


Details:
The observational Phase 4 EMPOWER study, evaluating real-world HAE attack rates before and after treatment with TAKHZYRO in patients with HAE types I and II, is ongoing. Full results of the EMPOWER study are expected to be published in 2024.
Lead Product(s): Lanadelumab
Therapeutic Area: Genetic Disease Product Name: Takhzyro
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 February 28, 2022
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.
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:
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.
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:
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.
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 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.
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:
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.
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