Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
BioMarin Announces Updated Strategy for ROCTAVIAN® to Focus on U.S., Germany and Italy
Details : Roctavian (valoctocogene roxaparvovec) is a gene therapy approved for severe hemophilia A, that uses an adeno-associated virus 5, codes for human Factor VIII.
Brand Name : Roctavian
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
August 05, 2024
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : Roctavian
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
June 29, 2023
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : Roctavian
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
March 06, 2023
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : Roctavian
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
January 09, 2023
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : Roctavian
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
November 23, 2022
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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 Fact...
Brand Name : Roctavian
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
November 09, 2022
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : Roctavian
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
October 12, 2022
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : BMN 270
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
September 29, 2022
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Details : Overall, single 6e13 vg/kg dose of Roctavian (valoctocogene roxaparvovec) has been well tolerated with no delayed-onset treatment related adverse events.
Brand Name : Roctavian
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
September 15, 2022
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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...
Brand Name : Roctavian
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
August 24, 2022
Lead Product(s) : Valoctocogene Roxaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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