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★Half-year financing has reached hundreds of millions, and the oncolytic virus has entered the fast laneYimai New Observation Click on the picture, Sign up now July 21, 2021/eMedClub News/--Recently, BioMarin announced at the International Society of Thrombosis and Hemostasis (ISTH) 2021 virtual conference on hemophilia gene therapy favaloctocogene roxaparvovec (hereinafter referred to as: valrox) The latest results in the phase 3 clinical study GENEr8-1 prove that valrox is superior to the baseline of factor VIII preventive therapy in terms of key clinical efficacy endpoints
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It is worth noting that the European Medicines Agency has accepted the marketing authorization application for the company's investigational gene therapy valrox (MAA), for severe hemophilia A adult patients
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MAA will initiate a review, and CHMP opinions are expected to be released in the first half of 2022
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Recommended reading: BioMarin's application for the AAV gene therapy for hemophilia has been accepted by the EMA.
Yimai Meng revealed that the new data provided on ISTH includes the annualized bleeding rate (ABR) of all study participants and the average annualized factor VIII usage rate.
More detailed information, expressed in international units/kg/year (IU/kg/year) of coagulation factor VIII
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For more details, scan the QR code for a complete reading of the listing application.
The FDA requires more complete follow-up data.
In August 2020, the FDA rejected BioMarin’s application for the marketing of hemophilia A gene therapy because of the announcement by BioMarin.
In the Phase 1/2 study data, the average annual bleeding rate (ABR) fluctuates, and the patient's factor VIII level continues to decline.
The FDA believes that this requires more complete follow-up data
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▲Three-year ABR of two cohorts of patients in the Phase 1/2 study (picture source: BioMarin official website) FDA hopes that BioMarin can complete the Phase 3 study of valrox and read the complete two-year follow-up data
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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
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The FDA requires long-term valid data, which has slowed the pace of BioMarin’s listing in the United States, but it is still actively seeking breakthroughs: On March 8 this year, the FDA granted valrox Regenerative Medicine Advanced Therapy Designation (RMAT), which was obtained by BioMarin in 2017 The addition of the name of breakthrough therapy aims to accelerate the development of new regenerative therapies
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Cell therapy that has been certified by RMAT also has the benefits of fast-track qualification and breakthrough therapy certification
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These advantages include the FDA’s early guidance for manufacturing and clinical development, such as identifying intermediate endpoints that support accelerated approval
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Recommended reading: The gene therapy for hemophilia A, which was rejected by the FDA, is now designated as an advanced therapy for regenerative medicine.
Yimai Meng broke the news that it withdrew its European listing application and listed in the United States.
BioMarin became more cautious, November 4, 2020 Voluntarily withdrew Roctavian's listing application to EMA because BioMarin has determined that it cannot provide the required long-term stability data within the specified time to resolve the main objections of the Advanced Therapy Committee (CAT) related to clinical research results in the current application process
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Fortunately, the time range of clinical data required by EMA is different.
EMA requires relevant data from patients in phase III clinical trials of the past 1 year to prove long-term stability, instead of the 2 years required by the FDA
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In January 2021, BioMarin announced the positive top-line results obtained in the ongoing global Phase 3 clinical study GENEr8-1 based on clinical trial data
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The GENEr8-1 study is a clinical trial for adults with severe hemophilia A.
A total of 134 participants received valrox injections of 6e13vg/kg and completed a one-year or longer follow-up to evaluate single-dose treatment adult efficacy and safety of severe hemophilia a patients
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 Recommended reading: heavy data! BioMarin announced the one-year follow-up data of the Phase 3 trial of gene therapy for hemophilia A.
Yimai Meng broke the news that the sustained efficacy and good safety demonstrated by GENEr8-1 have received positive feedback from EMA.
BioMarin is also based on this The trial was submitted to MAA
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In the United States, BioMarin plans to submit two-year follow-up safety and efficacy data for all patients in the GENEr8-1 study in response to the FDA's requirements for these data and support the FDA's assessment of the benefits and risks of valrox
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BioMarin's goal is to submit a Biological Products Licensing Application (BLA) to the US FDA in the second quarter of 2022, after which the FDA will conduct a six-month priority review process
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Reference materials: 1.
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