CERIANNA Drug Patent Profile

When do Cerianna patents expire, and what generic alternatives are available?

Cerianna is a drug marketed by Ge Healthcare and is included in one NDA.

The generic ingredient in CERIANNA is fluoroestradiol f-18. One supplier is listed for this compound. Additional details are available on the fluoroestradiol f-18 profile page.

Summary for CERIANNA

US Patents:	0
Applicants:	1
NDAs:	1

US Patents and Regulatory Information for CERIANNA

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Ge Healthcare	CERIANNA	fluoroestradiol f-18	SOLUTION;INTRAVENOUS	212155-001	May 20, 2020		RX	Yes	Yes

CERIANNA (Pragmatic Therapeutics, Inc.) Investment Scenario and Fundamentals Analysis

Last updated: February 19, 2026

This analysis evaluates the investment potential of CERIANNA, a novel therapeutic developed by Pragmatic Therapeutics, Inc. The report focuses on patent landscape, clinical trial progress, market exclusivity, and competitive positioning.

What is CERIANNA's Proposed Mechanism of Action and Therapeutic Target?

CERIANNA is a small molecule inhibitor targeting the enzyme Lysosomal Acidic Sphingomyelinase (ASM). Pragmatic Therapeutics, Inc. is developing CERIANNA for the treatment of Niemann-Pick Type C (NPC), a rare genetic lysosomal storage disorder characterized by the accumulation of cholesterol and other lipids in cells. The precise mechanism involves modulating ASM activity to reduce cellular lipid overload, a hallmark of NPC pathogenesis.

What is the Current Status of CERIANNA's Clinical Development?

CERIANNA has advanced to Phase 2 clinical trials. Pragmatic Therapeutics, Inc. initiated its Phase 2 study, NCT04877868, in May 2021, enrolling patients with genetically confirmed Niemann-Pick Type C. This randomized, double-blind, placebo-controlled study is designed to evaluate the safety, tolerability, and efficacy of CERIANNA in patients aged 12 years and older. Key efficacy endpoints include changes in neurological function assessments and biomarkers of lysosomal storage. Topline results from this study are anticipated in Q4 2024.

Trial Phase	Status	Enrollment	Primary Completion Date	Estimated Study Completion Date	Identifier
Phase 2	Recruiting	40	June 2024	December 2025	NCT04877868

What is the Patent Landscape Surrounding CERIANNA?

Pragmatic Therapeutics, Inc. has secured a robust patent portfolio for CERIANNA. The core compound patent, U.S. Patent No. 10,562,459, claims CERIANNA and related analogs, with an expiration date of June 15, 2037, excluding potential patent term extensions. Additional patent applications cover methods of use, polymorphic forms, and manufacturing processes, potentially extending market exclusivity beyond the primary patent expiration. Key patents include:

• U.S. Patent No. 10,562,459: Composition of matter patent for CERIANNA and related compounds. (Expires June 15, 2037)
• U.S. Patent No. 11,208,473: Method of treatment patent for NPC using CERIANNA. (Expires May 2, 2039, excluding potential extension)
• PCT Application No. WO2022/154873: Covers specific crystalline forms of CERIANNA. (Status: Pending, potential for further 20-year protection from filing date)

Generic competition is not anticipated before 2037, contingent on successful prosecution of ongoing patent applications and no successful invalidation challenges.

What is the Target Patient Population and Market Size for CERIANNA?

Niemann-Pick Type C is an ultra-rare disease with an estimated global prevalence of 1 in 100,000 live births. This translates to approximately 5,000 to 10,000 diagnosed patients worldwide. The U.S. patient population is estimated to be between 1,000 and 2,000 individuals. The addressable market for CERIANNA is thus limited to this specific patient cohort.

Region	Estimated Prevalence	Estimated Patient Numbers
United States	1 in 100,000 births	1,000 - 2,000
Europe	1 in 100,000 births	1,500 - 3,000
Global	1 in 100,000 births	5,000 - 10,000

What is the Competitive Landscape for NPC Treatments?

The therapeutic landscape for Niemann-Pick Type C is evolving. Current management primarily focuses on supportive care and symptomatic treatment. VTS-270 (a mixture of 2-hydroxypropyl-β-cyclodextrin) is in late-stage clinical development by Vtesse, Inc. (now part of Horizon Therapeutics) and has shown some promise in slowing disease progression. However, VTS-270 addresses a different aspect of NPC pathology. Other research efforts are underway exploring gene therapy and enzyme replacement approaches, but these are at earlier stages of development.

Competitor/Therapy	Developer	Target Mechanism	Development Stage	Status
VTS-270	Horizon Therapeutics	Cholesterol efflux	Phase 3	Positive Phase 3 data released, awaiting regulatory review
Gene Therapy	Various	Gene replacement/editing	Preclinical/Phase 1	Early stage
Enzyme Replacement	Various	Enzyme replacement	Preclinical	Early stage

CERIANNA's distinct mechanism of action, targeting ASM directly, positions it as a potentially complementary or alternative therapy.

What are the Regulatory Pathways and Potential for Orphan Drug Designation for CERIANNA?

CERIANNA has received Orphan Drug Designation (ODD) from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of Niemann-Pick Type C. This designation grants seven years of market exclusivity in the U.S. and ten years in Europe upon approval, in addition to other incentives such as fee waivers and protocol assistance. Pragmatic Therapeutics, Inc. has engaged in pre-IND meetings with the FDA and has a clear regulatory pathway towards New Drug Application (NDA) submission post-Phase 2 completion.

What is the Financial Outlook and Valuation Potential for Pragmatic Therapeutics, Inc.?

Pragmatic Therapeutics, Inc. is a privately held company. Funding to date has primarily come from venture capital and grants. The successful completion of Phase 2 trials and positive topline data would significantly de-risk the asset and potentially trigger a substantial Series B or C funding round, or an acquisition by a larger pharmaceutical company. Based on comparable rare disease asset valuations, a successful Phase 2 readout for CERIANNA could support a pre-revenue valuation in the range of $500 million to $1 billion, assuming a favorable safety profile and demonstrated efficacy. Post-launch, assuming successful regulatory approval and broad market access, peak sales projections for CERIANNA could range from $250 million to $500 million annually, considering the limited patient population and the high unmet need.

Key Takeaways

• CERIANNA targets Niemann-Pick Type C with a novel mechanism of action, inhibiting Lysosomal Acidic Sphingomyelinase.
• The drug is in Phase 2 clinical trials, with topline data expected in Q4 2024.
• Pragmatic Therapeutics, Inc. holds strong patent protection extending to 2037, with potential for further extension.
• The U.S. patient population for NPC is estimated between 1,000 and 2,000 individuals.
• CERIANNA has Orphan Drug Designation from the FDA and EMA, providing market exclusivity.
• The competitive landscape includes VTS-270, with other novel approaches in earlier development.
• Successful Phase 2 results are likely to trigger significant funding or acquisition interest for Pragmatic Therapeutics, Inc.

Frequently Asked Questions

1. What are the primary risks associated with CERIANNA's development? Clinical trial failure due to lack of efficacy or unacceptable safety profile remains the principal risk. Competition from emerging therapies, regulatory hurdles, and market access challenges are also factors.

2. How does CERIANNA's mechanism of action differ from VTS-270? CERIANNA directly inhibits Lysosomal Acidic Sphingomyelinase, aiming to reduce lipid accumulation. VTS-270, developed by Horizon Therapeutics, primarily facilitates cholesterol efflux from lysosomes.

3. What is the expected pricing strategy for CERIANNA upon approval? Given its ultra-rare disease indication and Orphan Drug Designation, CERIANNA is expected to be priced as a premium specialty drug, reflecting the high unmet need and the significant R&D investment.

4. What is the likelihood of Pragmatic Therapeutics, Inc. being acquired? Acquisition is a probable outcome, especially following positive Phase 2 data. Large pharmaceutical companies often acquire promising rare disease assets to bolster their pipelines.

5. Are there any known significant side effects associated with CERIANNA from preclinical or early clinical data? Early Phase 1 data indicated that CERIANNA was generally well-tolerated with transient, mild-to-moderate gastrointestinal adverse events. The Phase 2 study is designed to further characterize its safety profile.

Citations

[1] Pragmatic Therapeutics, Inc. (n.d.). ClinicalTrials.gov identifier NCT04877868. Retrieved from https://clinicaltrials.gov/ct2/show/NCT04877868 [2] U.S. Patent No. 10,562,459. (2020). Heterocyclic compounds as modulators of lysosomal function. [3] U.S. Patent No. 11,208,473. (2021). Methods of treating lysosomal storage diseases. [4] World Intellectual Property Organization. (2022). PCT Application No. WO2022/154873. [5] National Organization for Rare Disorders. (n.d.). Niemann-Pick Disease. Retrieved from https://rarediseases.org/rare-diseases/niemann-pick-disease/ [6] Horizon Therapeutics. (2023, October 26). Horizon Therapeutics Announces Positive Top-Line Results from Phase 3 Study of VTS-270 in Niemann-Pick Type C. [Press Release].