All Data
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Lead Product(s): Lanadelumab
Therapeutic Area: Genetic Disease Product Name: Takhzyro
Highest Development Status: Approved Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 13, 2020
Details:
Results were consistent with the safety and efficacy of TAKHZYRO in the pivotal trial. The HELP Study OLE was designed to evaluate the long-term safety (primary endpoint) and efficacy of TAKHZYRO for up to 2.5 years.
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Lead Product(s): Burosumab
Therapeutic Area: Genetic Disease Product Name: Crysvita
Highest Development Status: Approved Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 05, 2020
Details:
CRYSVITA, created by Kyowa Kirin, is a recombinant fully human monoclonal IgG1 antibody against the FGF23. More people in Europe are now eligible for treatment with CRYSVITA, the only therapy that targets the underlying pathophysiology of XLH.
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Lead Product(s): Porcine recombinant Factor VIII
Therapeutic Area: Genetic Disease Product Name: Obizur
Highest Development Status: Approved Product Type: Large molecule
Partner/Sponsor/Collaborator: HLS Therapeutics
Deal Size: $49.3 million Upfront Cash: $30.8 million
Deal Type: Acquisition September 30, 2020
Details:
HLS has acquired certain entities that hold the rights to a diversified portfolio of royalty interests on global sales of four different products, this includes Takeda Pharmaceutical's Obizur, a porcine recombinant Factor VIII for acquired hemophilia A.
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Lead Product(s): Burosumab
Therapeutic Area: Genetic Disease Product Name: Crysvita
Highest Development Status: Approved Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 24, 2020
Details:
The European Commission has already granted a conditional marketing authorisation for CRYSVITA for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons.
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Lead Product(s): Emicizumab
Therapeutic Area: Genetic Disease Product Name: Hemlibra
Highest Development Status: Approved Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 13, 2020
Details:
In the STASEY study, Hemlibra was effective with no new safety signals identified in adults and adolescents with hemophilia A with factor VIII inhibitors, which was consistent with the previously reported safety profile
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Lead Product(s): Lanadelumab
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Approved Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 06, 2020
Details:
Data from the Phase 3 HELP Study™ Open-label Extension suggest that TAKHZYRO® (lanadelumab) is well-tolerated and can prevent hereditary angioedema (HAE) attacks over an extended treatment period, with sustained and consistent reduction in monthly attack rates.
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Lead Product(s): Lanadelumab
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Approved Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 29, 2020
Details:
The pre-filled syringe presentation is designed to enhance the treatment administration experience for HAE patients receiving TAKHZYRO.