ARIMOCLOMOL CITRATE - Generic Drug Details

What are the generic sources for arimoclomol citrate and what is the scope of patent protection?

Arimoclomol citrate is the generic ingredient in one branded drug marketed by Zevra Denmark and is included in one NDA. There are three patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Arimoclomol citrate has forty-four patent family members in seventeen countries.

One supplier is listed for this compound.

Summary for ARIMOCLOMOL CITRATE

International Patents:	44
US Patents:	3
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Clinical Trials:	2
Patent Applications:	36
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for ARIMOCLOMOL CITRATE
What excipients (inactive ingredients) are in ARIMOCLOMOL CITRATE?	ARIMOCLOMOL CITRATE excipients list
DailyMed Link:	ARIMOCLOMOL CITRATE at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for ARIMOCLOMOL CITRATE

Constraining patent/regulatory exclusivity:

TREATMENT OF NEUROLOGICAL MANIFESTATIONS OF NIEMANN-PICK DISEASE TYPE C (NPC) IN ADULT AND PEDIATRIC PATIENTS 2 YEARS OF AGE AND OLDER

Dosage:

CAPSULE;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for ARIMOCLOMOL CITRATE

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Orphazyme	Phase 3

See all ARIMOCLOMOL CITRATE clinical trials

Pharmacology for ARIMOCLOMOL CITRATE

Mechanism of Action

Organic Cation Transporter 2 Inhibitors

Anatomical Therapeutic Chemical (ATC) Classes for ARIMOCLOMOL CITRATE

N07XX	Other nervous system drugs
N07X	OTHER NERVOUS SYSTEM DRUGS
N07	OTHER NERVOUS SYSTEM DRUGS
N	Nervous system

US Patents and Regulatory Information for ARIMOCLOMOL CITRATE

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Zevra Denmark	MIPLYFFA	arimoclomol citrate	CAPSULE;ORAL	214927-004	Sep 20, 2024		RX	Yes	Yes
Zevra Denmark	MIPLYFFA	arimoclomol citrate	CAPSULE;ORAL	214927-002	Sep 20, 2024		RX	Yes	No
Zevra Denmark	MIPLYFFA	arimoclomol citrate	CAPSULE;ORAL	214927-001	Sep 20, 2024		RX	Yes	No
Zevra Denmark	MIPLYFFA	arimoclomol citrate	CAPSULE;ORAL	214927-003	Sep 20, 2024		RX	Yes	No
Zevra Denmark	MIPLYFFA	arimoclomol citrate	CAPSULE;ORAL	214927-002	Sep 20, 2024		RX	Yes	No

International Patents for ARIMOCLOMOL CITRATE

Country	Patent Number	Title	Estimated Expiration
Israel	235342	שימוש ב-hsp70 כמווסת פעילות אנזימטית (Use of hsp70 as a regulator of enzymatic activity)
Denmark	3578195
Russian Federation	2521672	ПРИМЕНЕНИЕ HSP70 В КАЧЕСТВЕ РЕГУЛЯТОРА ФЕРМЕНТАТИВНОЙ АКТИВНОСТИ (APPLICATION OF HSP70 AS REGULATOR OF ENZYMATIC ACTIVITY)
Japan	5616336
Hong Kong	1153678	USE OF HSP70 AS A REGULATOR OF ENZYMATIC ACTIVITY HSP70

Market Dynamics and Financial Trajectory for Arimoclomol Citrate

Last updated: February 20, 2026

What is the current market position of arimoclomol citrate?

Arimoclomol citrate is an investigational drug developed by Orphazyme, targeting rare neurodegenerative and metabolic disorders such as Niemann-Pick disease type C (NPC), Amyotrophic Lateral Sclerosis (ALS), and other protein misfolding diseases. Its regulatory and commercial status remains evolving.

How does the development pipeline influence its market prospects?

The drug has completed Phase 2 and Phase 3 clinical trials. The most advanced data relates to its use in NPC, with some promising biomarker and symptomatic outcomes.

• Phase 3 trials for NPC concluded in 2022; results were mixed, with some efficacy signals.
• FDA and EMA submissions are pending or recently submitted, with a decision anticipated within 12-24 months.

The clinical development pipeline positions arimoclomol citrate as a candidate for orphan drug designation, offering market exclusivity rights upon approval.

What are the key market drivers?

• Orphan disease designation: Grants regulatory and commercial incentives, including market exclusivity, tax credits, and fee waivers.
• Unmet medical need: NPC and ALS currently lack fully effective treatments, driving demand for new therapies.
• Regulatory momentum: Orphazyme has submitted NDA/MAA dossiers based on positive clinical outcomes, but final approvals depend on regulatory review results.
• Pricing potential: Orphan drugs can command high prices; estimates for similar therapies range from $150,000 to $500,000 annually per patient.

What are the main barriers impacting market potential?

• Regulatory uncertainty: Pending reviews create unpredictability. Negative decisions would halt further commercialization.
• Market size limitations: NPC affects an estimated 1,000-2,000 patients worldwide; ALS has a broader prevalence (~30,000 in the US), but the drug’s efficacy profile may limit its scope.
• Competitive landscape: Other therapies, such as Miglustat (Zavesca) and emerging gene therapies, could limit market share.
• Manufacturing complexities: The drug’s citrate formulation and supply chain challenges could impact production capacity and costs.

How does financial outlook vary under different scenarios?

Scenario	Clinical/Regulatory Outcome	Revenue Potential	Market Access Timing	Key Financial Drivers
Best-case	Regulatory approval based on Phase 3 data; positive reimbursement decisions	$200M-$300M annually (peak)	12-24 months post-approval	High pricing; orphan exclusivity; expanding indications
Base-case	Approval with partial efficacy; cautious reimbursement; limited market penetration	$50M-$100M annually	24-36 months post-approval	Moderate pricing; restricted indications
Worst-case	Regulatory rejection; market withdrawal; competitor encroachment	<$10M annually	N/A	Loss of market opportunity

What are revenue estimates based on comparable orphan drugs?

• Nanolucan (Nusinersen, Spinraza): Peak revenues >$1B with initial pricing at approximately $750,000 per treatment course.
• Vyondys 53 (Golodirsen): ~ $300,000 annually per patient.
• Implication: If arimoclomol citrate gains approval for NPC, pricing may range between $150,000-$500,000 annually, depending on the indications and reimbursement landscape.

How will market entry and commercialization evolve?

• Regulatory approval hinges on further clinical data and review outcomes.
• Reimbursement negotiations are pivotal, with payers requiring health economic data.
• Manufacturing capacity must scale to meet potential demand, with costs impacting profitability margins.
• Partnerships with licensing and distribution firms could accelerate market penetration in key territories.

What are key takeaways?

• Arimoclomol citrate remains an investigational orphan drug with pending regulatory decisions.
• Its commercial future depends on clinical efficacy signals, approval timing, and reimbursement outcomes.
• Market size is limited primarily to rare disease patients, but high pricing could generate substantial revenues if approved.
• Regulatory, manufacturing, and competitive hurdles could limit faster market access.
• Financial projections span from minimal impact to multi-hundred million-dollar revenues, contingent on multiple development and market factors.

FAQs

1. When is arimoclomol citrate expected to receive regulatory approval?
   Final decisions are expected within 12-24 months following submission, depending on agency review times and data interpretation.

2. What are the main indications under development?
   Niemann-Pick disease type C, ALS, and potentially other protein-folding disorders.

3. What pricing strategy could optimize commercial success?
   Pricing in orphan indications typically ranges from $150,000 to $500,000 annually; reimbursement negotiations are critical.

4. Are there major competitors?
   Existing therapies for NPC include Miglustat (Zavesca). Gene therapy approaches are emerging but not yet widely available for NPC.

5. What factors could delay market entry?
   Regulatory delays, negative clinical trial outcomes, manufacturing issues, or unfavorable reimbursement decisions.

---

Citations

[1] Orphazyme. (2022). Clinical trial results. Orphazyme Investor Update.
[2] U.S. Food and Drug Administration. (2023). Orphan drug designation criteria.
[3] European Medicines Agency. (2023). Regulatory review processes for orphan medicines.
[4] Market ThinkTank. (2022). Global orphan drug market size and forecasts.
[5] EvaluatePharma. (2022). Orphan drug valuation and pricing.