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Global Blood Therapeutics

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GBT Announces Plans to Seek Expanded Labeling for Oxbryta® (voxelotor) to Treat Children Ages 4 to 11 Years with Sickle Cell Disease

Lead Product(s): Voxelotor

Therapeutic Area: Genetic Disease Product Name: Oxbryta

Highest Development Status: Approved Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 08, 2020

Details:

GBT intends to submit a New Drug Application for Oxbryta, which will include a new age-appropriate formulation, for the treatment of SCD in children ages 4 to 11 years. The NDA will include clinical data from the ongoing Phase 2a HOPE-KIDS 1 study.

Aptevo Therapeutics

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Medexus Provides an Update on the IXINITY® Acquisition and its Overall Business in the COVID-19 Environment

Lead Product(s): Recombinant coagulation factor IX

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Approved Product Type: Large molecule

Partner/Sponsor/Collaborator: MEDEXUS INC

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Acquisition May 22, 2020

Details:

The revenues from IXINITY® for March 2020 are in line with the Company’s expectations, including that the majority of the quarterly revenue from IXINITY® would be recognized in the final month of Q4 2020.

Vertex Pharmaceuticals

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Vertex Announces Innovative Reimbursement Agreement in Switzerland for ORKAMBI® and SYMDEKO® for Eligible Cystic Fibrosis Patients

Lead Product(s): Lumacaftor,Ivacaftor

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Approved Product Type: Small molecule

Partner/Sponsor/Collaborator: FSIO

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Agreement April 21, 2020

Details:

Agreement enables the possibility of rapid patient access to future triple combination regimen (elexacaftor/tezacaftor/ivacaftor and ivacaftor) once approved in Switzerland .

Emmaus Life Sciences

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Emmaus Life Sciences Announces SFDA (Saudi Food & Drug Authority) Accepts Endari® Priority Review Request

Lead Product(s): Glutamine

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Approved Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 16, 2020

Details:

Acceptance by Saudi Food & Drug Authority will expedite the review process for Emmaus Life Sciences's lead candidate Endari® for treatment of sickle cell disease.

Aptevo Therapeutics

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Medexus expands US product portfolio by acquiring hematology asset IXINITY

Lead Product(s): Recombinant coagulation factor IX

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Approved Product Type: Large molecule

Partner/Sponsor/Collaborator: MEDEXUS INC

Deal Size: $30.0 million Upfront Cash: $30.0 million

Deal Type: Acquisition February 28, 2020

Details:

Acquisition strengthens Medexus’ specialty product portfolio and represents a strategic fit with other products in the Medexus business development pipeline.

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Companies

Global Blood Therapeutics

GBT Announces Plans to Seek Expanded Labeling for Oxbryta® (voxelotor) to Treat Children Ages 4 to 11 Years with Sickle Cell Disease

Aptevo Therapeutics

Medexus Provides an Update on the IXINITY® Acquisition and its Overall Business in the COVID-19 Environment

Vertex Pharmaceuticals

Vertex Announces Innovative Reimbursement Agreement in Switzerland for ORKAMBI® and SYMDEKO® for Eligible Cystic Fibrosis Patients

Emmaus Life Sciences

Emmaus Life Sciences Announces SFDA (Saudi Food & Drug Authority) Accepts Endari® Priority Review Request

Aptevo Therapeutics

Medexus expands US product portfolio by acquiring hematology asset IXINITY