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Myalepta (metreleptin) is a USFDA approved leptin receptor activator, which is now approved by Health Canada for the treatment of congenital or acquired generalized lipodystrophy.
Lead Product(s): Metreleptin
Therapeutic Area: Genetic Disease Product Name: Myalepta
Highest Development Status: ApprovedProduct Type: Peptide
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 05, 2024
Details:
Filsuvez (birch triterpenes) is a topical gel which is approved for the treatment of wounds associated with dystrophic and junctional epidermolysis bullosa.
Lead Product(s): Birch Triterpenes
Therapeutic Area: Genetic Disease Product Name: Filsuvez
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 19, 2023
Details:
Elfabrio (pegunigalsidase alfa-iwxj) is a PEGylated enzyme replacement therapy (ERT) for the treatment of adult patients with Fabry disease. It is a recombinant human α–Galactosidase–A enzyme expressed in plant-cell culture that is designed to provide a long half-life.
Lead Product(s): Pegunigalsidase alfa-iwxj
Therapeutic Area: Genetic Disease Product Name: Elfabrio
Highest Development Status: ApprovedProduct Type: Large molecule
Recipient: Protalix BioTherapeutics
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 10, 2023
Details:
PRX-102 (pegunigalsidase alfa), EC authorized, is a novel PEGylated, covalently crosslinked form of α-galactosidase-A developed as enzyme replacement therapy for fabry disease, was designed to increase plasma half-life and reduce immunogenicity, thereby enhancing efficacy.
Lead Product(s): Pegunigalsidase alfa
Therapeutic Area: Genetic Disease Product Name: PRX-102
Highest Development Status: ApprovedProduct Type: Large molecule
Recipient: Protalix BioTherapeutics
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 05, 2023
Details:
Ferriprox™ (deferiprone) is a chelating agent with an affinity for ferric ion (iron III), being developed for the treatment of patients with transfusional iron overload due to thalassemia syndromes.
Lead Product(s): Deferiprone
Therapeutic Area: Genetic Disease Product Name: Ferriprox
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 31, 2023
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