BioMarin's hemophilia AAV gene therapy application is accepted by EMA

Recent popular reports from Yimaike ★Free access to the poster of "Modern Biotechnology Chromatography Filler and Fiber Selection Guide" ★Not only the new crown, mRNA will enter the sea of ​​stars for disease treatmentNew Observation of Yimai Click on the picture and register now July 17, 2021 /Healthcare News eMedClub PR News/--BioMarin Pharmaceutical Inc.
(NASDAQ: BMRN) announced yesterday that the European Medicines Agency (EMA) has accepted the company’s research gene therapy valoctocogene roxaparvovec (hereinafter referred to as valrox) for marketing authorization ( MAA), for adults with severe hemophilia A patients
.

With today’s acceptance, MAA will initiate a review and is expected to issue CHMP opinions in the first half of 2022
.

The submitted materials include the safety and efficacy data of 134 subjects who have been followed up for 1 year or more in the valrox phase 3 clinical study GENEr8-1; and the ongoing phase 1/2 dose escalation study of valrox, 6e13 vg/kg The 4-year follow-up data of the dose group and the 3-year follow-up data of the 4e13 vg/kg group
.

Hemophilia A is a bleeding disorder caused by the lack of coagulation factor VIII (FVIII).
The current treatment method mainly replenishes the coagulation factor through multiple blood transfusions per week.
Long-term blood transfusion brings many side effects and a great economic burden to patients
.

Valrox is a gene therapy that uses AAV5 viral vectors to deliver transgenes expressing FVIII
.

Its advantage is that patients may only need to receive one treatment, and liver cells can continue to express FVIII, eliminating the need for long-term prophylactic infusion of coagulation factors
.

Hank Fuchs, BioMarin Global R&D President, said: "We look forward to cooperating with EMA and believe that the submitted data can resolve previous requests made during the MAA review
.

This pivotal study proves the superiority of valrox compared to standard treatments for hemophilia
.

The large-scale data we have obtained today are attributed to the support of decades of scientific and clinical research in the field of gene therapy
.

We will continue to contribute to scientific knowledge, and will be held next week at the International Society for Thrombosis and Hemostasis (ISTH) in 2021.
) The latest results
of
our Phase 1/2 and Phase 3 studies were shared at the conference .
We believe that this gene therapy may meet the unmet medical needs of the rare disease community
.

▲ Dr.
Hank Fuchs, President of BioMarin Global R&D (Source: official website) ) After the brutal blow, a turning point occurred this year.
In August 2020, the FDA rejected the marketing application of BioMarin's hemophilia A gene therapy valrox, because of the average annualized bleeding rate in the Phase 1/2 study data published by BioMarin (ABR) fluctuates, and the level of factor VIII in patients continues to decline.
The FDA believes that more complete follow-up data is needed to prove the durability of this therapy
.

Recommended reading: Blockbuster! FDA rejected hemophilia A What's wrong with gene therapyYimai Meng broke the news ➤BioMarin, which was frustrated in its listing in the United States, became more cautious.
On November 4, 2020, it voluntarily filed with EMA to withdraw the listing application of valrox because BioMarin has determined that it cannot be within the specified time.
Provide the required long-term stability data to resolve the main objections of the Advanced Therapy Committee (CAT) related to the clinical research results in the current application process
.

Fortunately, the EMA requires a different follow-up time range for clinical data, and the EMA requires nearly 1 The relevant data of the patients in the phase III clinical trial of 2 years to prove the long-term stability, instead of the 2 years required by the FDA
.

➤ In January of this year, BioMarin announced the positive top-line results achieved in the ongoing global phase 3 clinical study GENEr8-1 of valrox
.

The GENEr8-1 study is a clinical trial for severe hemophilia A.
A total of 134 participants received a 6e13vg/kg valrox injection and completed a one-year or longer follow-up
.

The enrolled subjects are adult patients with severe hemophilia A, and the coagulation factor VIII activity is less than or equal to 1 IU/dL
.

One year later, the average blood coagulation factor VIII expression of all subjects was 42.
9 IU/dL, which supports that valrox has significant clinical benefits in eliminating bleeding events and reducing exogenous FVIII infusion
.

Recommended reading: heavy data! BioMarin announces the one-year follow-up data of the Phase 3 gene therapy trial for hemophilia AYi Mai Meng broke the news ➤ Valrox has received multiple qualifications this year, including the Regenerative Medicine Advanced Therapy Designation (RMAT) awarded by the FDA in March, and Application for accelerated evaluation approved by EMA in May
.

Previously, valrox has been granted Breakthrough Drug Designation (BTD) and Regenerative Medicine Advanced Therapy Designation (RMAT) by the US FDA; it has received the European Union’s EMA Priority Drug Designation (PRIME); and the FDA and EMA have granted Orphan Drug Designation (ODD)
.

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 ➤ Based on these positive effects, BioMarin regrouped and resubmitted the marketing authorization of valrox to EMA on June 25 Application (MAA)
.

Recommended reading: Regroup! Hemophilia A gene therapy reapplied to the EMA based on the one-year follow-up data.
Yimai Meng broke the news.
Now, valrox's MAA has been accepted by the European Union, waiting for the final opinion of the CHMP in 2022
.

In the United States, BioMarin plans to submit two-year follow-up data to support the benefit/risk assessment of valrox in accordance with the previous requirements of the FDA; and aims to resubmit the biologics license application (BLA) in the second quarter of 2022, if the research results are good , FDA will conduct a six-month review
.

Reference materials: 1.
https:// -301334635.
htmlYiMike has always been committed to original news reports such as cutting-edge technology, industry trends, and industry insights in bio-innovative drugs.
The high-end matrix users of all media have reached 160,000+, of which industrial users account for more than 50%, and scientific research and clinical users are about 30%, and more than 5% of investment institutions users
.

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
.