Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Medicure Receives US FDA Fast Track Designation for MC-1 for PNPO Deficiency
Details : MC-1 (pyridoxal phosphate) is a naturally occuring purinergic receptor antagonist, which is being evaluated for the treatment of Pyridox(am)Ine 5'-Phosphate Oxidase (PNPO) Deficiency.
Product Name : MC-1
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
April 23, 2024
Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : FDA provides complete approval to enroll patients in its pivotal clinical trial to evaluate "MC-1" (a naturally occurring metabolite of pyridoxine) for treatment of a rare pediatric disease called pyridox(am)ine 5′-phosphate oxidase deficiency.
Product Name : MC-1
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
November 23, 2023
Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Human Immunoglobulin
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Multicenter study of the efficacy, safety, tolerability, and pharmacokinetics of PlasmaCap IG in adults and children with primary immune deficiency disease met all primary and secondary endpoints.
Product Name : PlasmaCap IG
Product Type : Other Large Molecule
Upfront Cash : Inapplicable
September 02, 2021
Lead Product(s) : Human Immunoglobulin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Kedrion
Deal Size : $22.0 million
Deal Type : Divestment
Details : Kedrion will enter into an option agreement with Liminal BioSciences to acquire the remainder of the company’s plasma-derived business, as well as Plasminogen business operated through its subsidiaries including Prometic Biotherapeutics which holds the...
Product Name : Ryplazim
Product Type : Protein
Upfront Cash : Undisclosed
May 17, 2021
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Kedrion
Deal Size : $22.0 million
Deal Type : Divestment
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Liminal BioSciences Provides Update on Progress on BLA for Ryplazim®(plasminogen)
Details : The BLA resubmission for Ryplazim® is supported by data from a Phase 2/3 clinical study which evaluated 15 patients, both pediatric and adults, with congenital plasminogen deficiency over 48 weeks of therapy with Ryplazim®.
Product Name : Ryplazim
Product Type : Protein
Upfront Cash : Inapplicable
September 21, 2020
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Liminal BioSciences Announces FDA Extension of Review Period for Ryplazim®(Plasminogen) BLA
Details : The BLA resubmission for Ryplazim® was supported by data from a Phase 2/3 clinical trial, which evaluated 15 patients, both pediatric and adults, with C-PLGD over 48 weeks of therapy with Ryplazim®.
Product Name : Ryplazim
Product Type : Protein
Upfront Cash : Inapplicable
August 11, 2020
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : In a pivotal phase 2/3 clinical trial for the treatment of C-PLGD, all of the patients treated with Ryplazim® achieved at least the targeted increase from baseline in their individual trough plasminogen activity levels through 12 weeks of therapy.
Product Name : Ryplazim
Product Type : Protein
Upfront Cash : Inapplicable
August 09, 2020
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Two posters were presented that highlighted individual case histories of patients with C-PLGD treated with the investigational intravenous Ryplazim® (plasminogen) in compassionate use and expanded access cases.
Product Name : Ryplazim
Product Type : Protein
Upfront Cash : Inapplicable
July 12, 2020
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Two abstracts related to the Company’s clinical development activities of Ryplazim® for the treatment of Congenital Plasminogen Deficiency will be presented at the 62nd American Society of Hematology Annual Meeting and Exposition scheduled to take pla...
Product Name : Ryplazim
Product Type : Protein
Upfront Cash : Inapplicable
May 11, 2020
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable