Details:
Voydeya (danicopan) is an investigational oral medicine in development as an add-on to C5 inhibitor therapy eculizumab or ravulizumab for patients with paroxysmal nocturnal hemoglobinuria (PNH) who have residual haemolytic anaemia.
Lead Product(s): Danicopan,Ravulizumab
Therapeutic Area: Rare Diseases and Disorders Product Name: Voydeya
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, 2024
Details:
Through the acquisition, Kyowa expands its portfolio, enables the development of promising candidates with a clinically differentiated platform, and helps resource the ongoing and future launches of Libmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy.
Lead Product(s): Atidarsagene Autotemcel
Therapeutic Area: Rare Diseases and Disorders Product Name: Libmeldy
Highest Development Status: Approved Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Kyowa Kirin
Deal Size: $477.6 million Upfront Cash: $387.4 million
Deal Type: Acquisition January 24, 2024
Details:
Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. It is being recommended for the treatment of neuromyelitis optica spectrum disorder.
Lead Product(s): Ravulizumab
Therapeutic Area: Rare Diseases and Disorders Product Name: Ultomiris
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 September 06, 2023
Details:
Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. It is being recommended for the treatment of neuromyelitis optica spectrum disorder.
Lead Product(s): Ravulizumab
Therapeutic Area: Rare Diseases and Disorders Product Name: Ultomiris
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 26, 2023
Details:
Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. It is EU approved for the treatment of neuromyelitis optica spectrum disorder.
Lead Product(s): Ravulizumab
Therapeutic Area: Rare Diseases and Disorders Product Name: Ultomiris
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 10, 2023
Details:
Soliris (eculizumab) is a first-in-class C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system.
Lead Product(s): Eculizumab
Therapeutic Area: Rare Diseases and Disorders Product Name: Soliris
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 11, 2022
Details:
Positive high-level results from CHAMPION-NMOSD trial showed that Ultomiris (ravulizumab-cwvz) achieved a statistically significant and clinically meaningful reduction in risk of relapse in adults with anti-aquaporin-4 (AQP4) (Ab+) in NMOSD compared to external placebo arm.
Lead Product(s): Ravulizumab
Therapeutic Area: Rare Diseases and Disorders Product Name: Ultomiris
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 05, 2022
Details:
Nucala (mepolizumab) is the first-in-class monoclonal antibody targets IL-5 from binding to its receptor on the surface of eosinophils, reducing blood eosinophils and maintaining them within normal levels.
Lead Product(s): Mepolizumab
Therapeutic Area: Rare Diseases and Disorders Product Name: Nucala
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 17, 2021
Details:
Submissions based on positive data from pivotal studies in hypereosinophilic syndrome, chronic rhinosinusitis with nasal polyps and eosinophilic granulomatosis with polyangiitis.
Lead Product(s): Mepolizumab
Therapeutic Area: Rare Diseases and Disorders Product Name: Nucala
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 29, 2020