-
Categories
-
Pharmaceutical Intermediates
-
Active Pharmaceutical Ingredients
-
Food Additives
- Industrial Coatings
- Agrochemicals
- Dyes and Pigments
- Surfactant
- Flavors and Fragrances
- Chemical Reagents
- Catalyst and Auxiliary
- Natural Products
- Inorganic Chemistry
-
Organic Chemistry
-
Biochemical Engineering
- Analytical Chemistry
-
Cosmetic Ingredient
- Water Treatment Chemical
-
Pharmaceutical Intermediates
Promotion
ECHEMI Mall
Wholesale
Weekly Price
Exhibition
News
-
Trade Service
Recent popular reports from Yimaike★How to gain a place in the fierce competition? Focus on the protein expression market and master the secrets of biopharmaceuticals ★ CStone Pharmaceuticals' Tumor Precision Therapy Forum and Taijihua® China Launch Conference will be held in Shanghai to enter the era of precision targeted therapy for gastrointestinal stromal tumors.
Click the picture and register now May 28, 2021 /Healthcare News eMedClub News/--Recently, BioMarin plans to resubmit Roctavian's listing application in June.
The European Medicines Agency (EMA) will accelerate the evaluation of the second application of BioMarin pharmaceutical company Roctavian, and it is expected to obtain CHMP opinion in the first half of 2022.
BioMarin is a global biotechnology company focused on developing innovative therapies for the treatment of serious and life-threatening rare and ultra-rare genetic diseases.
Last year, BioMarin's Roctavian listing application was rejected by the European EMA and the US FDA.
The regulatory agency requires BioMarin to provide previous research data from the GENEr8-1 Phase 3 clinical trial (BMN 270-301) to evaluate the effectiveness of the drug.
Recommended reading: Heavy! The FDA rejected the gene therapy for hemophilia A, what went wrongYimai Meng broke the news.
At present, the two departments of EMA, namely the Committee for Human Medicines (CHMP) and the Committee for Advanced Therapies (CAT), believe that the drug is good for the public.
Health is of great significance, especially in the research of innovative drugs.
Not only that, this drug has been designated as an orphan drug and a priority drug in Europe; it has been designated as an orphan drug, breakthrough therapy and advanced regenerative medicine in the United States for the treatment of severe hemophilia A, so its development will be accelerated And the review speed, the regulatory agency’s regulatory review of BioMarin has been shortened from the standard 210 days to 150 days.
Dr.
Hank Fuchs, President of BioMarin’s Global Research and Development, said: “BioMarin is pleased that EMA has provided an accelerated assessment for Roctavian’s review, affirming its potential for therapeutic innovation and unmet medical needs.
” Roctavian for hemophilia A is currently working on hemophilia There are mainly two types of diseases, A and B.
People with hemophilia A lack blood clotting factor VIII or have a lower level of VIII, which is the most common type of hemophilia.
This type of hemophilia is more common among boys, and the incidence is estimated to be 1 in 4000-5000 males born.
When blood vessels in the human body are injured, coagulation factors can help platelets stick together, block the cuts and breaks on the blood vessels, and play a role in hemostasis.
Many people with hemophilia experience spontaneous bleeding events, which can lead to progressive and debilitating tissue damage.
BioMarin is an alternative therapy designed to increase the lack of coagulation factor VIII (FVIII) in patients with hemophilia A, thereby reducing or eliminating the lack of VIII.
This alternative therapy can reduce spontaneous bleeding in patients.
This therapy can be administered through a single intravenous injection, modified harmless adeno-associated virus, and delivers a short but effective copy of F8 (this gene provides the gene for FVIII) to liver cells.
Its advantage is that the patient may only Only one treatment is needed, and liver cells can continue to express FVIII, which eliminates the need for long-term prophylactic injections of coagulation factors.
▲ BioMarin pipeline (picture source: official website) BioMarin showed in Roctavian's first GENEr8-1 trial and 3-year follow-up data of phase I/II clinical trial (NCT02576795), Roctavian's effect of maintaining FVIII production levels to control bleeding continued to decline This may also be one of the reasons why EMA and FDA request more GENEr8-1 Phase 3 clinical trial data.
The Phase 3 clinical study of GENEr8-1 is a clinical trial for adults with severe hemophilia A, evaluating the effectiveness and safety of a single dose of valrox at a dose of 6e13 vg/kg in the treatment of adult patients with severe hemophilia A.
In the phase 3 study, 134 participants who received a single valrox (dose of 6e13 vg/kg) tolerated valrox well, and none of the participants had a need for FVIII inhibitors or thromboembolic events.
Recommended reading: heavy data! BioMarin announces one-year follow-up data for the Phase 3 trial of gene therapy for hemophilia AYi Mai Meng broke the news.
Currently, BioMarin has recruited up to 20 adults with severe hemophilia A to participate in the Phase 3b GENEr8-13 trial.
(NCT04323098), this experiment is a one-arm open-label study to study and evaluate BMN 270 (Adeno-associated virus vector-mediated human factor VIII gene transfer and preventive glucocorticoid therapy), which may help prevent Immune response of virus carriers.
The therapy is being evaluated in two other phase 1/2 clinical trials, including patients with pre-existing AAV5 antibodies (NCT03520712) and patients with pre-existing anti-FAV activity or previous inhibitors (neutralizing antibodies) (NCT04684940) for evaluation The safety and effectiveness of the drug for poisoning hemophilia A.
In addition, valrox has the longest data of all the gene therapies under study for hemophilia A.
This time, BioMarin will reapply for listing in June.
The results will be known in the first half of 2022.
Summary Gene therapy was first developed to treat hereditary genetic diseases.
After positive results have been achieved, gene therapy has developed faster and faster.
Most of the gene therapy products currently on the market are developed abroad, and the domestic market is still relatively in the early stages of development in this field.
In recent years, the field of gene therapy in my country is gradually emerging.
As a new medical field, it is profoundly changing the face of the pharmaceutical industry.
With the strong domestic policy support for the research and development of new drugs, a large number of outstanding local gene therapy companies and scientific research institutions have also emerged in the domestic gene therapy field.
Recommended Reading: List of Domestic Gene Therapy CompaniesHemophilia Inventory Year-end Resumption: Financing and Transactions in the Field of Domestic Gene Therapy in 2020Hemophilia's New Observation Reference Materials: 1.
https://hemophilianewstoday.
com/2021/05/26/ biomarin-resubmit-gene-therapy-roctavian-eu-approval-june-hemophilia-a-accelerated-review There are 160,000+ users, of which industrial users account for more than 50%, scientific research and clinical users are about 30%, and investment institutions account for more than 5%.
In order to promote interactive exchanges in industry segments, we have established a number of professional WeChat groups, welcome to scan the QR code to add groups.
Click the picture and register now May 28, 2021 /Healthcare News eMedClub News/--Recently, BioMarin plans to resubmit Roctavian's listing application in June.
The European Medicines Agency (EMA) will accelerate the evaluation of the second application of BioMarin pharmaceutical company Roctavian, and it is expected to obtain CHMP opinion in the first half of 2022.
BioMarin is a global biotechnology company focused on developing innovative therapies for the treatment of serious and life-threatening rare and ultra-rare genetic diseases.
Last year, BioMarin's Roctavian listing application was rejected by the European EMA and the US FDA.
The regulatory agency requires BioMarin to provide previous research data from the GENEr8-1 Phase 3 clinical trial (BMN 270-301) to evaluate the effectiveness of the drug.
Recommended reading: Heavy! The FDA rejected the gene therapy for hemophilia A, what went wrongYimai Meng broke the news.
At present, the two departments of EMA, namely the Committee for Human Medicines (CHMP) and the Committee for Advanced Therapies (CAT), believe that the drug is good for the public.
Health is of great significance, especially in the research of innovative drugs.
Not only that, this drug has been designated as an orphan drug and a priority drug in Europe; it has been designated as an orphan drug, breakthrough therapy and advanced regenerative medicine in the United States for the treatment of severe hemophilia A, so its development will be accelerated And the review speed, the regulatory agency’s regulatory review of BioMarin has been shortened from the standard 210 days to 150 days.
Dr.
Hank Fuchs, President of BioMarin’s Global Research and Development, said: “BioMarin is pleased that EMA has provided an accelerated assessment for Roctavian’s review, affirming its potential for therapeutic innovation and unmet medical needs.
” Roctavian for hemophilia A is currently working on hemophilia There are mainly two types of diseases, A and B.
People with hemophilia A lack blood clotting factor VIII or have a lower level of VIII, which is the most common type of hemophilia.
This type of hemophilia is more common among boys, and the incidence is estimated to be 1 in 4000-5000 males born.
When blood vessels in the human body are injured, coagulation factors can help platelets stick together, block the cuts and breaks on the blood vessels, and play a role in hemostasis.
Many people with hemophilia experience spontaneous bleeding events, which can lead to progressive and debilitating tissue damage.
BioMarin is an alternative therapy designed to increase the lack of coagulation factor VIII (FVIII) in patients with hemophilia A, thereby reducing or eliminating the lack of VIII.
This alternative therapy can reduce spontaneous bleeding in patients.
This therapy can be administered through a single intravenous injection, modified harmless adeno-associated virus, and delivers a short but effective copy of F8 (this gene provides the gene for FVIII) to liver cells.
Its advantage is that the patient may only Only one treatment is needed, and liver cells can continue to express FVIII, which eliminates the need for long-term prophylactic injections of coagulation factors.
▲ BioMarin pipeline (picture source: official website) BioMarin showed in Roctavian's first GENEr8-1 trial and 3-year follow-up data of phase I/II clinical trial (NCT02576795), Roctavian's effect of maintaining FVIII production levels to control bleeding continued to decline This may also be one of the reasons why EMA and FDA request more GENEr8-1 Phase 3 clinical trial data.
The Phase 3 clinical study of GENEr8-1 is a clinical trial for adults with severe hemophilia A, evaluating the effectiveness and safety of a single dose of valrox at a dose of 6e13 vg/kg in the treatment of adult patients with severe hemophilia A.
In the phase 3 study, 134 participants who received a single valrox (dose of 6e13 vg/kg) tolerated valrox well, and none of the participants had a need for FVIII inhibitors or thromboembolic events.
Recommended reading: heavy data! BioMarin announces one-year follow-up data for the Phase 3 trial of gene therapy for hemophilia AYi Mai Meng broke the news.
Currently, BioMarin has recruited up to 20 adults with severe hemophilia A to participate in the Phase 3b GENEr8-13 trial.
(NCT04323098), this experiment is a one-arm open-label study to study and evaluate BMN 270 (Adeno-associated virus vector-mediated human factor VIII gene transfer and preventive glucocorticoid therapy), which may help prevent Immune response of virus carriers.
The therapy is being evaluated in two other phase 1/2 clinical trials, including patients with pre-existing AAV5 antibodies (NCT03520712) and patients with pre-existing anti-FAV activity or previous inhibitors (neutralizing antibodies) (NCT04684940) for evaluation The safety and effectiveness of the drug for poisoning hemophilia A.
In addition, valrox has the longest data of all the gene therapies under study for hemophilia A.
This time, BioMarin will reapply for listing in June.
The results will be known in the first half of 2022.
Summary Gene therapy was first developed to treat hereditary genetic diseases.
After positive results have been achieved, gene therapy has developed faster and faster.
Most of the gene therapy products currently on the market are developed abroad, and the domestic market is still relatively in the early stages of development in this field.
In recent years, the field of gene therapy in my country is gradually emerging.
As a new medical field, it is profoundly changing the face of the pharmaceutical industry.
With the strong domestic policy support for the research and development of new drugs, a large number of outstanding local gene therapy companies and scientific research institutions have also emerged in the domestic gene therapy field.
Recommended Reading: List of Domestic Gene Therapy CompaniesHemophilia Inventory Year-end Resumption: Financing and Transactions in the Field of Domestic Gene Therapy in 2020Hemophilia's New Observation Reference Materials: 1.
https://hemophilianewstoday.
com/2021/05/26/ biomarin-resubmit-gene-therapy-roctavian-eu-approval-june-hemophilia-a-accelerated-review There are 160,000+ users, of which industrial users account for more than 50%, scientific research and clinical users are about 30%, and investment institutions account for more than 5%.
In order to promote interactive exchanges in industry segments, we have established a number of professional WeChat groups, welcome to scan the QR code to add groups.
