Orphazyme

What Orphazyme does

Orphazyme A/S is a Danish biopharmaceutical company founded in 2009 and headquartered in Copenhagen, Denmark, that historically developed novel therapeutics for rare neurodegenerative diseases. Its core technology was a heat-shock protein (HSP) amplification platform built around arimoclomol, an orally administered small molecule that crosses the blood-brain barrier and has been evaluated in Niemann-Pick disease Type C (NPC), Gaucher disease, sporadic Inclusion Body Myositis (sIBM), and Amyotrophic Lateral Sclerosis (ALS). The lead asset arimoclomol, now marketed as MIPLYFFA by Zevra Therapeutics, was supported by seven Phase I and three Phase II clinical trials and received FDA Breakthrough Therapy Designation for NPC, Fast Track Designation for sIBM, and Orphan Drug Designation in both the EU and USA.

Name

Orphazyme

Legal name

Orphazyme A/S

Website

https://orphazyme.com

Company type

Public

Founded year

2009

Operating status

Operating

Headcount range

1–10 employees

Short description

Orphazyme A/S is a Danish biopharmaceutical holding company that previously developed arimoclomol, a heat-shock protein amplifier for rare neurodegenerative diseases such as NPC, and sold substantially all of its assets to Zevra Therapeutics in May 2022.

Ownership category

akta.pro rank

Product category

Biopharmaceuticals

NAICS

Pharmaceutical and Medicine Manufacturing (3254)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Enzyme Replacement & Metabolic Disorder Therapies (HLAIAIAB)

akta.pro secondary industry

Rare Immunology, Autoimmune & Autoinflammatory Therapies (HLAIAIAE)

Keywords

• Biopharmaceutical therapeutics
• Lysosomal storage diseases
• Heat-shock protein amplification
• Rare neurodegenerative treatments
• Orphan drug development

Headquarters

HQ city

Copenhagen

HQ country

Denmark

HQ region

Europe

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Others

Distribution channels2 records

Marketing channels4 records

Core offering

Orphazyme developed arimoclomol, an investigational oral small molecule that amplifies the production of heat-shock proteins (HSPs), intended to rescue misfolded proteins, clear protein aggregates, and improve lysosomal function. The drug was studied for the treatment of Niemann-Pick disease type C (NPC), Gaucher disease, sporadic Inclusion Body Myositis (sIBM), and Amyotrophic Lateral Sclerosis (ALS). In May 2022, substantially all assets, including arimoclomol, were sold to KemPharm Denmark A/S (now Zevra Therapeutics), which now commercializes the product as MIPLYFFA.

Product overview

Orphazyme was a biopharmaceutical company focused on developing novel therapeutics for neurodegenerative diseases. In May 2022, substantially all assets and business activities, including those relating to the development and approval of arimoclomol (marketed as MIPLYFFA), were sold to KemPharm Denmark A/S (Zevra A/S). The company's primary product was arimoclomol, a heat-shock protein amplifier for the treatment of Niemann-Pick disease type C (NPC), Gaucher disease, sporadic Inclusion Body Myositis (sIBM), and Amyotrophic Lateral Sclerosis (ALS).

Differentiator

Problem solved

Functional benefit

Products and services

• Arimoclomol (marketed as MIPLYFFA) Investigational oral small molecule that amplifies the production of heat-shock proteins (HSPs), used to treat rare neurodegenerative diseases including Niemann-Pick disease type C (NPC), Gaucher disease, ALS, and sporadic Inclusion Body Myositis (sIBM). The drug crosses the blood-brain barrier and was studied across seven Phase I and three Phase II trials. Substantially all assets related to arimoclomol were sold to KemPharm Denmark A/S (now Zevra Therapeutics) in May 2022, and the drug is now commercialized by Zevra as MIPLYFFA.

Quantifiable outcome

• 74% reduction in disease progression (5-domain NPC-CSS) after 12 months compared to placebo (p=0.0506)
• +3 more outcomes

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Partnerships

Two partnerships are on record, tiered minor.

• Kansas Life Sciences Development Company (KLSDC)minorStrategic or Co-development PartnerLicensing partner receiving bonus shares as part of technology licensing arrangement. Capital increases of 26,060 shares in January 2019 and 20,650 shares in January 2020 issued as bonus shares.
• UCL Business PLC (UCLB)minorStrategic or Co-development PartnerAcademic licensing partner receiving bonus shares as part of technology licensing arrangement from University College London.

Scale indicators6 records

Recent moves9 records

Expansion highlights3 records

Direct peers

• IntraBio: Late-stage rare disease company with approved and pipeline therapies for NPC and other lysosomal and neurodegenerative disorders. Direct competitor in the NPC indication that Orphazyme pioneered.
• Ultragenyx Pharmaceutical: Commercial-stage rare disease company developing therapies for metabolic and neurodegenerative disorders, including LSDs. Highly comparable business model targeting ultra-rare patient populations with novel mechanisms of action.
• Zevra Therapeutics: Formerly KemPharm Denmark A/S, Zevra acquired substantially all of Orphazyme's assets in May 2022 and now commercializes arimoclomol (MIPLYFFA) for NPC globally. The most directly comparable company — it owns and continues to develop the platform technology Orphazyme invented.

Broad incumbents

• Alexion (AstraZeneca Rare Disease): Global rare disease franchise focused on metabolic and neurological disorders, including lysosomal storage diseases. Comparable business model of developing and commercializing high-priced orphan therapeutics for very small patient populations.
• Sanofi Genzyme: Sanofi's rare disease unit built around enzyme replacement therapies for LSDs (e.g., Cerezyme, Fabrazyme, Myozyme). Most established incumbent in the same metabolic disorder therapeutic category Orphazyme targeted.
• BioMarin Pharmaceutical: Established rare disease leader with a multi-product lysosomal storage disease franchise (Naglazyme, Vimizim, Aldurazyme). Closest large-cap peer in the LSD therapeutics space where Orphazyme originated.
• Takeda Pharmaceutical (Shire Rare Diseases): Inherited a large rare disease franchise through the Shire acquisition, including LSD and neurological disorder programs. Comparable diversified rare disease strategy with global commercialization reach.
• Actelion (now Janssen / J&J): Historical rare disease leader now part of Janssen, with portfolio overlap in neurological and metabolic orphan indications. Comparable incumbent in bringing first-in-class orphan therapies to ultra-rare patient populations.

Emerging players

• Mandos Health: Emerging company developing cyclodextrin-based therapy for NPC, offering an alternative mechanism of action to Orphazyme's HSP amplification approach. Direct emerging competitor in the NPC niche.
• Gain Therapeutics: Early-stage biotech developing small molecule allosteric regulators for lysosomal storage disorders including Gaucher disease. Comparable emerging player targeting protein misfolding via a different mechanism than Orphazyme's HSP amplification.

Market position

Strengths3 records

Weaknesses4 records

Competitive moat2 records

Key risks6 records

Key highlights5 records

Customer concentration

• LinkedInlinkedin.com/company/orphazyme
• Xx.com/orphazyme_aps

Revenue estimate

Valuation estimate

Number of profiles

Profiles2 records

Funding overview

Funding rounds8 records

Investors9 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments1 record

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Orphazyme