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Lead Product(s): Nusinersen
Therapeutic Area: Genetic Disease Product Name: Spinraza
Highest Development Status: Phase IV Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 08, 2021
Details:
The RESPOND study aims to evaluate if Spinraza can benefit SMA patients who have experienced a ‘suboptimal’ response to Zolgensma (onasemnogene abeparvovec). It will be conducted at around 20 sites globally, with researchers aiming to enrol up to 60 children with SMA.
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Lead Product(s): Recombinant coagulation factor IX
Therapeutic Area: Genetic Disease Product Name: Ixinity
Highest Development Status: Phase IV Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 16, 2020
Details:
Medexus reached the 50% enrollment target in its Phase 4 clinical trial investigating IXINITY® as a prophylactic treatment for pediatric patients under 12 years of age with hemophilia B.
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Lead Product(s): Nusinersen
Therapeutic Area: Genetic Disease Product Name: Spinraza
Highest Development Status: Phase IV Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 21, 2020
Details:
The RESPOND study will seek to understand if the proven efficacy of SPINRAZA and its continuous production of SMN protein may also benefit patients previously treated with gene therapy.
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Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor
Therapeutic Area: Genetic Disease Product Name: Trikafta
Highest Development Status: Phase IV Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 20, 2020
Details:
The results of this study demonstrate that the triple combination provides significant additional benefit compared to existing CFTR modulator therapy for F/G and F/RF patients and supports the benefit of the medicine for patients with at least one F508del mutation.
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Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor
Therapeutic Area: Genetic Disease Product Name: Kaftrio
Highest Development Status: Phase IV Product Type: Small molecule
Partner/Sponsor/Collaborator: Vertex Pharmaceuticals
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement June 30, 2020
Details:
As part of the agreement with NHS England, Vertex has committed to submit ORKAMBI®, SYMKEVI® and KAFTRIO® to the National Institute for Health and Care Excellence (NICE) within an agreed upon timetable, allowing for a period of real-world data collection on the medicines.
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