Our Research

We are focused on understanding the cell-protective properties of heat shock proteins (HSPs) and translating this science to create a novel treatment for rare neurodegenerative diseases.

ARIMOCLOMOL: Clinical Development Program

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DESIGNATIONS
STAGE OF DEVELOPMENT
ANTICIPATED MILESTONES
Orphan Drug
Fast Track
BTD
RPD*
PC
Ph1
Ph2
Ph3
Filed
Anticipated Milestones
Niemann-Pick disease type C^

DESIGNATIONS

Orphan Drug
Fast Track
BTD
RPD*
Orpha Drug
STAGE OF DEVELOPMENT
PC
Ph1
Ph2
Ph3
Filed
EU: MAA filing submitted to EMA
ANTICIPATED MILESTONES

CHMP opinion Q1 2022

Niemann-Pick disease type C (NPC) is a rare progressive neurodegenerative disease. It belongs to the family of lysosomal storage diseases. Niemann-Pick disease type C can be difficult to recognize, which leads to diagnostic delays. There are no approved treatments in the United States for Niemann-Pick disease type C.
Niemann-Pick disease type C^

DESIGNATIONS

None

Orphan Drug
Fast Track
BTD
RPD*
STAGE OF DEVELOPMENT
PC
Ph1
Ph2
Ph3
Filed
UK: Reliance route application pending
ANTICIPATED MILESTONES

Apply for reliance on EU decision after CHMP opinion

Niemann-Pick disease type C (NPC) is a rare progressive neurodegenerative disease. It belongs to the family of lysosomal storage diseases. Niemann-Pick disease type C can be difficult to recognize, which leads to diagnostic delays. There are no approved treatments in the United States for Niemann-Pick disease type C.
Niemann-Pick disease type C^

DESIGNATIONS

Orphan Drug
Fast Track
BTD
RPD*
Orpha Drug
Orpha Drug
Orpha Drug
Orpha Drug
STAGE OF DEVELOPMENT
PC
Ph1
Ph2
Ph3
Filed
U.S. NDA received CRL§; Type A mtg held
ANTICIPATED MILESTONES

Provide supplemental information & engage in further FDA interactions

Niemann-Pick disease type C (NPC) is a rare progressive neurodegenerative disease. It belongs to the family of lysosomal storage diseases. Niemann-Pick disease type C can be difficult to recognize, which leads to diagnostic delays. There are no approved treatments in the United States for Niemann-Pick disease type C.
^Early Access Program in US, FR, and DE ongoing; Breakthrough Therapy Designation; * Rare Pediatric Disease Designation; § Complete Response Letter

We are focused on lysosomal storage diseases (LSDs):

LYSOSOMAL STORAGE DISEASES (LSDs) are inherited metabolic disorders in which enzyme deficiencies result in an accumulation of toxic materials in the cells of the body. These deficiencies are often caused by mutations leading to premature misfolding and degradation of the enzymes. In Niemann-Pick disease type C (NPC), as well as other LSDs, mutations lead to misfolding and loss of enzyme functions involved in the breakdown and recycling of critical cellular components within the cells recycling centers, the lysosomes. Our research in LSDs focuses on the natural cellular machinery that helps proteins to remain folded in their active state. In particular, we are evaluating HSP family members called HSP70. By amplifying the production of HSPs, this pathological cascade can be addressed by rescuing the function of the recycling enzymes and helping them perform better in the lysosomes.


In July 2020, we completed the rolling submission of a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for arimoclomol for the treatment of NPC. In June 2021, the FDA issued a Complete Response Letter (CRL) following its review of the NDA. Orphazyme remains committed to assessing a path forward for arimoclomol in the U.S.

In October 2021, we held a Type A meeting with the FDA to discuss the key topics in the CRL. Progress was made in understanding the potential resolution of topics outlined in the CRL, including the need for additional data to support NDA. Additionally, the FDA recommended the company provides supplemental information and analyses, and the FDA offered to have further interactions to identify a path to resubmission for arimoclomol in NPC.

We submitted a marketing authorization application to the European Medicines Agency (EMA) for NPC in November 2020.

Research Update

Orphazyme conducted a Phase 2/3 pivotal study of arimoclomol in Inclusion Body Myositis (IBM). Topline results of the study were reported on March 29, 2021; the study did not meet its primary or secondary endpoints. Orphazyme is conducting additional analyses and expects to conclude the study at the appropriate time in the near future.

Orphazyme conducted a Phase 3 pivotal study of arimoclomol in Amyotrophic Lateral Sclerosis (ALS). Topline results of the study were reported on May 7, 2021; the study did not meet its primary or secondary endpoints. Orphazyme is conducting additional analyses and expects to conclude the study at the appropriate time in the near future.

Clinical Trials

To learn more about our clinical trials go to https://www.clinicaltrials.gov/

Early Access Program

Orphazyme has an Early Access Program (EAP) in France and Germany for people living with Niemann-Pick disease type C (NPC). EAPs are sometimes referred to as Expanded Access Programs, compassionate use or similar locally defined pre-approval access programs. We expect the EAP to remain open until the investigational treatment arimoclomol becomes commercially available in these markets.

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