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Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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            Lead Product(s): Risdiplam

            Therapeutic Area: Genetic Disease Product Name: Evrysdi

            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 February 26, 2021

            Details:

            Evrysdi is indicated for the treatment of 5q SMA in patients 2 months of age and older, with a clinical diagnosis of Type 1, Type 2 or Type 3 SMA or with one to four SMN2 copies.

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            Lead Product(s): Casimersen

            Therapeutic Area: Genetic Disease Product Name: AMONDYS 45

            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 February 25, 2021

            Details:

            The indication is based on a statistically significant increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45, which is reasonably likely to predict clinical benefit for those patients who are exon 45 amenable.

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            Lead Product(s): Berotralstat

            Therapeutic Area: Genetic Disease Product Name: Orladeyo

            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 February 25, 2021

            Details:

            The European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of ORLADEYO™ (berotralstat) for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older.

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            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 February 04, 2021

            Details:

            The EAP is designed to provide access to Oxbryta prior to approval for children ages 4 to 11 years with SCD in the U.S who have no alternative treatment options. GBT plans to submit an NDA for a pediatric formulation of Oxbryta for SCD in children aged 4 to 11 by mid-2021.

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            Lead Product(s): Icatibant Acetate

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Sandoz B2B

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement January 28, 2021

            Details:

            Sandoz in-licensed Icatibant injection, a critical anti-inflammatory medicine used to treat acute attacks of hereditary angioedema, from Slayback Pharma. Deal allows Sandoz to further grow its injectable medicine portfolio and supply another important medicine to US patients.