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Baloxavir Marboxil

Phase 3

Influenza | Small molecule | Infectious Disease |Roche Holding AG|Last Updated: May 1, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials6
Total Enrollment5,923

FDA Designations

No designations recorded

Clinical trial landscape

Baloxavir Marboxil · 6 trials · 1 indication

Phase 3 6
NCT06774859Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Chinese Pediatric Participants 1 to <12 Years of Age With Influenza SymptomsInfluenza
COMPLETED100 Analytics
NCT06094010A Surveillance Study of Susceptibility to Baloxavir Marboxil in Pediatric Participants With Influenza and Transmission of Influenza to Household ContactsInfluenza
RECRUITING1,100 Analytics
NCT03969212Study to Assess the Efficacy of Baloxavir Marboxil Versus Placebo to Reduce Onward Transmission of Influenza A or B in HouseholdsInfluenza
COMPLETED4,138 Analytics
NCT03653364Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Healthy Pediatric Participants From Birth to < 1 Year With Influenza-Like SymptomsInfluenza
COMPLETED49 Analytics
NCT03684044Study to Assess Efficacy and Safety of Baloxavir Marboxil In Combination With Standard-of-Care Neuraminidase Inhibitor In Hospitalized Participants With Severe InfluenzaInfluenza
COMPLETED363 Analytics
NCT03629184Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Healthy Pediatric Participants With Influenza-Like SymptomsInfluenza
COMPLETED173 Analytics
PHASE3COMPLETED
Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Chinese Pediatric Participants 1 to <12 Years of Age With Influenza Symptoms
InfluenzaUnlock trial analytics
PHASE3RECRUITING
A Surveillance Study of Susceptibility to Baloxavir Marboxil in Pediatric Participants With Influenza and Transmission of Influenza to Household Contacts
InfluenzaUnlock trial analytics
PHASE3COMPLETED
Study to Assess the Efficacy of Baloxavir Marboxil Versus Placebo to Reduce Onward Transmission of Influenza A or B in Households
InfluenzaUnlock trial analytics
PHASE3COMPLETED
Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Healthy Pediatric Participants From Birth to < 1 Year With Influenza-Like Symptoms
InfluenzaUnlock trial analytics
PHASE3COMPLETED
Study to Assess Efficacy and Safety of Baloxavir Marboxil In Combination With Standard-of-Care Neuraminidase Inhibitor In Hospitalized Participants With Severe Influenza
InfluenzaUnlock trial analytics
PHASE3COMPLETED
Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Healthy Pediatric Participants With Influenza-Like Symptoms
InfluenzaUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Up to Day 29

An AE was defined as any untoward medical occurrence in a participant or clinical study participant temporally associated with the use of a study treatment, whether or not considered related to the study treatment. An AE was therefore any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study intervention. An SAE was defined as any untoward medical occurrence that, at any dose, resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent disability or incapacity, was a congenital anomaly or birth defect.

Part A: Percentage of Participants With Resistance-associated Pre-treatment Substitutions
Day 1 (At Baseline)
Part A: Percentage of Participants With Resistance-associated Treatment-emergent Substitutions
Days 4, 6 and 10
Percentage of HHCs With Virological Influenza Transmission by Day 5
Baseline (Day 1) to Day 5

The virological transmission was determined based on Polymerase Chain Reaction Positive (PCR+) influenza test results. The adjusted incidence (cumulative proportion of events by Day 5) rate is reported here. This is defined as percentage of HHCs who tested PCR+ for influenza by Day 5 post IP randomization with virus subtype matching with that of the respective IP, irrespective of being symptomatic or asymptomatic. The adjusted incidence rates presented were estimated using a generalized estimating equations (GEE) approach.

Time to Clinical Improvement
Up to Day 35

Time to Clinical Improvement (TTCI) is defined as Time to Hospital Discharge OR Time to NEWS2 (National Early Warning Score 2) of ≤ 2 maintained for 24 hours.

Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Up to Day 29

An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the medicinal (investigational) product. A serious adverse event (SAE) is any significant hazard, contraindication, side effect that is fatal or life-threatening, requires hospitalization or prolongation of an existing hospitalization, results in persistent or significant disability/ incapacity, is a congenital anomaly/ birth defect, is medically significant or requires intervention to prevent one or other of the outcomes listed above.

Secondary Endpoints

Time to Alleviation of Influenza Signs and Symptoms (TTAS)
Up to Day 15
Duration of Fever
Up to Day 15
Duration of Symptoms
Up to Day 15
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Baloxavir MarboxilEXPERIMENTALParticipants will receive a single oral dose of baloxavir marboxil on Day 1 based on body weight.
OseltamivirACTIVE_COMPARATORParticipants will receive oseltamivir twice a day for 5 days based on body weight.
PlaceboPLACEBO_COMPARATORParticipants who are IPs will receive a single oral dose of placebo. HHCs of the IPs will not receive study medication.

Interventions

NameTypeDescription
Baloxavir MarboxilDRUGBaloxavir marboxil will be administered as oral suspension: 2 milligrams per kilograms (mg/kg) (if weight \< 20 kg), 40 mg (if weight ≥ 20 kg to \< 80 kg), or 80 mg (if weight ≥ 80 kg).
OseltamivirDRUGOseltamivir will be administered as oral capsule: 30 mg (if weight ≤15 kg), 45 mg (if weight \> 15 kg to ≥ 23 kg), 60 mg (if weight \> 23 kg to ≤ 40 kg) or 75 mg (if weight \> 40 kg), twice daily (BID) for 5 days.
PlaceboDRUGIPs less than 12 years old will receive placebo oral suspension and those above 12 years will receive placebo tablets. HHCs of IPs will not receive study medication.
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Eligibility Criteria

Age Range1 Year to 11 Years
SexALL
Healthy VolunteersNo
Study Sites16

Inclusion Criteria: * A participant who has a diagnosis of influenza virus infection and meets all the following conditions: * Fever ≥ 38°C (tympanic temperature) at screening * At least one of the respiratory symptoms of influenza virus infection * A rapid influenza diagnostic test (RIDT) or ...

Countries:ChinaUnited StatesBulgariaPolandSpainCosta RicaGreeceHungaryIndiaIsraelJapanMexicoPuerto RicoSouth AfricaTurkey (Türkiye)United KingdomFinlandRussiaArgentinaAustraliaBelgiumBrazilCanadaCzechiaEstoniaFranceGermanyHong KongNetherlandsNew ZealandPeruRomaniaSerbiaSingaporeSouth KoreaSwedenUkraine
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Frequently asked questions about Baloxavir Marboxil

What is Baloxavir Marboxil used for?

Baloxavir Marboxil is an investigational small molecule being developed for the treatment of influenza. It is currently in Phase 3 clinical trials, which are evaluating its safety and efficacy in various patient populations, including pediatric and hospitalized patients with severe influenza.

How does Baloxavir Marboxil work?

Baloxavir Marboxil is a small molecule antiviral that targets the influenza virus. It works by inhibiting the cap-dependent endonuclease activity of the viral polymerase, which is essential for viral replication. This mechanism is distinct from neuraminidase inhibitors, which are the standard of care for influenza.

Who is developing Baloxavir Marboxil?

Baloxavir Marboxil is being developed by Roche Holding AG, a multinational healthcare company. Roche is conducting Phase 3 clinical trials to assess the drug's safety, pharmacokinetics, and efficacy in treating influenza across different age groups and severity levels.

What phase is Baloxavir Marboxil in?

Baloxavir Marboxil is in Phase 3 clinical development. It is an investigational drug and has not yet been approved by regulatory authorities. The ongoing and completed Phase 3 trials are designed to support a potential regulatory submission for the treatment of influenza.

What clinical trials is Baloxavir Marboxil in?

Baloxavir Marboxil has been studied in several Phase 3 trials, including NCT03629184 in pediatric participants, NCT03653364 in infants under 1 year, NCT03684044 in hospitalized patients with severe influenza, and NCT03969212 to reduce onward transmission in households. These trials are completed.

Is Baloxavir Marboxil the same as Xofluza?

Baloxavir Marboxil is the generic name for the drug marketed as Xofluza. It is an antiviral medication used to treat influenza. The drug is being developed by Roche and is currently in Phase 3 clinical trials for additional indications and patient populations.