Drugs in Dev.
Genetic Disease
Phase III
Canada 
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Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
April 23, 2024
Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
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 : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
November 23, 2023
Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : XORLO (proprietary oxypurinol formulation) is in under development for the treatment of individuals with progressing autosomal dominant polycystic kidney disease (ADPKD).
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
August 29, 2023
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
FDA Confirms Eligibility of XORLO™ for Accelerated Approval
Details : XORLO (proprietary oxypurinol formulation) is in under development for the treatment of individuals with progressing autosomal dominant polycystic kidney disease (ADPKD).
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
May 04, 2023
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
XORTX Initiates Dosing of Human Subjects in the XRX-OXY-101 Clinical Trial
Details : Following administration of first dose of drug, blood sampling and bioanalytical evaluation will be conducted to characterize pharmacokinetics and bioavailability of XRx-008 novel proprietary formulations of oxypurinol.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
May 03, 2022
Lead Product(s) : Oxypurinol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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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

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Lead Product(s) : Human Immunoglobulin
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
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 : Protein
Upfront Cash : Inapplicable
February 09, 2021
Lead Product(s) : Human Immunoglobulin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
Details : Pyridoxal Phosphate is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Pyridoxamine 5-Prime-Phosphate Oxidase Deficiency.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
January 12, 2021
Lead Product(s) : Pyridoxal Phosphate
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
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
December 07, 2020
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
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
November 08, 2020
Lead Product(s) : Plasminogen
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
