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Lead Product(s): Nitisinone
Therapeutic Area: Genetic Disease Product Name: Orfadin
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 18, 2020
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.
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Lead Product(s): rFVIIIFc-VWF-XTEN
Therapeutic Area: Genetic Disease Product Name: BIVV001
Highest Development Status: Phase III Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 08, 2020
Details:
Data for Doptelet® (avatrombopag) in treatment for thrombocytopenia within Chronic Liver Disease (CLD) and Chronic Immune Thrombocytopenia (ITP) will be presented.
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Lead Product(s): Oxalobacter formigenes
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase III Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 06, 2020
Details:
Study OC5-DB-02 (ePHex) is a 12-month randomized, double-blind, placebo-controlled, global, multicenter Phase 3 study to evaluate the efficacy and safety of Oxabact OC5.
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