All Data
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): Arimoclomol,Miglustat
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 February 04, 2021
Details:
Orphazyme plans to present a series of data on its investigational drug arimoclomol during the 17th Annual WORLDSymposium Scientific Meeting, to be held virtually on February 8-12, 2021.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): Arimoclomol,Miglustat
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 22, 2021
Details:
The long-term data demonstrate a continued positive impact on disease progression over two years. Furthermore, separate data from a post-hoc genetic sub-group analysis of patients provides more evidence of the efficacy of arimoclomol.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 December 27, 2020
Details:
U.S. Food and Drug Administration (FDA) has extended the review period of the New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC) by a standard extension period of three months.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 October 23, 2020
Details:
There are currently no approved products for NPC in the US and arimoclomol has the potential to make a significant difference to patients with this devastating disease.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 July 20, 2020
Details:
Orphazyme has completed the rolling submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational therapy arimoclomol for the treatment of Niemann-Pick disease Type C (NPC).
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 June 01, 2020
Details:
Orphazyme has initiated the submission of its New Drug Application (NDA) for a rolling review by the US Food and Drug Administration (FDA) for arimoclomol, based on the promising results reported from clinical trials. Trial conducted in collaboration with CytRx Corporation.
Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE
Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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.