Orphazyme
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.
- Company typePublic
- Founded2009
- HeadquartersCopenhagen, Denmark
- Headcount1–10
- GTM typeB2B
- OfferingHardware or Manufacturing
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.
Orphazyme firmographics
Firmographics- 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
Orphazyme industry classification
Industry- 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
Where Orphazyme is headquartered
LocationHeadquarters
- HQ city
- Copenhagen
- HQ country
- Denmark
- HQ region
- Europe
Offices3 records
Markets served
Orphazyme business model
Business model- 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
Orphazyme product offering
Product offeringCore 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
Companies that use Orphazyme
Customer profileSegments1 record
Ideal customer profiles2 records
Orphazyme technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature1 record
Orphazyme partnerships and signals
Strategic signalPartnerships
Two partnerships are on record, tiered minor.
- Kansas Life Sciences Development Company (KLSDC)minorLicensing 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)minorAcademic licensing partner receiving bonus shares as part of technology licensing arrangement from University College London.
Scale indicators6 records
Recent moves9 records
Expansion highlights3 records
Orphazyme competitors and assessment
Company assessmentDirect 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
Orphazyme social profiles
Digital presenceOrphazyme financial estimates
Financial estimateRevenue estimate
Valuation estimate
Orphazyme leadership team
Management profileNumber of profiles
Profiles2 records
Orphazyme funding detail
Funding detailFunding overview
Funding rounds8 records
Investors9 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Orphazyme M&A and investment
M&A and investmentM&A
Investments1 record
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Orphazyme
What does Orphazyme do?
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.
Is Orphazyme a public or private company?
Orphazyme is a public company. It is classified as public and is currently operating.
When was Orphazyme founded?
Orphazyme was founded in 2009. It employs 1 to 10 people.
Where is Orphazyme based?
Orphazyme is headquartered in Copenhagen, Denmark, in the Europe region.
Who are Orphazyme's main competitors?
Direct peers on record are IntraBio, Ultragenyx Pharmaceutical and Zevra Therapeutics. Broad incumbents are Alexion (AstraZeneca Rare Disease), Sanofi Genzyme, BioMarin Pharmaceutical, Takeda Pharmaceutical (Shire Rare Diseases) and Actelion (now Janssen / J&J). Emerging players are Mandos Health and Gain Therapeutics.
Does Orphazyme have an API?
No public API is recorded for Orphazyme.
What industry is Orphazyme in?
Orphazyme's product category is Biopharmaceuticals. Its primary akta.pro industry code is HLAIAIAB, Enzyme Replacement & Metabolic Disorder Therapies, with a secondary code of HLAIAIAE, Rare Immunology, Autoimmune & Autoinflammatory Therapies. Its NAICS code is 3254 and its SIC code is 2836.