Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development

Lead Product(s): Olipudase alfa

Therapeutic Area: Genetic Disease

Highest Development Status: Phase II/ Phase III Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 30, 2020

Details:

Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

Lead Product(s): Cannabidiol

Therapeutic Area: Genetic Disease

Highest Development Status: Phase II/ Phase III Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 13, 2020

Details:

The trial has enrolled more severely affected population than that enrolled in the previously completed Phase 2 FAB-C trial as measured by baseline behavioral symptoms.

Lead Product(s): Arimoclomol

Therapeutic Area: Genetic Disease

Highest Development Status: Phase II/ Phase III Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 03, 2020

Details:

Orphazyme expects to receive approval for NPC in H1 2021 in the US through the fast track approval process, with the aim to bring the product to patients shortly thereafter.