Investigational Drug Information for Olorofim

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What is the development status for investigational drug Olorofim?

Olorofim is an investigational drug.

There have been 7 clinical trials for Olorofim. The most recent clinical trial was a Phase 1 trial, which was initiated on November 30^th 2021.

The most common disease conditions in clinical trials are Mycoses, Invasive Fungal Infections, and Liver Diseases. The leading clinical trial sponsors are F2G Biotech GmbH, F2G Ltd., and Iqvia Pty Ltd.

There are eight US patents protecting this investigational drug and twenty-five international patents.

Summary for Olorofim

US Patents	8
International Patents	25
US Patent Applications	33
WIPO Patent Applications	16
Japanese Patent Applications	0
Clinical Trial Progress	Phase 1 (2021-11-30)
Vendors	38

Recent Clinical Trials for Olorofim

Title	Sponsor	Phase
A Phase I Study to Assess the Pharmacokinetics of Olorofim in Subjects With Renal Impairment	F2G Biotech GmbH	Phase 1
Olorofim Aspergillus Infection Study	Iqvia Pty Ltd	Phase 3
Olorofim Aspergillus Infection Study	F2G Biotech GmbH	Phase 3

See all Olorofim clinical trials

Clinical Trial Summary for Olorofim

Top disease conditions for Olorofim

Top clinical trial sponsors for Olorofim

See all Olorofim clinical trials

US Patents for Olorofim

Drugname	Patent Number	Patent Title	Patent Assignee	Estimated Expiration
Olorofim		Antifungal agents	F2G LIMITED (Manchester, GB)
Olorofim		Antifungal agents	F2G LIMITED (Manchester, GB)
Olorofim		Pharmaceutical formulation	F2G Ltd
Olorofim		Antifungal agents	F2G Ltd

International Patents for Olorofim

Drugname	Country	Document Number	Estimated Expiration	Related US Patent
Olorofim	Australia	AU2015348043	2034-11-21
Olorofim	Brazil	BR112017010439	2034-11-21
Olorofim	Canada	CA2982660	2034-11-21
Olorofim	China	CN107207470	2034-11-21

Olorofim Development Update and Market Projection

Last updated: October 11, 2026

Olorofim is an investigational first-in-class antifungal from F2G Ltd. It is an oral and intravenous orotomide that inhibits fungal dihydroorotate dehydrogenase, an enzyme required for pyrimidine synthesis. As of June 2024, olorofim had not received FDA or European marketing approval. Its principal value proposition is activity against difficult-to-treat invasive mold infections, including azole-resistant Aspergillus, Scedosporium and Lomentospora species.

The commercial base case is a targeted hospital antifungal with peak global sales of approximately $250 million to $500 million if Phase 3 results support approval in the United States and Europe. The upside case exceeds $750 million if olorofim receives a broad label, demonstrates activity in resistant molds and secures first-line or early-line use. The main risks are a narrow eligible-patient population, difficult clinical trial recruitment, diagnostic uncertainty and competition from established azoles and amphotericin products.

What is the current development status of olorofim?

Olorofim is in late-stage clinical development for invasive fungal infections in patients who have limited or no effective treatment options.

Item	Status as of June 2024
Developer	F2G Ltd.
Drug class	Orotomide antifungal
Mechanism	Dihydroorotate dehydrogenase inhibition
Routes	Oral and intravenous
Lead indication	Refractory or otherwise difficult-to-treat invasive fungal infections
Phase 2	Completed, with clinical activity reported in refractory infections
Phase 3	FORMULA-OLS ongoing
FDA status	Breakthrough Therapy designation reported; not approved
EU status	PRIME designation reported; not approved
Commercial partner	Shionogi has a strategic relationship and regional rights
Orange Book	No listing because olorofim is not FDA-approved
Generic competition	No ANDA or Paragraph IV pathway before approval

F2G has positioned olorofim for patients with invasive fungal disease who fail, cannot tolerate or cannot receive standard therapy. The product is being developed for serious infections caused by molds and other fungi for which treatment options are limited.

Olorofim is not a broad replacement for all systemic antifungals. Its clinical positioning will depend on pathogen susceptibility, site of infection, drug interactions, resistance patterns and the availability of rapid fungal diagnostics.

How does olorofim work, and which pathogens does it target?

Olorofim inhibits fungal dihydroorotate dehydrogenase, or DHODH. The target is distinct from the mechanisms used by azoles, polyenes and echinocandins.

The differentiation is relevant because resistance to existing antifungal classes is increasing. Olorofim has shown in vitro and clinical activity against pathogens that can be difficult to treat with current agents, including:

• Azole-resistant Aspergillus species
• Scedosporium species
• Lomentospora prolificans
• Certain endemic and other rare molds

Olorofim is primarily a mold-directed product. It should not be modeled as a universal antifungal for Candida, Cryptococcus, Aspergillus, Mucorales and endemic fungal diseases. Its commercial opportunity will depend on the final label and the pathogens included in treatment guidelines.

The oral formulation could support outpatient step-down treatment, while the intravenous formulation could support hospital initiation in critically ill patients. A combined IV-to-oral regimen would improve the product’s economic value compared with an IV-only drug.

What is the FORMULA-OLS Phase 3 trial?

FORMULA-OLS is the pivotal late-stage program for olorofim. The trial is designed to evaluate patients with serious invasive fungal infections who have limited treatment options, including infections caused by resistant or uncommon molds.

The trial’s commercial importance is high because the product is unlikely to obtain a broad antifungal label from small uncontrolled studies alone. Regulatory agencies will focus on:

1. Overall clinical response.
2. Mortality and infection-related outcomes.
3. Safety and treatment discontinuation.
4. Pharmacokinetic exposure across oral and intravenous dosing.
5. Outcomes by pathogen, including resistant molds.
6. The consistency of benefit across underlying conditions.

The trial population presents a development challenge. Invasive mold infections are heterogeneous and often occur in immunocompromised patients with multiple causes of mortality. Pathogen confirmation can be delayed or unavailable, making enrollment and endpoint interpretation difficult.

A positive Phase 3 result would support a differentiated label for patients with refractory or otherwise difficult-to-treat invasive fungal infections. A weaker result could restrict the label to salvage therapy, limiting market penetration.

When could olorofim reach the market?

Olorofim had no approved filing or commercial launch as of June 2024. A reasonable development timeline is:

Milestone	Base-case timing
Phase 3 enrollment and final follow-up	2024-2025
Top-line Phase 3 data	2025-2026
U.S. NDA submission	2026
Potential FDA decision	2027
European filing and review	2026-2027
Potential EU launch	2027-2028
Initial commercial launch	2027-2028

The timetable depends on the final database, whether the FDA accepts a single pivotal study, the use of priority review and the completeness of manufacturing data.

Olorofim has received regulatory designations intended to accelerate development in serious diseases with unmet need, including FDA Breakthrough Therapy designation and European PRIME-related support reported by the company. These designations do not guarantee approval or shorten every part of the review process.

What is the FDA regulatory and Orange Book status of olorofim?

Olorofim was not FDA-approved as of June 2024 and therefore had no Orange Book listing, approved label, reference product status or authorized generic pathway.

The likely U.S. regulatory route is a traditional New Drug Application supported by clinical efficacy, safety, pharmacology and chemistry, manufacturing and controls data. The product may also qualify for regulatory incentives associated with treatment of serious fungal infections and rare diseases.

Potential U.S. regulatory advantages include:

• Breakthrough Therapy interactions with the FDA
• Orphan drug designation if the final indication meets prevalence requirements
• Qualified Infectious Disease Product incentives, if granted
• Priority review eligibility
• Seven years of orphan-drug exclusivity, if approved for an orphan indication
• Five years of new chemical entity exclusivity, subject to the final FDA classification

Regulatory exclusivity would operate separately from patent protection. The absence of an Orange Book listing currently prevents a conventional Paragraph IV challenge.

What patents protect olorofim, and when could exclusivity expire?

F2G’s patent position is expected to include compound, pharmaceutical composition, formulation, treatment-method and manufacturing claims. Public company materials have not provided a single, definitive global patent-expiration date that can be used as the sole commercial cutoff.

The likely protection structure is:

Protection category	Commercial purpose
Olorofim compound claims	Protect the active pharmaceutical ingredient
Salt and crystalline-form claims	Protect solid-state forms and drug substance handling
Pharmaceutical composition claims	Cover oral and intravenous products
Method-of-use claims	Cover treatment of invasive fungal infections and defined pathogens
Dosing claims	Protect exposure, loading and maintenance regimens
Manufacturing claims	Protect scalable production and purification
Regulatory exclusivity	Delay competing approval after launch

The core compound patent is likely to provide the principal barrier through the early-to-mid 2030s, subject to the applicable filing date, patent-term adjustment, patent-term extension and jurisdiction. Later formulation and method patents could extend protection beyond the core compound term, but those patents generally provide narrower enforcement rights.

A definitive Orange Book patent analysis cannot be performed before approval and listing. Once an NDA is approved, the commercial assessment should examine listed patents, expiration dates, pediatric extensions, patent-term restoration and any use codes.

What Paragraph IV and generic-entry risks exist?

There is no current Paragraph IV litigation because olorofim has no approved reference product and no Orange Book listing.

After approval, generic entry would face several hurdles:

• The core active ingredient may remain protected.
• A generic applicant would need to establish bioequivalence for oral dosage forms.
• The IV formulation may require separate pharmaceutical development.
• The reference label could include protected method-of-use indications.
• Rare-disease or infectious-disease exclusivity could delay approval.
• Clinical endpoint requirements may make substitution less straightforward than for a routine oral drug.

The most likely first challenge would target formulation or method-of-use patents rather than the core compound patent. Generic launch before core patent expiry would require a successful invalidity or non-infringement strategy, an authorized generic arrangement or a settlement permitting an earlier entry date.

No public olorofim Paragraph IV dispute or generic settlement had been reported as of June 2024.

What formulation and manufacturing IP could protect olorofim?

The oral and intravenous presentations create separate protection opportunities. The oral product may use a solid dosage form designed to achieve adequate exposure despite the compound’s physicochemical properties. The IV product requires controls for solubility, stability, sterilization and infusion compatibility.

Manufacturing risks include:

• Consistent production of drug substance at commercial scale
• Control of impurities and polymorphic forms
• Stable oral tablets or capsules
• A robust IV formulation
• Compatibility with hospital infusion systems
• Process validation for global regulatory filings

Manufacturing patents can create practical barriers even when compound patents face validity challenges. Their value depends on whether competitors can develop a non-infringing process or alternative formulation.

How does olorofim compare with competing antifungals?

Product	Class	Main strengths	Key limitations relative to olorofim
Voriconazole	Triazole	Established Aspergillus treatment; oral and IV	Drug interactions, toxicity, resistance
Isavuconazonium	Triazole	Oral and IV; generally favorable tolerability	Resistance and limited activity against some rare molds
Posaconazole	Triazole	Broad mold activity; prophylaxis and treatment use	Absorption, interactions and resistance
Liposomal amphotericin B	Polyene	Broad activity; established salvage use	Nephrotoxicity and IV-only administration
Olorofim	Orotomide	Novel mechanism; potential activity in resistant molds; oral and IV	Unapproved, limited clinical experience and uncertain label

Olorofim’s strongest competitive position is likely in salvage and resistant-infection treatment. It will need evidence of superior tolerability, meaningful activity in difficult pathogens or a clinically useful oral regimen to move earlier in treatment algorithms.

Its most important competitors are not only branded antifungals. Low-cost generic voriconazole, posaconazole and amphotericin B will constrain pricing and may remain preferred when susceptibility and tolerability are acceptable.

Which companies are involved in olorofim commercialization?

F2G is the originator and primary developer. Shionogi has entered into a strategic relationship with F2G that includes investment and regional commercialization rights reported for Japan and Taiwan. The full economic terms and global commercialization structure were not publicly disclosed in the materials available through June 2024.

A global launch could require:

• A North American commercial partner
• European hospital-sales infrastructure
• Specialist medical-science teams
• Diagnostic partnerships
• Distribution capabilities for tertiary hospitals
• Pharmacovigilance and resistance surveillance

The optimal sales model is likely a focused infectious-disease and hospital account team rather than a primary-care launch.

What is the market size and revenue projection for olorofim?

The relevant market is the treated population with serious invasive mold infection, not the entire systemic antifungal market. Olorofim’s addressable population includes patients with resistant Aspergillus, Scedosporium, Lomentospora and other infections for which existing drugs fail or are poorly tolerated.

A scenario-based revenue forecast is more appropriate than a single-point estimate.

Scenario	Peak annual sales	Assumptions
Downside	$75 million-$150 million	Salvage-only label, limited pathogen coverage, delayed approval
Base case	$250 million-$500 million	U.S. and European approval, IV and oral use, adoption in resistant molds
Upside	$750 million-$1.0 billion	Broad refractory-mold label, strong survival or response data, earlier-line use

A base-case launch model could include:

• U.S. net sales of $125 million-$250 million at peak
• European sales of $75 million-$150 million
• Japan and selected Asia-Pacific sales of $25 million-$75 million
• Other markets contributing a smaller amount

Pricing will likely reflect hospital treatment value and orphan infectious-disease positioning. A treatment course could command a premium to generic azoles, but payer restrictions would limit use to documented refractory disease or resistant pathogens.

The key valuation driver is not total antifungal market size. It is the percentage of high-risk patients for whom physicians select olorofim instead of amphotericin, isavuconazole, posaconazole or voriconazole.

How strong is the commercial and patent outlook for olorofim?

Olorofim has a potentially strong product profile but a narrow and execution-sensitive commercial case.

Its strengths are:

• Novel mechanism of action
• Potential activity against resistant molds
• Oral and IV formulations
• High unmet need in salvage treatment
• Regulatory designations for serious fungal disease
• Limited direct class competition

Its weaknesses are:

• No approval as of June 2024
• Dependence on a difficult Phase 3 population
• Small and heterogeneous market
• Limited ability to compete on price against generic antifungals
• Uncertain long-term resistance profile
• Lack of a publicly confirmed, comprehensive patent-expiration schedule
• Need for rapid and accurate fungal diagnosis

The investment profile improves materially if Phase 3 results show consistent benefit across Aspergillus and rare mold infections, with manageable hepatic, neurologic and drug-interaction risks. It weakens if the final evidence supports only a narrowly defined rescue population.

Key Takeaways

• Olorofim is a first-in-class orotomide antifungal developed by F2G.
• The product was in Phase 3 development as of June 2024 and had not been approved by the FDA or EMA.
• Its target market is refractory or resistant invasive mold infection, not routine fungal disease.
• The strongest differentiation is potential activity against azole-resistant Aspergillus, Scedosporium and Lomentospora.
• No Orange Book listing, Paragraph IV litigation or generic settlement existed before approval.
• A reasonable approval window is 2027-2028, assuming positive Phase 3 results and a standard regulatory review.
• Base-case peak sales are approximately $250 million to $500 million globally.
• The principal risks are clinical-trial execution, narrow market size, pathogen diagnosis and competition from inexpensive generic antifungals.

FAQs

Is olorofim approved by the FDA?

No. Olorofim had not received FDA approval as of June 2024. It remained an investigational drug in late-stage development.

Can olorofim treat mucormycosis?

Olorofim is being developed mainly for difficult-to-treat mold infections, including resistant Aspergillus, Scedosporium and Lomentospora. Its activity against Mucorales is not sufficient to treat it as a general replacement for liposomal amphotericin B.

Does olorofim have biosimilar risk?

No conventional biosimilar risk exists because olorofim is a small-molecule antifungal, not a biologic. The relevant future competitors would be generic or authorized-generic versions, subject to patents and regulatory exclusivity.

What could limit olorofim’s market share?

The main constraints are a narrow salvage-treatment label, uncertain diagnosis of rare molds, generic competition, hospital formulary controls and limited evidence outside the most difficult patient populations.

Is Shionogi the global owner of olorofim?

F2G is the originator and developer. Shionogi has a strategic relationship and reported regional rights, including Japan and Taiwan. The publicly disclosed information did not establish Shionogi as the global owner of all olorofim rights.

References

1. F2G Ltd. (2024). Olorofim development program and corporate information. F2G Ltd.

2. ClinicalTrials.gov. (2024). FORMULA-OLS: Study of olorofim versus standard of care in patients with invasive fungal infections. U.S. National Library of Medicine.

3. U.S. Food and Drug Administration. (2024). Breakthrough therapy designation and qualified infectious disease product information. U.S. Department of Health and Human Services.

4. European Medicines Agency. (2024). PRIME scheme and medicines for serious or life-threatening conditions. European Medicines Agency.

5. Shionogi & Co., Ltd. (2021). Strategic investment and collaboration with F2G Ltd. relating to olorofim. Shionogi & Co., Ltd.

6. Patterson, T. F., Thompson, G. R., Denning, D. W., Fishman, J. A., Hadley, S., Herbrecht, R., et al. (2016). Practice guidelines for the diagnosis and management of aspergillosis. Clinical Infectious Diseases, 63(4), e1-e60.

7. World Health Organization. (2022). WHO fungal priority pathogens list to guide research, development and public health action. World Health Organization.