Drugs in Dev. Genetic Disease
Phase III
Sweden 
Lead Product(s) : Efanesoctocog Alfa
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : PSI CRO
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Efanesoctocog Alfa is a Protein drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Hemophilia A.
Product Name : Undisclosed
Product Type : Protein
Upfront Cash : Inapplicable
December 04, 2024
Lead Product(s) : Efanesoctocog Alfa
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : PSI CRO
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Efanesoctocog Alpha
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Sobi® Gains Positive CHMP Opinion for Efanesoctocog Alfa in Haemophilia A
Details : BIVV001 (efanesoctocog alfa), a once-weekly Factor VIII therapy, is being evaluated for bleed prevention and perioperative prophylaxis in hemophilia A.
Product Name : Undisclosed
Product Type : Protein
Upfront Cash : Inapplicable
April 26, 2024
Lead Product(s) : Efanesoctocog Alpha
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Efanesoctocog Alfa
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Syneos Health
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Efanesoctocog Alfa is a Protein drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Hemophilia A.
Product Name : Undisclosed
Product Type : Protein
Upfront Cash : Inapplicable
April 18, 2023
Lead Product(s) : Efanesoctocog Alfa
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Syneos Health
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Efanesoctocog Alpha
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophili...
Product Name : Undisclosed
Product Type : Protein
Upfront Cash : Inapplicable
January 26, 2023
Lead Product(s) : Efanesoctocog Alpha
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Efanesoctocog Alpha
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
FDA Grants Priority Review to Efanesoctocog Alfa for People With Hemophilia A
Details : Efanesoctocog alfa (BIVV001) delivers high sustained factor activity levels in the normal to nearnormal range for the majority of the week with once weekly prophylaxis dosing, providing higher protection for longer.
Product Name : Undisclosed
Product Type : Protein
Upfront Cash : Inapplicable
August 30, 2022
Lead Product(s) : Efanesoctocog Alpha
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Efanesoctocog Alpha
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : XTEND-Kids is an open-label, non-randomised interventional, single-arm study. Participants will receive a weekly prophylactic dose of efanesoctocog alfa for 52 weeks.
Product Name : Undisclosed
Product Type : Protein
Upfront Cash : Inapplicable
April 13, 2021
Lead Product(s) : Efanesoctocog Alpha
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Nitisinone
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Sobi's Orfadin® (nitisinone) Receives Positive Opinion from CHMP for Treatment of AKU
Details : European Medicines Agency (EMA) has adopted a positive opinion for Orfadin® (nitisinone) for the treatment of adult patients with alkaptonuria (AKU). The opinion is based on the scientific results of the DevelopAKUre clinical development programme.
Product Name : Orfadin
Product Type : Miscellaneous
Upfront Cash : Inapplicable
September 18, 2020
Lead Product(s) : Nitisinone
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Efanesoctocog Alpha
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Data for Doptelet® (avatrombopag) in treatment for thrombocytopenia within Chronic Liver Disease (CLD) and Chronic Immune Thrombocytopenia (ITP) will be presented.
Product Name : Undisclosed
Product Type : Protein
Upfront Cash : Inapplicable
July 08, 2020
Lead Product(s) : Efanesoctocog Alpha
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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