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Details:
Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 29, 2023
Details:
Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 06, 2023
Details:
Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 09, 2023
Details:
RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 23, 2022
Details:
Roctavian (valoctocogene roxaparvovec) also received orphan drug designation from the EMA and FDA for severe hemophilia A. Phase 1/2 study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 09, 2022
Details:
The GENEr8-1 Phase 3 study for valoctocogene roxaparvovec demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate (ABR) and the mean annualized Factor VIII infusion rate.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 12, 2022
Details:
Overall, to date, a single 6e13 vg/kg dose of BMN 270 (valoctocogene roxaparvovec) has been well tolerated with no delayed-onset treatment related adverse events.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: BMN 270
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 29, 2022
Details:
Overall, single 6e13 vg/kg dose of Roctavian (valoctocogene roxaparvovec) has been well tolerated with no delayed-onset treatment related adverse events.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 15, 2022
Details:
European Commission has granted conditional MA to ROCTAVIAN (valoctocogene roxaparvovec) gene therapy for treatment of severe hemophilia A in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 24, 2022
Details:
BMN 270 (valoctocogene roxaparvovec) (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)-based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: BMN 270
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 11, 2022
Details:
Roctavian (valoctocogene roxaparvovec) for treatment of severe haemophilia A in adults who do not have factor VIII inhibitors (auto-antibodies produced by immune system which make factor VIII medicines less effective) and no antibodies to adeno-associated virus serotype 5.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 24, 2022
Details:
Presentation of data at WFH follows positive two-year results from the ongoing, global phase 3 GENEr8-1 study of BMN 270 (valoctocogene roxaparvovec) presented at a medical meeting earlier in the year, as well as publication of one-year results from the pivotal clinical trial.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: BMN 270
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 05, 2022
Details:
Original research article reports that single infusion of valoctocogene roxaparvovec participants experienced substantially reduced annualized bleeding rates, reduced factor VIII utilization, and increased factor VIII activity, than they did in year prior to study enrollment.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: BMN 270
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 17, 2022
Details:
Phase 3 GENEr8-1 study of BMN 270 (valoctocogene roxaparvovec), investigational gene therapy for the treatment of adults with severe hemophilia A demonstrated superiority to factor VIII prophylaxis and reduction in annualized bleeding rate by 85% from baseline.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: BMN 270
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 09, 2022
Details:
Five-year and four-year post-treatment follow-up of the 6e13 vg/kg and 4e13 vg/kg cohorts, respectively, shows a sustained treatment benefit of valoctocogene roxaparvovec. All participants in both cohorts remain off prophylactic Factor VIII treatment.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: BMN 270
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 21, 2021
Details:
The safety profile of valoctocogene roxaparvovec in the Phase 1/2 study remains consistent with previously reported data with no delayed-onset treatment related adverse events. All participants continue to remain off corticosteroids since the first year.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: BMN 270
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 19, 2021
Details:
Data from the GENEr8-1 phase 3 study with a mean follow-up of 71.6 weeks showed that in the pre-specified primary analysis for annualized bleeding rate a single dose of valoctocogene roxaparvovec significantly reduced annualized bleeding rate by 84%.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: BMN 270
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 10, 2021
Details:
FDA introduces new recommendation for 2-year annualized bleeding rate (ABR) as a primary endpoint for ongoing phase 3 study 270-301.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: BMN 270
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 19, 2020
Details:
BioMarin Pharmaceutical reports additional data from its previously reported four-year update of an open-label Phase 1/2 study of valoctocogene roxaparvovec. valoctocogene roxaparvovec resulted in 96% mean reduction in Factor VIII usage.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 17, 2020
Details:
The four-year update for the 6e13 vg/kg and three-year update for the 4e13 vg/kg cohorts demonstrated that all subjects in both cohorts remain off prophylactic Factor VIII treatment since receiving their single dose of valoctocogene roxaparvovec.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 31, 2020
Details:
The acceptance by the FDA marks the first marketing application accepted for a gene therapy product for any type of hemophilia in the United States.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 20, 2020
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