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Orphazyme A / S
Business Announcement
N ° 18/2019
Company number 32266355
- United States Food and Drug Administration (FDA) approves Orphazyme plan
to file a New Drug Application (NDA) in the United States - Filing a NDA and a Marketing Authorization Application (MAA) in NPCs
in the United States and in the EU in the first half of 2020
Copenhagen, 21 July 2019 – Orphazyme A / S (Symbol: ORPHA.CO), a biopharmaceutical company dedicated to the development of treatments for patients with rare diseases, today announced that it has had a positive meeting with the FDA and remains on track to submit an NDA for arimoclomol NPC in the first half of 2020.
Anders Hinsby, CEO of Orphazyme, said:We are very pleased with the collaborative interactions we have had with the FDA, which has provided us with detailed instructions on the submission of data in the NDA for arimoclomol in the US for NPC. The valuable advice we have received from US and European health authorities reinforces our optimism that we will soon be able to provide an important treatment option for NPC, an extremely debilitating and deadly disease that primarily affects children."
Following the encouraging feedback from regulators, Orphazyme plans to introduce an early access program for NPCs in the fall of 2019, to further accelerate access to treatment through arimoclomol for people living with this disease.
As previously stated, Orphazyme also intends to file a MAA in Europe in the first half of 2020.
For more information, please contact
Orphazyme A / S
Anders Hinsby, CEO +45 31 44 31 35
Anders Vadsholt, CFO +45 28 98 90 55
About Orphazyme A / S
Orphazyme is a biopharmaceutical company specializing in the development of new treatments for patients with life-threatening or debilitating rare diseases. Our research focuses on the development of therapies for diseases caused by protein misfolding and lysosomal dysfunction. Arimoclomol, the company's lead candidate, is in clinical development for four orphan diseases: Niemann-Pick type C disease, Gaucher disease, sporadic inclusion body myositis, and amyotrophic lateral sclerosis. The company based in Denmark is listed on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit www.orphazyme.com.
About arimoclomol
Arimoclomol is an experimental drug candidate that boosts the production of heat shock proteins (HSP). HSPs can recover defective misfolded proteins, eliminate protein aggregates, and improve lysosome function. Arimoclomol is administered orally, crosses the blood-brain barrier and has been the subject of seven phase 1 and 3 phase 2 studies. Arimoclomol is under clinical development for the treatment of NPC, Gaucher disease, IBS and ALS.
About NPCs
Niemann-Pick type C disease (NPC) is a genetic neurovisceral disease that is progressively debilitating and often fatal. It belongs to a family known as lysosomal storage diseases and is caused by mutations leading to a defective NPC protein. As a result, lipids that are normally eliminated by the accumulation of lysosomes in tissues and organs, including the brain, promote pathology. The estimated prevalence of NPCs in the United States and Europe combined is between 1,000 and 2,000. There is no approved treatment for NPC in the United States and only one product approved in Europe. Arimoclomol has been granted orphan drug designation (EU and US), Pediatric Disease designation (US) and Fast Track designation (US) for CPN treatment.
Forward-looking statement
This press release may contain certain forward-looking statements. Although the Company believes that its expectations are based on reasonable badumptions, all statements other than statements of historical fact included in this news release regarding future events are subject to (i) changes without notice and (ii) factors beyond the control of society. Such statements may include, without limitation, all statements preceded, followed by or including words such as "target", "believe", "expect", "aim", "intend to "," Power "," forecast "," estimate "," plan "," project "," will "," may "," likely "," should "," would "," could ", and other words and terms of similar meaning or the negative of these. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company's control that could cause the Company's results, performance or achievements to differ materially from the results, performance or achievements expected, expressed or implied in the future. these statements. Unless required by law, the Company badumes no obligation to publicly update these forward-looking statements, or to update the reasons why actual results could differ materially from those anticipated in forward-looking statements, even if if new information becomes available in the future.
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18-2019 Orphazyme prepares for arimoclomol deposit in the United States for NPC
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