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Orphazyme to prepare for filing of arimoclomol in US for Niemann-Pick disease Type C (NPC)

Posted: 22.07.19

Company announcement Orphazyme A/S
No. 18/2019 Ole Maaløes Vej 3

DK-2200 Copenhagen N
Company Registration No. 32266355

Orphazyme to prepare for filing of arimoclomol in US for Niemann-Pick disease Type C (NPC)

• US Food and Drug Administration (FDA) agrees to Orphazyme’s plan
to file New Drug Application (NDA) in US
• On track to file NDA and Marketing Authorisation Application (MAA) in NPC
in both US and EU in H1 2020

Copenhagen, July 21, 2019 – Orphazyme A/S (ticker: ORPHA.CO), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announces that it has had a positive meeting with the FDA and remains on track to submit an NDA for arimoclomol for NPC in H1 2020.

Anders Hinsby, Chief Executive Officer of Orphazyme, said: “We are very pleased with the collaborative interactions we have had with the FDA who provided us with thorough guidance on data presentation in the NDA for arimoclomol in the US for NPC. The valuable advice we have received from health authorities in both the US and Europe increases our optimism that we may soon be able to provide an important treatment option for NPC, a severely debilitating and fatal disease that predominantly affects children.” Following the encouraging regulatory agency feedback, Orphazyme plans to introduce an Early Access Program for NPC in the fall of 2019, to further accelerate access to treatment with arimoclomol for people living with NPC.

As previously disclosed, Orphazyme also intends to file an MAA in Europe in H1 2020.

For additional 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 focused on bringing novel treatments to patients living with life-threatening or
debilitating rare diseases. Our research focuses on developing therapies for diseases caused by misfolding of proteins and lysosomal
dysfunction. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease
Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. The Denmark-based company is listed
on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit

About arimoclomol
Arimoclomol is an investigational drug candidate that amplifies the production of heat-shock proteins (HSPs). HSPs can rescue
defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally,
crosses the blood brain barrier, and has been studied in seven phase 1 and three phase 2 trials. Arimoclomol is in clinical
development for NPC, Gaucher disease, sIBM, and ALS.

About NPC
Niemann-Pick disease Type C (NPC) is a genetic, progressively debilitating, and often fatal neurovisceral disease. It belongs to a
family know as lysosomal storage diseases and is caused by mutations leading to defective NPC protein. As a consequence, lipids
that are normally cleared by the lysosome build-up in tissues and organs, including the brain, and drive the disease pathology. The
estimated prevalence of NPC in the USA and Europe combined is 1,000-2,000. There are no approved treatments for NPC in the USA
and only one approved product in Europe. Arimoclomol has been granted Orphan Drug Designation (EU and USA), Rare Pediatric
Disease Designation (USA), and Fast Track designation (USA) for the treatment of NPC.

Forward-looking statement
This press release may contain certain forward-looking statements. Although the Company believes its expectations are based on
reasonable assumptions, all statements other than statements of historical fact included in this press release about future events
are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without
limitation, any statements preceded by, followed by or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,”
“anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of
similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the
Company’s control that could cause the Company’s actual results, performance or achievements to be materially different from the
expected results, performance or achievements expressed or implied by such forward-looking statements. Except as required by
law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual
results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available
in the future.