X
[{"orgOrder":0,"company":"OxThera AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"OxThera Announces Completion of Recruitment in Phase 3 ePHex Study With Oxabact\u00ae in Patients With Primary Hyperoxaluria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Large molecule","productStatus":"Undisclosed","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"OxThera AB"},{"orgOrder":0,"company":"OxThera AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"OxThera reports Results from Phase 3 ePHex Study with Oxabact in Primary Hyperoxaluria Patients with Maintained Kidney Function","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"OxThera AB"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Data to be Presented at ISTH Virtual Congress Highlights Sobi's Commitment to Advancing Rare Haematology Treatments","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sobi's Orfadin\u00ae (nitisinone) Receives Positive Opinion from CHMP for Treatment of AKU","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"First Patient Dosed in Phase 3 XTEND-Kids Study with Efanesoctocog Alfa in Children with Haemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Priority Review to Efanesoctocog Alfa for People With Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NEJM Publishes Data Demonstrating Clinically Meaningful Prevention of Bleeds and Superior Bleed Protection for Efanesoctocog Alfa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Swedish Orphan Biovitrum AB"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
Filters
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 haemophilia A.
Lead Product(s):
Efanesoctocog Alfa
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
January 26, 2023
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.
Lead Product(s):
Efanesoctocog Alfa
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
August 30, 2022
Details:
Oxabact is a bi-modal enteric biotherapy containing a lyophilized formulation of Oxalobacter formigenes, a non-pathogenic, oxalate-degrading commensal bacterium used for treatment of Primary Hyperoxaluria.
Lead Product(s):
Oxalobacter formigenes
Therapeutic Area: Genetic Disease
Product Name: Oxabact
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
June 11, 2021
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.
Lead Product(s):
Efanesoctocog alfa
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
April 13, 2021
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.
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:
Data for Doptelet® (avatrombopag) in treatment for thrombocytopenia within Chronic Liver Disease (CLD) and Chronic Immune Thrombocytopenia (ITP) will be presented.
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:
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.
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