LISRAYA Film-coated tablet Ref.[116910] Active ingredients: Brepocitinib

Source: FDA, National Drug Code (US)  Revision Year: 2026 

4. Contraindications

LISRAYA is contraindicated in patients with a history of hypersensitivity reactions to brepocitinib or any of the excipients in LISRAYA [see Warnings and Precautions (5.6)].

5. Warnings and Precautions

5.1 Serious Infections

LISRAYA increases the risk of infections, including serious bacterial, fungal, viral, and opportunistic infections that may lead to hospitalization or death. The most common serious infections reported with LISRAYA were pneumonia and sepsis [see Adverse Reactions (6.1)]. Other reported infections with use of JAK inhibitors, including LISRAYA, were tuberculosis, which may present with pulmonary or extrapulmonary disease, invasive fungal infections which may present with disseminated rather than localized disease, bacterial infections, viral infections (including herpes zoster), and other infections due to opportunistic pathogens.

Avoid use of LISRAYA in patients with an active, serious infection, including localized infections. Consider the risks and benefits of treatment prior to initiating LISRAYA in patients:

  • with chronic or recurrent infection
  • who have been exposed to TB
  • with a history of a serious or an opportunistic infection
  • who have resided or traveled in areas of endemic TB or endemic mycoses; or
  • with underlying conditions that may predispose them to infection.

Closely monitor patients for signs and symptoms of infection during and after treatment with LISRAYA. If a patient develops a serious infection, including a serious opportunistic infection, interrupt LISRAYA treatment until the infection resolves or is adequately treated. In patients who develop a new infection during treatment with LISRAYA, promptly complete diagnostic testing, initiate appropriate antimicrobial therapy, and monitor the patients closely. LISRAYA may be resumed once the infection resolves or is adequately treated.

Tuberculosis

Evaluate and test patients for latent and active TB infection prior to and during administration of LISRAYA. If positive, treat for TB prior to LISRAYA treatment.

Consider anti-TB therapy prior to initiation of LISRAYA in patients with a past history of latent or active TB in whom an adequate course of treatment cannot be confirmed. Consultation with a physician with expertise in the treatment of TB is recommended to aid in the decision about whether initiating anti-TB therapy is appropriate for an individual patient.

Monitor patients, including patients who tested negative for latent TB infection prior to LISRAYA treatment, for signs and symptoms of active TB during LISRAYA treatment.

Viral Reactivation

Viral reactivation, including cases of herpes virus reactivation (e.g., herpes zoster) were reported in patients who received LISRAYA. If a patient develops herpes zoster, consider interrupting LISRAYA until the episode resolves.

Prior to initiating LISRAYA treatment, perform viral hepatitis screening in accordance with clinical guidelines. Monitor patients for viral hepatitis reactivation during therapy with LISRAYA. Patients who were positive for hepatitis C antibody and hepatitis C virus RNA were excluded from clinical trials. Patients who were positive for hepatitis B surface antigen or hepatitis B virus DNA were excluded from clinical trials. If hepatitis B virus DNA is detected during LISRAYA treatment, consult a liver specialist. LISRAYA is not recommended in patients with active hepatitis B or hepatitis C.

5.2 Mortality

In a large, randomized, postmarketing safety study of another JAK inhibitor in patients with rheumatoid arthritis (RA) 50 years of age and older with at least one cardiovascular risk factor, a higher rate of all-cause mortality, including sudden cardiovascular death, was observed in patients treated with the JAK inhibitor compared with TNF blockers. Consider the benefits and risks for the individual patient prior to initiating or continuing LISRAYA treatment. LISRAYA is not approved for the treatment of RA.

5.3 Malignancy and Lymphoproliferative Disorders

Malignancies were observed in clinical trials of LISRAYA.

In a large, randomized, postmarketing safety study of another JAK inhibitor in patients with RA, a higher rate of malignancies (excluding non-melanoma skin cancer [NMSC]) and lymphomas were observed in patients treated with the JAK inhibitor compared to those treated with TNF blockers. A higher rate of lung cancers was observed in current or past smokers treated with the JAK inhibitor compared to those treated with TNF blockers. In this study, current or past smokers had an additional increased risk of overall malignancies. LISRAYA is not approved for the treatment of RA.

Consider the benefits and risks for the individual patient prior to initiating or continuing LISRAYA treatment, particularly in patients with a known malignancy (other than a successfully treated NMSC) and patients who develop a malignancy during treatment with LISRAYA.

Advise patients to limit exposure to ultraviolet (UV) light (natural or artificial) by wearing protective clothing and using a broad-spectrum sunscreen. Periodic skin examination is recommended for patients who are at increased risk for skin cancer.

5.4 Major Adverse Cardiovascular Events

Major adverse cardiovascular events (MACE), defined as cardiovascular death, non-fatal myocardial infarction (MI), and non-fatal stroke, were observed in patients treated with LISRAYA during clinical trials. In the dermatomyositis clinical trial, referred to as "Trial DM," inclusive of open-label treatment, adjudicated MACE occurred in 3 patients (1.1 per 100 patient-years) treated with LISRAYA.

In a large, randomized, postmarketing safety study of another JAK inhibitor in patients with RA 50 years of age and older with at least one cardiovascular risk factor, a higher rate of MACE was observed with the JAK inhibitor compared to those treated with TNF blockers. Patients who are current or past smokers are at additional increased risk. LISRAYA is not approved for the treatment of RA.

Consider the benefits and risks for the individual patient prior to initiating or continuing LISRAYA treatment, particularly in patients who are current or past smokers and patients with other cardiovascular risk factors. Inform patients about the symptoms of serious cardiovascular events and the steps to take if they occur. Discontinue LISRAYA in patients who have experienced a myocardial infarction or stroke.

5.5 Thrombosis

Thromboses, including deep venous thrombosis (DVT), pulmonary embolism (PE), and arterial thrombosis, have occurred in patients treated for inflammatory conditions with JAK inhibitors, including LISRAYA. Many of these adverse reactions were serious and some resulted in death. In a large, randomized, postmarketing safety study of another JAK inhibitor in patients with RA 50 years of age and older with at least one cardiovascular risk factor, higher rates of overall thrombosis, DVT, and PE were observed compared to those treated with TNF blockers. LISRAYA is not approved for the treatment of RA.

Avoid LISRAYA in patients who may be at increased risk of thrombosis. If a patient develops symptoms of thrombosis, discontinue LISRAYA, promptly evaluate, and appropriately treat.

5.6 Hypersensitivity Reactions

Hypersensitivity reactions were reported in patients who received LISRAYA. Some events were serious. If a clinically significant hypersensitivity reaction occurs, discontinue LISRAYA, and institute appropriate therapy.

5.7 Gastrointestinal Perforations

Gastrointestinal perforation has been reported in patients treated with JAK inhibitors, including LISRAYA.

Monitor LISRAYA-treated patients who may be at risk for gastrointestinal perforation (e.g., patients with a history of diverticulitis and those taking concomitant drugs that increases the risk for gastrointestinal perforation, including NSAIDs or corticosteroids). If a patient presents with new onset abdominal pain during LISRAYA treatment, promptly evaluate for gastrointestinal perforation.

5.8 Hypoglycemia in Patients with Diabetes

LISRAYA may cause hypoglycemia in patients with diabetes. Hypoglycemia, including severe hypoglycemia, has been reported following initiation of JAK inhibitors in patients with diabetes. During treatment with LISRAYA, consider increased monitoring of blood glucose as clinically indicated in patients with diabetes. Advise patients with diabetes to notify their healthcare provider if they develop signs or symptoms of hypoglycemia.

5.9 Laboratory Abnormalities

Neutropenia

Treatment with LISRAYA was associated with an increased incidence of neutropenia (i.e., ANC less than 1,000 cells/mm³).

Evaluate neutrophil counts at baseline and thereafter according to routine patient management. Avoid LISRAYA initiation, and interrupt LISRAYA treatment in patients with a low neutrophil count (i.e., ANC less than 1,000 cells/mm³) [see Dosage and Administration (2.1)].

Lymphopenia

ALC less than 500 cells/mm³ were reported in patients who received LISRAYA.

Evaluate lymphocyte counts at baseline and thereafter according to routine patient management. Avoid LISRAYA initiation or interrupt LISRAYA treatment in patients with a low lymphocyte count (i.e., less than 500 cells/mm³) [see Dosage and Administration (2.1)].

Anemia

Decreases in hemoglobin levels less than 8 g/dL were reported in LISRAYA-treated patients in clinical trials.

Evaluate hemoglobin at baseline and thereafter according to routine patient management. Avoid LISRAYA initiation or interrupt LISRAYA treatment in patients with a low hemoglobin level (i.e., less than 8 g/dL) [see Dosage and Administration (2.1)].

Lipids

Treatment with LISRAYA was associated with increases in lipid parameters, including total cholesterol, low-density lipoprotein (LDL) cholesterol, and high-density lipoprotein (HDL) cholesterol [see Adverse Reactions (6.1)]. The effect of these lipid parameter elevations on cardiovascular morbidity and mortality has not been determined. Assess lipid parameters approximately 12 weeks after initiation of LISRAYA treatment. Manage patients according to clinical guidelines for hyperlipidemia.

Liver Enzyme Elevations

Treatment with LISRAYA was associated with increased incidence of liver enzyme elevations compared to treatment with placebo. Evaluate liver enzymes, including AST, ALT, and gamma-glutamyl transferase (GGT), at baseline and according to routine patient management thereafter. In patients with dermatomyositis, elevations in AST and ALT may reflect underlying dermatomyositis disease activity, and therefore, it is important to obtain GGT. Promptly evaluate the cause of liver enzyme elevation to identify potential cases of drug-induced liver injury (DILI). If increases in ALT or AST are observed during routine patient management and DILI is suspected, interrupt LISRAYA treatment until DILI is excluded.

5.10 Immunizations

Avoid use of live vaccines immediately prior to and during LISRAYA treatment. Prior to initiating LISRAYA treatment, update immunizations, including prophylactic varicella zoster or herpes zoster vaccinations, according to current immunization guidelines.

5.11 Embryofetal Toxicity

Based on findings in animal studies, LISRAYA may cause fetal harm when administered to a pregnant woman. Administration of brepocitinib to pregnant rats and rabbits at exposures 1.6 and 3 times the exposure at the maximum recommended human dose (MRHD), respectively, during organogenesis resulted in fetal skeletal malformations and increased post-implantation loss. Verify the pregnancy status of females of reproductive potential prior to starting treatment. Advise pregnant women and females of reproductive potential of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with LISRAYA and for 3 days following the last dose [see Use in Specific Populations (8.1, 8.3)].

6. Adverse Reactions

The following clinically significant adverse reactions are described elsewhere in the labeling:

  • Serious Infections [see Warnings and Precautions (5.1)]
  • Mortality [see Warnings and Precautions (5.2)]
  • Malignancy and Lymphoproliferative Disorders [see Warnings and Precautions (5.3)]
  • Major Adverse Cardiovascular Events (MACE) [see Warnings and Precautions (5.4)]
  • Thrombosis [see Warnings and Precautions (5.5)]
  • Hypersensitivity Reactions [see Warnings and Precautions (5.6)]
  • Gastrointestinal Perforations [see Warnings and Precautions (5.7)]
  • Hypoglycemia in Patients with Diabetes [see Warnings and Precautions (5.8)]
  • Laboratory Abnormalities [see Warnings and Precautions (5.9)]

6.1. Clinical Trials Experience

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

The safety of LISRAYA in adult patients with dermatomyositis was evaluated in a 52-week Phase 3, double-blind, placebo-controlled trial (Trial DM) [see Clinical Studies (14)]. In this trial, 241 adult patients were randomized to receive LISRAYA 30 mg once daily (81 patients), brepocitinib 15 mg once daily (unapproved dosage) (81 patients), or placebo (79 patients) once daily for 52 weeks.

Table 2 summarizes the adverse reactions that occurred in ≥5% of LISRAYA-treated patients and ≥2% greater than placebo-treated patients in the 52-week placebo-controlled period. A total of 81 adult patients with dermatomyositis were exposed to LISRAYA during this period.

Table 2. Adverse Reactions Reported in ≥5% of Adult Patients with Dermatomyositis Who Received LISRAYA and ≥2% Greater Than Patients Who Received Placebo (Trial DM):

Adverse Reaction (any
severity)
PlaceboLISRAYA
N=79
(%)
N=81
(%)
Upper Respiratory Tract Infection1115
Headache315
Fatigue312
Urinary Tract Infection511
Nausea411
Bronchitis410
Arthralgia810
Diarrhea59
Back pain69
Fall37
Influenza46
Acne06

Specific Adverse Reactions

Thrombosis

Thromboses were observed in clinical trials of LISRAYA. During the open-label extension period of Trial DM, a case of peripheral arterial thrombosis was reported in a LISRAYA-treated patient.

Overall Infections

During the 52-week treatment period of Trial DM, infections were reported in 45 (57%) placebo-treated patients and 56 (69%) LISRAYA-treated patients. The most commonly reported infections with LISRAYA were upper respiratory tract infections, urinary tract infections, bronchitis, and COVID-19.

  • Serious Infections: During the 52-week treatment period of Trial DM, serious infections were reported in one (1%) placebo-treated patient and 8 (10%) LISRAYA-treated patients. The most common serious infections in LISRAYA-treated patients were pneumonia (two patients) and sepsis (two patients).
  • Viral Reactivation: During the 52-week treatment period of Trial DM, viral reactivations were reported in 4 (5%) LISRAYA-treated patients. All viral reactivations with LISRAYA were herpes zoster.

Laboratory Abnormalities

  • Hepatic Transaminase Elevations: During the 52-week treatment period of Trial DM,
    • Alanine transaminase (ALT) ≥3 x upper limit of normal (ULN) were observed in 7 (9%) placebo-treated patients and 8 (9.9%) LISRAYA-treated patients.
    • Aspartate transaminase (AST) elevations ≥3 x upper limit of normal (ULN) were observed in 3 (3.8%) placebo-treated patients and 4 (4.9%) LISRAYA-treated patients.
    • One case of probable drug-induced liver injury (mixed pattern with marked GGT elevation, normal bilirubin, and low concurrent creatine kinase [CK]) was reported in a patient who received brepocitinib 15 mg. The event resolved following discontinuation of brepocitinib.
  • Lipid Elevations: During the 52-week treatment period of Trial DM, LISRAYA treatment was associated with increases in total cholesterol, HDL cholesterol, and LDL cholesterol. Elevation in LDL and HDL cholesterol peaked by Week 8 and remained stable thereafter. In the 52-week treatment period of Trial DM, changes from baseline in lipid parameters in LISRAYA-treated patients are summarized below:
    • Mean LDL cholesterol increased by 3.8 mg/dL.
    • Mean HDL cholesterol increased by 4.5 mg/dL.
    • Mean LDL/HDL ratio remained stable.

7. Drug Interactions

7.1 Effects of Other Drugs on LISRAYA

Smoking

Smoking causes induction of CYP1A1 and CYP1A2 levels. The exposure of brepocitinib in current smokers is lower than in non-current smokers, and therefore, the effectiveness of LISRAYA may be reduced in smokers [see Clinical Pharmacology (12.3)].

7.2 Effects of LISRAYA on Other Drugs

Substrates of P-gp and BCRP

Brepocitinib is a P-gp and BCRP inhibitor. Concomitant use of LISRAYA with an orally administered P-gp or BCRP substrate may increase the systemic exposure of the P-gp or BCRP substrate [see Clinical Pharmacology (12.3)].

Substrates of OCT2 or MATEs Transporters

Brepocitinib inhibits renal uptake transporters, OCT2 and MATEs (MATE1, MATE2-K) [see Clinical Pharmacology (12.3)]. Concomitant use of LISRAYA with drugs that are substrates of OCT2 and MATEs transporters may increase plasma concentrations of the substrates. Closely monitor patients when LISRAYA is concomitantly used with drugs that are substrates of OCT2 or MATEs transporters for which minimal concentration changes in substrate plasma concentration may lead to serious adverse reactions.

8.1. Pregnancy

Risk Summary

Based on findings in animal studies, LISRAYA may cause fetal harm when administered to a pregnant woman. Available data from LISRAYA use in pregnant women are insufficient to establish a drug associated risk of major birth defects, miscarriage or adverse maternal or fetal outcomes.

In animal reproduction studies, fetal skeletal malformations, and post-implantation loss were observed when brepocitinib was administered to pregnant rats and rabbits during the period of organogenesis at 1.6- and 3-times the exposure at the MRHD, respectively. In a pre- and postnatal study in rats, brepocitinib did not cause adverse effects in maternal animals or offspring at exposures up to 5.5 times the MRHD.

The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

There is a pregnancy safety study for LISRAYA. If LISRAYA is administered during pregnancy, healthcare providers or patients should report LISRAYA exposure to Priovant Therapeutics by calling 1-800-511-9141 or by emailing contactcenter@priovant.com.

Clinical Considerations

Disease-Associated Maternal and/or Embryo/Fetal Risk

Published data suggest that increased disease activity is associated with the risk of developing adverse pregnancy outcomes in women with dermatomyositis. Adverse pregnancy outcomes include preterm delivery (before 37 weeks of gestation), low birth weight (less than 2500 g) infants, and small for gestational age at birth.

Data

Animal Data

In rat embryofetal developmental studies, pregnant rats were administered brepocitinib orally during the period of organogenesis. Skeletal malformations, early and late resorptions, post-implantation loss, and lower mean numbers of viable fetuses were observed with exposures 1.6 times the MRHD. No adverse effects were observed at 1.3 times the MRHD.

In a rabbit embryofetal developmental study, pregnant rabbits were administered brepocitinib orally during the period of organogenesis. Increase of late resorptions, post-implantation loss, lower mean numbers of viable fetuses, and skeletal malformations were observed at 3 times the MRHD. No developmental toxicity was observed in rabbits at 0.8 times the MRHD.

In a pre- and postnatal development study, pregnant rats were administered brepocitinib orally from gestation day 6 through day 21 of lactation. No effects on postnatal developmental, neurobehavioral, or reproductive performance of offspring were noted at 5.5 times the MRHD.

8.2. Lactation

Risk Summary

There are no data on the presence of brepocitinib in human or animal milk, the effects on the breastfed infant, or the effects on milk production. Because of the potential for serious adverse reactions in the breastfed infant, including infections, GI perforation, and malignancy, advise patients that breastfeeding is not recommended during treatment with LISRAYA and for 3 days (approximately 5 half-lives) after the last dose.

8.3. Females and Males of Reproductive Potential

Based on animal studies, brepocitinib may cause fetal harm when administered to pregnant women [see Use in Specific Populations (8.1)].

Pregnancy Testing

Verify the pregnancy status of females of reproductive potential prior to starting treatment with LISRAYA [see Use in Specific Populations (8.1)].

Contraception

Females

Advise females of reproductive potential to use effective contraception during treatment with LISRAYA and for 3 days after the last dose.

8.4. Pediatric Use

The safety and effectiveness of LISRAYA have not been established in pediatric patients.

8.5. Geriatric Use

Of the 81 LISRAYA-treated patients, 14 (17%) were 65 years of age and older. No overall differences in effectiveness of LISRAYA have been observed between patients 65 years of age and older and younger adult patients.

During the 52-week treatment period of Trial DM, overall rates of adverse events were similar between patients 65 years of age and older and younger adult patients; however, older adults experienced higher rates of serious adverse events (SAEs). In the general study population, SAEs occurred in 16% of LISRAYA-treated patients compared to 13% of placebo-treated patients. Among patients 65 years of age and older, SAEs were reported in 2 placebo-treated patients (15%) compared to 3 LISRAYA-treated patients (21%), including one viral reactivation (herpes zoster).

Viral reactivations were reported in 2 LISRAYA-treated patients (15 per 100 patient-years) 65 years of age and older, compared to 2 LISRAYA-treated patients (3 per 100 patient-years) 18 to less than 65 years of age.

8.6. Renal Impairment

The recommended dosage in patients with mild (eGFR: 60 to 89 mL/min) or moderate (eGFR: 30 to 59 mL/min) renal impairment is the same as in patients without renal impairment.

LISRAYA is not recommended in patients with severe renal impairment (eGFR: <30 mL/min).

8.7. Hepatic Impairment

The recommended dosage in patients with mild (Child-Pugh A) or moderate (Child-Pugh B) hepatic impairment is the same as in patients without hepatic impairment.

LISRAYA has not been evaluated in patients with severe hepatic impairment (Child-Pugh C) and therefore, is not recommended in this population.

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