November 29, 2021

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AAV gene therapy for hemophilia may be submit to EMA by BioMarin in June

AAV gene therapy for hemophilia may be submit to EMA by BioMarin in June

AAV gene therapy for hemophilia reapplies for listing! BioMarin intends to submit a listing application to EMA in June

AAV gene therapy for hemophilia may be submit to EMA by BioMarin in June.  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 data from previous studies from the GENEr8-1 Phase 3 clinical trial (BMN 270-301) to evaluate the effectiveness of the drug.

At present, the two departments of EMA, namely the Committee on Medicines for Human Use (CHMP) and the Committee on Advanced Therapies (CAT), believe that the drug is of great significance to public health, especially in the area of ‚Äč‚Äčinnovative drug research. 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 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 and affirmed its therapeutic innovation potential and unmet medical needs.”

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.

The 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 patients may only After receiving one treatment, the liver cells can continue to express FVIII, which eliminates the need for long-term prophylactic injections of coagulation factors.

AAV gene therapy for hemophilia may be submit to EMA by BioMarin in June

BioMarin route

BioMarin showed in Roctavian’s first GENEr8-1 trial and the 3-year follow-up data of phase I/II clinical trial (NCT02576795) that Roctavian’s effect of maintaining FVIII production levels to control bleeding continued to decline, which may also be required by EMA and FDA One of the reasons for obtaining 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.

Currently, BioMarin has recruited up to 20 adults with severe hemophilia A to participate in the Phase 3b GENEr8-13 trial (NCT04323098), which is a single-arm, open-label study for the evaluation of BMN 270 ( Adeno-associated virus vector-mediated human factor VIII gene transfer and preventive glucocorticoid therapy), which may help prevent immune responses against 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.

(source:internet, reference only)


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