentioning that valrox was awarded the RMAT title, just in the "Bleeding Disorders Awareness Month" (Bleeding Disorders Awareness Month), which was initiated by the National Hemophilia Foundation to celebrate and commemorate the bleeding disorder community .
In August 2020, the FDA rejected BioMarin’s application for the marketing of hemophilia A gene therapy because of the fluctuations in the average annualized bleeding rate (ABR) in the Phase 1/2 study data published by BioMarin and the patients The level of factor VIII in the body continues to decline, and the FDA believes that this requires more complete follow-up data.
The FDA hopes that BioMarin can complete the Phase 3 study of valrox and read the complete two-year follow-up data.
However, according to the press release, the last patient of the valrox phase 3 study was enrolled in November 2019, and the final data cannot be read until November 2021.
▲ Image source: 3-year ABR of two cohorts of patients in the Phase 1/2 study (Image source: BioMarin official website) This news caused BioMarin's stock to fall by 35.
28% on the same day, and its market value evaporated by 7 billion US dollars.
Although based on the disclosure, the reason for the FDA's refusal is not to question the safety and effectiveness of valoctocogene roxaparvovec (hereinafter referred to as: valrox), but its tolerability.
According to the requirements of the US FDA, the launch schedule of valrox is expected to be postponed to 2022.
Subsequently, BioMarin withdrew valrox's European listing application.
In January 2021, BioMarin announced the 1-year follow-up data of the global phase 3 clinical study of valrox.
The data showed that the expression level of endogenous FVIII decreased slowly compared with the phase 1/2 study and remained within a certain range.
Provides hemostatic effect inside.
It also shows that valrox is well tolerated.
Hemophilia A gene therapy track, you catch up with me.
In recent years, thanks to advances in gene therapy and protein engineering, gene therapy has achieved significant breakthroughs in the clinical application of hemophilia, and the drug market for hemophilia is also growing.
Significant changes have taken place.
According to the latest report published by Coherent Market Insights, the hemophilia gene therapy market will exceed US$780 million in 2026, with a compound annual growth rate (CAGR) of 56.
0% from 2018 to 2026. At present, many companies in the world are deploying hemophilia A gene therapy products.
The following examples are some candidate products that are progressing quickly: Pfizer/Sangamo: Pfizer/Sangamo's hemophilia A candidate gene therapy SB-525 ( (Or PF-07055480) has entered the phase 3 clinical stage, and will evaluate the efficacy and safety of SB-525 treatment for patients with moderate to severe hemophilia A.
The observation period of the study is 5 years to further evaluate the durability and effectiveness of this gene therapy.
Roche/Spark: At the 14th European Association for Hemophilia and Related Diseases (EAHAD 2020) Annual Meeting in 2021, Spark Therapeutics, a subsidiary of Roche, submitted a preliminary clinical trial of its candidate gene therapy SPK-8016 for hemophilia A data.
SPK-8016 is a gene therapy based on the AAV vector expressing coagulation factor VIII developed by Spark.
The data released this time comes from the ongoing phase 1/2 study of an open-label, non-random, dose-escalation study, which aims to To evaluate the safety and effectiveness of SPK-8016 in adult male patients with severe hemophilia A and no history of anti-factor VIII inhibitors.
Novo Nordisk/bluebird: In October 2019, Novo Nordisk and bluebird jointly announced a three-year cooperation agreement to develop next-generation genome editing therapies for the treatment of genetic diseases.
The focus of the cooperation will be to use bluebird's mRNA-based homing endonuclease gene editing technology-megaTAL technology to develop gene therapy for hemophilia A.
Recommended reading: Novo Nordisk and bluebird have reached a cooperation on the development of hemophilia gene therapy, and major pharmaceutical companies continue to inject fresh blood | Yimai Meng broke the news Bayer: Bayer's hemophilia A gene therapy DTX201 The phase 1/2 clinical trial of (AAVhu37 FVIII) also achieved positive results.
The preliminary data published on SAH 2020 showed that DTX201 can safely and effectively increase the level of coagulation factor VIII (FVIII), and prevent or reduce the level of coagulation factor VIII (FVIII) in clinical trials.
The bleeding status of the first two patients with severe hemophilia A. Bayer has also established a joint venture called Casebia Therapeutics with the Swiss company CRISPR Therapeutics to try new treatments for hemophilia based on CRISPR gene editing technology.
Recommended reading: ASH2019: Bayer's AAV Gene Therapy has positive results in Phase 1/2 clinical trials in hemophilia ARegeneron reveals that Regeneron: Regeneron announces that it will deliver a prepayment of US$70 million to Intellia Therapeutics Funds and 30 million US dollars of equity investment, totaling 100 million US dollars, to expand the cooperation between the two parties since 2016.
The main focus this time is to develop potential CRISPR/Cas9 editing gene therapy products for the treatment of hemophilia A and hemophilia B.
In preclinical research, the company demonstrated the first CRISPR/Cas9-mediated targeted transgene insertion in the liver of non-human primates, which can produce normal or higher levels of circulating human factor IX.
Factor IX is a missing or defective coagulation protein in patients with hemophilia B.
These results indicate that the transgene insertion can provide the functional factor 9 gene, which encodes an important protein.
▲ Picture source: Intellia official website.
At present, gene therapy has achieved relatively positive results in blood diseases, and gene therapy products have been successfully approved for marketing.
In 2022, both Pfizer/Sangamo and Roche/Spark will turn their attention to gene therapy.
In this regard, the development prospects are also worth looking forward to.
And at the upcoming 2021 BPIT Biopharmaceutical Innovation Technology Conference in Shanghai in April, Dong Biao, Chairman and General Manager of Zhishan Weixin, will give a speech on the process development strategy of rAAV gene medicine for hemophilia A.
Let us Focus on hot topics in the field of biomedicine together, and focus on new trends and developments in the industry together.
The Biopharmaceutical Innovation Technology Conference (BPIT) will kick off in Shanghai from April 9th to 10th, 2021.
The four major conference venues are exciting (click to view the focus topics). Participants who successfully registered for the Shanghai 2021 BPIT conference will receive a limited edition of "CAR-T Cell Therapy Industry Research Report" 2021.
V3.
1 produced by Xingyao Research Institute under Medical Media.
