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Lead Product(s): Givosiran
Therapeutic Area: Genetic Disease Product Name: ALN-AT3SC
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 05, 2020
Details:
The WFH, EHC and NHF have learned of and subsequently confirmed a decision by Sanofi Genzyme to initiate a voluntary sponsor-led global dosing hold on its full clinical development program for fitusiran due to the identification of new adverse events.
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Lead Product(s): Avalglucosidase alfa
Therapeutic Area: Genetic Disease Product Name: GZ402666
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 October 02, 2020
Details:
The MAA is based on positive data from two trials: Phase 3, pivotal COMET trial, which evaluated the safety and efficacy of avalglucosidase alfa compared to alglucosidase alfa (standard of care) in patients with late-onset Pompe disease, and Phase 2 mini-COMET trial.
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Lead Product(s): Avalglucosidase alfa
Therapeutic Area: Genetic Disease Product Name: GZ402666
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 October 02, 2020
Details:
The MAA is based on positive data from two trials Phase 3 pivotal COMET trial, which evaluated the safety and efficacy of avalglucosidase alfa compared to alglucosidase alfa in patients with late-onset Pompe disease and Phase 2 mini-COMET trial.
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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: Swedish Orphan Biovitrum AB
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 10, 2020
Details:
BIVV001 is the first investigational factor VIII therapy independent of von Willebrand Factor and has the potential to transform replacement therapy for people with hemophilia A.
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Lead Product(s): rFVIIIFc-VWF-XTEN,Recombinant Antihemophilic Factor
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 June 29, 2020
Details:
Presentations include findings on hemophilia and acquired thrombotic thrombocytopenic purpura portfolio therapies and pipeline candidates.
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Lead Product(s): Givosiran
Therapeutic Area: Genetic Disease Product Name: ALN-AT3SC
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 19, 2020
Details:
Long-term interim results from the Phase 2 open-label extension (OLE) study reinforce fitusiran’s potential to restore hemostatic balance and to lower annualized bleed rates (ABRs) over a period up to 57 months.
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Lead Product(s): Avalglucosidase alfa
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 June 16, 2020
Details:
Avalglucosidase alfa showed a 2.4-point improvement in percent-predicted forced vital capacity, an important measure of respiratory function in Pompe disease, compared to alglucosidase alfa (standard of care).
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Lead Product(s): Avalglucosidase alfa
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 June 08, 2020
Details:
Sanofi will host a virtual scientific session to present data from the Phase 3 COMET trial of investigational enzyme replacement therapy (ERT) avalglucosidase alfa in patients with late-onset Pompe disease (LOPD).
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Lead Product(s): Avalglucosidase alfa
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 February 10, 2020
Details:
In patients with late-onset Pompe disease (LOPD), 5.5 years of safety and exploratory efficacy results showed avalglucosidase alfa was well-tolerated and stabilized pulmonary and motor function.
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Lead Product(s): Odiparcil
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 October 19, 2020
Details:
The Fast Track designation for odiparcil in MPS VI follows the acceptance by the FDA in August 2020 of Inventiva’s IND application for odiparcil in the same indication and the publication at the end of 2019 of positive results from its iMProveS Phase IIa clinical trial.
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