[{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme reports positive arimoclomol data from open-label phase 2\/3 extension in Niemann-Pick disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Orphazyme"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Initiates Rolling Submission Of New Drug Application For Arimoclomol With FDA in Niemann-Pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular 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Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development in DENMARK
Decision to withdraw the European Marketing Authorisation Application for BRX-345 (arimoclomol) for the treatment of Niemann-Pick disease Type C (NPC) ahead of a final vote and opinion by the CHMP on the MAA scheduled for later this month.
Orphazyme has made progress towards resubmission of the NDA for BRX-345 (arimoclomol citrate), amplifies the production of heat shock proteins for the treatment of NPC to the FDA and plans to request a Type C Meeting in Q2 2022.
Arimoclomol, is an investigational drug candidate that amplifies the production of heat shock proteins (HSPs). Arimoclomol is administered orally, and has now been studied in Phase 1, Phase 2, and pivotal Phase 2/3 trials.
arimoclomol, a heat shock protein co-inducer that promotes nascent protein folding, in patients with rapidly progressive SOD1 amyotrophic lateral sclerosis (ALS) and Niemann-Pick disease type C.
NPC patients receiving routine clinical care was estimated to be a mean increase of 5.2 points after three years, based on a statistical model combining placebo data from the NPC-002 double-blind study and prospective data from the observational NPC-001 study.
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.
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.
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.
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.
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).
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