Pyquvi

Deflazacort · Suspension · Oral

Uses

1 INDICATIONS AND USAGE PYQUVI is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older. Additional pediatric use information is approved for PTC Therapeutics, Inc.'s Emflaza ® (deflazacort) oral suspension. However, due to PTC Therapeutics, Inc.'s marketing exclusivity rights, this drug product is not labeled with that information. PYQUVI is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older ( 1 )

Dosage and administration

The recommended once-daily dosage is approximately 0.9 mg/kg/day administered orally ( 2.2 ) Discontinue gradually when administered for more than a few days ( 2.3 ) 2.1 Assessments Prior to First Dose of PYQUVI Administer all immunizations according to immunization guidelines prior to starting PYQUVI. Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting PYQUVI [see Warnings and Precautions ( 5.8 )] . 2.2 Dosing Information The recommended oral dosage of PYQUVI is approximately 0.9 mg/kg/day once daily. If the oral suspension is used, round up to the nearest tenth of a milliliter (mL). 2.3 Discontinuation Dosage of PYQUVI must be decreased gradually if the drug has been administered for more than a few days [see Warnings and Precautions ( 5.1 )] . 2.4 Important Preparation and Administration Instructions PYQUVI Oral Suspension can be taken with or without food. Do not administer PYQUVI with grapefruit juice [see Drug Interactions ( 7.1 )] Shake PYQUVI Oral Suspension well before administration. Use only the oral dispenser provided with the product. After withdrawing the appropriate dose into the oral dispenser, slowly add the PYQUVI Oral Suspension into 3 to 4 ounces of juice (except grapefruit juice) or milk and mix well. The dose should then be administered immediately. Discard any unused PYQUVI Oral Suspension remaining after 1 month of first opening the bottle. 2.5 Dosage Modification for Use with CYP3A4 Inhibitors and Inducers CYP3A4 Inhibitors Give one third of the recommended dosage when PYQUVI is administered with moderate or strong CYP3A4 inhibitors. For example, a 36 mg per day dose would be reduced to a 12 mg per day dose when used with moderate or strong CYP3A4 inhibitors [see Drug Interactions ( 7.1 ) and Clinical Pharmacology ( 12.3 )] . CYP3A4 Inducers Avoid use with moderate or strong CYP3A4 inducers with PYQUVI [see Drug Interactions ( 7.1 ) and Clinical Pharmacology ( 12.3 )] .

Dosage forms and strengths

Oral Suspension 22.75 mg/mL: White to off-white color suspension Oral Suspension: 22.75 mg/mL ( 3 )

Contraindications

4 CONTRAINDICATIONS PYQUVI is contraindicated in patients with known hypersensitivity to deflazacort or to any of the inactive ingredients. Instances of hypersensitivity, including anaphylaxis, have occurred in patients receiving corticosteroid therapy [see Warnings and Precautions ( 5.15 ) and Adverse Reactions ( 6.2 )] . Hypersensitivity to deflazacort or any of the inactive ingredients in PYQUVI ( 4 )

Warnings and precautions

Alterations in Endocrine Function: Hypothalamic-pituitary-adrenal axis suppression, Cushing’s syndrome, and hyperglycemia can occur; Monitor patients for these conditions with chronic use of PYQUVI ( 2.3 , 5.1 ) Immunosuppression and Increased Risk of Infection: Increased risk of new, exacerbation, dissemination, or reactivation of latent infections, which can be severe and at times fatal; Signs and symptoms of infection may be masked ( 5.2 ) Alterations in Cardiovascular/Renal Function: Monitor for elevated blood pressure and sodium, and for decreased potassium levels ( 5.3 ) Gastrointestinal Perforation: Increased risk in patients with certain GI disorders; Signs and symptoms may be masked ( 5.4 ) Behavioral and Mood Disturbances: May include euphoria, insomnia, mood swings, personality changes, severe depression, and psychosis ( 5.5 ) Effects on Bones: Monitor for decreases in bone mineral density with chronic use of PYQUVI ( 5.6 ) Ophthalmic Effects: May include cataracts, infections, and glaucoma; Monitor intraocular pressure if PYQUVI is continued for more than 6 weeks ( 5.7 ) Vaccination: Do not administer live or live attenuated vaccines to patients receiving immunosuppressive doses of corticosteroids. Administer live-attenuated or live vaccines at least 4 to 6 weeks prior to starting PYQUVI ( 5.8 ) Serious Skin Rashes: Discontinue at the first sign of rash, unless the rash is clearly not drug related ( 5.9 ) 5.1 Alterations in Endocrine Function Corticosteroids, such as PYQUVI, can cause serious and life-threatening alterations in endocrine function, especially with chronic use. Monitor patients receiving PYQUVI for Cushing’s syndrome, hyperglycemia, and adrenal insufficiency after PYQUVI withdrawal. In addition, patients with hypopituitarism, primary adrenal insufficiency or congenital adrenal hyperplasia, altered thyroid function, or pheochromocytoma may be at increased risk for adverse endocrine events. Risk of Adrenal Insufficiency Following Corticosteroid Withdrawal Corticosteroids produce reversible hypothalamic-pituitary-adrenal (HPA) axis suppression, with the potential for the development of secondary adrenal insufficiency after withdrawal of corticosteroid treatment. Acute adrenal insufficiency can occur if corticosteroids are withdrawn abruptly, and can be fatal. The degree and duration of adrenocortical insufficiency produced is variable among patients and depends on the dose, frequency, and duration of corticosteroid therapy. The risk is reduced by gradually tapering the corticosteroid dose when withdrawing treatment. This insufficiency may persist, however, for months after discontinuation of prolonged therapy; therefore, in any situation of stress occurring during that period of discontinuation, corticosteroid therapy should be reinstituted. For patients already taking corticosteroids during times of stress, the dosage may need to be increased. A steroid “withdrawal syndrome”, seemingly unrelated to adrenocortical insufficiency, may also occur following abrupt discontinuance of corticosteroids. This syndrome includes symptoms such as anorexia, nausea, vomiting, lethargy, headache, fever, joint pain, desquamation, myalgia, and/or weight loss. These effects are thought to be due to the sudden change in corticosteroid concentration rather than to low corticosteroid levels. Cushing’s Syndrome Cushing’s syndrome (hypercortisolism) occurs with prolonged exposure to exogenous corticosteroids, including PYQUVI. Symptoms include hypertension, truncal obesity and thinning of the limbs, purple striae, facial rounding, facial plethora, muscle weakness, easy and frequent bruising with thin fragile skin, posterior neck fat deposition, osteopenia, acne, amenorrhea, hirsutism and psychiatric abnormalities. Hyperglycemia Corticosteroids can increase blood glucose, worsen pre-existing diabetes, predispose those on long-term therapy to diabetes mellitus, and may reduce the effect of anti-diabetic drugs. Monitor blood glucose at regular intervals. For patients with hyperglycemia, anti-diabetic treatment should be initiated or adjusted accordingly. Considerations for Use in Patients with Altered Thyroid Function Metabolic clearance of corticosteroids is decreased in hypothyroid patients and increased in hyperthyroid patients. Changes in thyroid status of the patient may necessitate a dose adjustment of the corticosteroid. When concomitant administration of corticosteroids and levothyroxine is required, administration of corticosteroid should precede the initiation of levothyroxine therapy to reduce the risk of adrenal crisis. Pheochromocytoma Crisis There have been reports of pheochromocytoma crisis, which can be fatal, after administration of systemic corticosteroids. In patients with suspected or identified pheochromocytoma, consider the risk of pheochromocytoma crisis prior to administering corticosteroids. 5.2 Immunosuppression and Increased Risk of Infection Corticosteroids, including PYQUVI, suppress the immune system and increase the risk of infection with any pathogen, including viral, bacterial, fungal, protozoan, or helminthic pathogens. Corticosteroids can:
• Reduce resistance to new infections
• Exacerbate existing infections
• Increase the risk of disseminated infections
• Increase the risk of reactivation or exacerbation of latent infections
• Mask some signs of infections Corticosteroid-associated infections can be mild but can be severe, and at times fatal. The rate of infectious complications increases with increasing corticosteroid dosages. Monitor for the development of infection and consider PYQUVI withdrawal of corticosteroids or dosage reduction as needed. Tuberculosis If PYQUVI is used to treat a condition in patients with latent tuberculosis or tuberculin reactivity, reactivation of tuberculosis may occur. Closely monitor such patients for reactivation.

Side effects

The following serious adverse reactions are discussed in more detail in other sections: Alterations in Endocrine Function [see Warnings and Precautions ( 5.1 )] Immunosuppression and Increased Risk of Infection [see Warnings and Precautions ( 5.2 )] Alterations in Cardiovascular/Renal Function [see Warnings and Precautions ( 5.3 )] Gastrointestinal Perforation [see Warnings and Precautions ( 5.4 )] Behavioral and Mood Disturbances [see Warnings and Precautions ( 5.5 )] Effects on Bones [see Warnings and Precautions ( 5.6 )] Ophthalmic Effects [see Warnings and Precautions ( 5.7 )] Immunizations [see Warnings and Precautions ( 5.8 )] Serious Skin Rashes [see Warnings and Precautions ( 5.9 )] Effects on Growth and Development [see Warnings and Precautions ( 5.10 )] Myopathy [see Warnings and Precautions ( 5.11 )] Kaposi’s Sarcoma [see Warnings and Precautions ( 5.12 )] Risk of Serious Adverse Reactions in Infants because of Benzyl Alcohol Preservative [see Warnings and Precautions ( 5.13 )] Thromboembolic Events [see Warnings and Precautions ( 5.14 )] Anaphylaxis [see Warnings and Precautions ( 5.15 )] The most common adverse reactions (≥ 10% for PYQUVI and greater than placebo) are Cushingoid appearance, weight increased, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, central obesity, and nasopharyngitis ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Aucta Pharmaceuticals, Inc. at 1-800-655-9902 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 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. In Study 1 [see Clinical Studies ( 14 )] , the adverse reactions that were associated with deflazacort treatment discontinuation, in decreasing order of frequency, were weight increased, obesity, cataract, and sleep disorder. Most Common Adverse Reactions in Clinical Studies Table 1 lists the adverse reactions that occurred in ≥ 5% of patients in the 0.9 mg/kg/day deflazacort-treated group and that occurred more frequently than in placebo patients in Study 1, which included patients with DMD between the ages of 5 and 15 years. Table 1: Adverse Reactions that Occurred in ≥ 5% of Deflazacort-Treated Patients and Occurred More Frequently than in Placebo Patients with DMD (Study 1) 1 At 12 weeks placebo patients were re-randomized to receive either deflazacort or an active comparator. Adverse Reaction Deflazacort 0.9 mg/kg/d (N=51) % at 12 weeks Placebo (N=50) % at 12 weeks 1 Cushingoid appearance 33 12 Weight increased 20 6 Increased appetite 14 2 Upper respiratory tract infection 12 10 Cough 12 6 Pollakiuria 12 2 Nasopharyngitis 10 6 Hirsutism 10 2 Central obesity 10 4 Erythema 8 6 Irritability 8 4 Rhinorrhea 8 0 Abdominal discomfort 6 2 Common adverse reactions (≥ 5% of deflazacort-treated patients) that occurred over 52 weeks of exposure to deflazacort 0.9 mg/kg/day in Study 1 and at a higher rate than deflazacort 0.9 mg/kg/day in the 12-week placebo-controlled phase of the trial include Cushingoid appearance (60%), hirsutism (35%), weight increased (28%), erythema (28%), central obesity (25%), abdominal pain/abdominal pain upper (18% combined), pollakiuria (15%), constipation (10%), irritability (10%), abnormal behavior (9%), pyrexia (9%), back pain (7%), rash (7%), contusion (6%), nausea (6%), psychomotor hyperactivity (6%), epistaxis (6%), and skin striae (6%). Study 1 also evaluated a higher dosage of deflazacort (1.2 mg/kg/day). Compared with the 0.9 mg/kg/day dosage, deflazacort 1.2 mg/kg/day over 52 weeks was associated with a higher incidence of certain adverse reactions, including Cushingoid appearance (69%), erythema (49%), hirsutism (37%), headache (34%), weight increased (32%), constipation (15%), abdominal pain upper (14%), skin striae (11%), acne (11%), and abdominal discomfort (8%). As there was no additional benefit with the 1.2 mg/kg/day dose of deflazacort, use of deflazacort oral suspension1.2 mg/kg/day is not recommended for the treatment of DMD [see Dosage and Administration ( 2.2 )] . In an additional clinical study of two years duration with extended follow-up (Study 2), many of the same adverse reactions were observed. In addition, musculoskeletal events associated with long-term steroid use were also observed, including muscle weakness, tendon disorder, and osteopenia. Less Common Adverse Reactions Observed in Clinical Studies Other adverse reactions (≥ 1% frequency in any deflazacort treatment group and greater than placebo) that were observed during the 12-week placebo-controlled phase of Study 1 are shown below.

Drug interactions

Moderate or strong CYP3A4 inhibitors: Give one third of the recommended dosage of PYQUVI ( 7.1 ) Avoid use of moderate or strong CYP3A4 inducers with PYQUVI, as they may reduce efficacy ( 7.1 ) See 17 for PATIENT COUNSELING INFORMATION and FDA-approved patient labeling. Additional pediatric use information is approved for PTC Therapeutics, Inc.'s Emflaza ® (deflazacort) oral suspension. However, due to PTC Therapeutics, Inc.'s marketing exclusivity rights, this drug product is not labeled with that information. 7.1 CYP3A4 Inhibitors and Inducers Moderate or Strong CYP3A4 Inhibitors The active metabolite of deflazacort, 21-desDFZ, is a substrate of CYP3A4 [see Clinical Pharmacology ( 12.3 )] . Co-administration of deflazacort with clarithromycin, a strong CYP3A4 inhibitor, increased total exposure to 21-desDFZ by about 3-fold. Therefore, give one third the recommended dosage of PYQUVI when moderate or strong CYP3A4 inhibitors (e.g., clarithromycin, fluconazole, diltiazem, verapamil, grapefruit juice) are used concomitantly with PYQUVI [see Dosage and Administration ( 2.5 ) and Clinical Pharmacology ( 12.3 )] . Moderate or Strong CYP3A4 Inducers Co-administration of deflazacort with rifampin, a strong CYP3A4 inducer, significantly decreased the exposure of 21-desDFZ. Avoid concomitant use of strong (e.g., efavirenz) or moderate (e.g., carbamazepine, phenytoin) CYP3A4 inducers with PYQUVI [see Dosage and Administration ( 2.5 ) and Clinical Pharmacology ( 12.3 )] . 7.2 Neuromuscular Blockers Patients receiving corticosteroids, including PYQUVI, and concomitant therapy with neuromuscular blocking drugs (e.g., pancuronium) may be at increased risk of developing an acute myopathy [see Warnings and Precautions ( 5.11 )] .

Use in specific populations

8.1 Pregnancy Risk Summary Corticosteroids should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Infants born to mothers who have received substantial doses of corticosteroids during pregnancy should be carefully observed for signs of hypoadrenalism. There are no adequate and well-controlled studies with PYQUVI in pregnant women to inform drug-associated risks. Corticosteroids, including PYQUVI, readily cross the placenta. Adverse developmental outcomes, including orofacial clefts (cleft lip, with or without cleft palate) and intrauterine growth restriction, and decreased birth weight, have been reported with maternal use of corticosteroids, including PYQUVI, during pregnancy. Some epidemiologic studies report an increased risk of orofacial clefts from about 1 per 1000 infants to 3 to 5 per 1000 infants; however, a risk for orofacial clefts has not been observed in all studies. Intrauterine growth restriction and decreased birth weight appear to be dose-related; however, the underlying maternal condition may also contribute to these risks (see Data ) . The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. Animal reproduction studies have not been conducted with deflazacort. Animal reproduction studies conducted with other corticosteroids in pregnant mice, rats, hamsters, and rabbits using clinically relevant doses have shown an increased incidence of cleft palate. An increase in embryofetal death, intrauterine growth retardation, and constriction of the ductus arteriosus were observed in some animal species. Data Human Data Multiple cohort and case-controlled studies in humans suggest that maternal corticosteroid use during the first trimester increases the rate of cleft lip, with or without cleft palate, from about 1/1000 infants to 3-5/1000 infants. Two prospective case-controlled studies showed decreased birth weight in infants exposed to maternal corticosteroids in utero. 8.2 Lactation Risk Summary Systemically administered corticosteroids appear in human milk and could suppress growth, interfere with endogenous corticosteroid production, or cause other untoward effects. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for PYQUVI and any potential adverse effects on the breastfed infant from PYQUVI. There are no data on the effects on milk production. 8.4 Pediatric Use The safety and effectiveness of PYQUVI for the treatment of DMD have been established in patients 5 years of age and older. Use of PYQUVI in pediatric patients is supported by a multicenter, randomized, double-blind, placebo- and active-controlled study in 196 males 5 to 15 years of age [see Clinical Studies ( 14 )] . Safety and effectiveness in pediatric patients below the age of 2 years have not been established. PYQUVI Oral Suspension contains benzyl alcohol and is not approved for use in pediatric patients less than 2 years of age. Serious adverse reactions including fatal reactions and “gasping syndrome” occurred in premature neonates and low birth weight infants in the neonatal intensive care unit who received drugs containing benzyl alcohol as a preservative. In these cases, benzyl alcohol dosages of 99 to 234 mg/kg/day produced high levels of benzyl alcohol and its metabolites in the blood and urine (blood levels of benzyl alcohol were 0.61 to 1.378 mmol/L). Additional adverse reactions included gradual neurological deterioration, seizures, intracranial hemorrhage, hematologic abnormalities, skin breakdown, hepatic and renal failure, hypotension, bradycardia, and cardiovascular collapse. Preterm, low-birth weight infants may be more likely to develop these reactions because they may be less able to metabolize benzyl alcohol. When prescribing PYQUVI Oral Suspension consider the combined daily metabolic load of benzyl alcohol from all sources including PYQUVI Oral Suspension (PYQUVI Oral Suspension contains 10.45 mg of benzyl alcohol per mL) and other drugs containing benzyl alcohol. The minimum amount of benzyl alcohol at which serious adverse reactions may occur is not known. At the recommended dose of 0.9 mg/kg/day of PYQUVI Oral Suspension, patients would receive approximately 0.4 mg/kg/day of benzyl alcohol [see Warnings and Precautions ( 5.13 )] . Juvenile Animal Toxicity Data Oral administration of deflazacort (0, 0.1, 0.3, and 1.0 mg/kg/day) to juvenile rats from postnatal day (PND) 21 to 80 resulted in decreased body weight gain and adverse effects on skeletal development (including decreased cellularity of growth plate and altered bone distribution) and on lymphoid tissue (decreased cellularity). A no-effect dose was not identified. In addition, neurological and neurobehavioral abnormalities were observed at the mid and/or high dose. Plasma 21-desDFZ exposure (AUC) at the lowest dose tested (0.1 mg/kg/day) was lower than that in humans at the recommended human dose of PYQUVI (0.9 mg/kg/day). Additional pediatric use information is approved for PTC Therapeutics, Inc.'s Emflaza ® (deflazacort) oral suspension. However, due to PTC Therapeutics, Inc.'s marketing exclusivity rights, this drug product is not labeled with that information. 8.5 Geriatric Use DMD is largely a disease of children and young adults; therefore, there is no geriatric experience with PYQUVI. 8.6 Renal Impairment No dose adjustment is required in patients with mild, moderate or severe renal impairment [see Clinical Pharmacology ( 12.3 )] . 8.7 Hepatic Impairment No dose adjustment is required in patients with mild or moderate hepatic impairment [see Clinical Pharmacology ( 12.3 )] .

Pregnancy

Pediatric use

8.4 Pediatric Use The safety and effectiveness of PYQUVI for the treatment of DMD have been established in patients 5 years of age and older. Use of PYQUVI in pediatric patients is supported by a multicenter, randomized, double-blind, placebo- and active-controlled study in 196 males 5 to 15 years of age [see Clinical Studies ( 14 )] . Safety and effectiveness in pediatric patients below the age of 2 years have not been established. PYQUVI Oral Suspension contains benzyl alcohol and is not approved for use in pediatric patients less than 2 years of age. Serious adverse reactions including fatal reactions and “gasping syndrome” occurred in premature neonates and low birth weight infants in the neonatal intensive care unit who received drugs containing benzyl alcohol as a preservative. In these cases, benzyl alcohol dosages of 99 to 234 mg/kg/day produced high levels of benzyl alcohol and its metabolites in the blood and urine (blood levels of benzyl alcohol were 0.61 to 1.378 mmol/L). Additional adverse reactions included gradual neurological deterioration, seizures, intracranial hemorrhage, hematologic abnormalities, skin breakdown, hepatic and renal failure, hypotension, bradycardia, and cardiovascular collapse. Preterm, low-birth weight infants may be more likely to develop these reactions because they may be less able to metabolize benzyl alcohol. When prescribing PYQUVI Oral Suspension consider the combined daily metabolic load of benzyl alcohol from all sources including PYQUVI Oral Suspension (PYQUVI Oral Suspension contains 10.45 mg of benzyl alcohol per mL) and other drugs containing benzyl alcohol. The minimum amount of benzyl alcohol at which serious adverse reactions may occur is not known. At the recommended dose of 0.9 mg/kg/day of PYQUVI Oral Suspension, patients would receive approximately 0.4 mg/kg/day of benzyl alcohol [see Warnings and Precautions ( 5.13 )] . Juvenile Animal Toxicity Data Oral administration of deflazacort (0, 0.1, 0.3, and 1.0 mg/kg/day) to juvenile rats from postnatal day (PND) 21 to 80 resulted in decreased body weight gain and adverse effects on skeletal development (including decreased cellularity of growth plate and altered bone distribution) and on lymphoid tissue (decreased cellularity). A no-effect dose was not identified. In addition, neurological and neurobehavioral abnormalities were observed at the mid and/or high dose. Plasma 21-desDFZ exposure (AUC) at the lowest dose tested (0.1 mg/kg/day) was lower than that in humans at the recommended human dose of PYQUVI (0.9 mg/kg/day). Additional pediatric use information is approved for PTC Therapeutics, Inc.'s Emflaza ® (deflazacort) oral suspension. However, due to PTC Therapeutics, Inc.'s marketing exclusivity rights, this drug product is not labeled with that information.

Geriatric use

8.5 Geriatric Use DMD is largely a disease of children and young adults; therefore, there is no geriatric experience with PYQUVI.

Overdosage

Treatment of acute overdosage is by immediate gastric lavage or emesis followed by supportive and symptomatic therapy. For chronic overdosage in the face of severe disease requiring continuous steroid therapy, the dosage of PYQUVI may be reduced temporarily, or alternate day treatment may be introduced.

Description

The active ingredient in PYQUVI is deflazacort (a corticosteroid). Corticosteroids are adrenocortical steroids, both naturally occurring and synthetic. The molecular formula for deflazacort is C 25 H 31 NO 6 . The chemical name for deflazacort is 16α,17-isoxazole-11β,21-dihydroxypregna-1,4-diene-3,20-dione 21acetate, and the structure is: Deflazacort is a white or almost white crystalline powder and has a molecular weight of 441.52. Deflazacort is soluble in dichloromethane and chloroform, slightly soluble in ethanol and ethyl acetate, almost insoluble in water. PYQUVI for oral administration is available as an immediate-release oral suspension in a strength of 22.75 mg/mL. The oral suspension contains deflazacort and the following inactive ingredients: Benzyl Alcohol, Carboxymethylcellulose Sodium, Colloidal silicon dioxide, Glacial Acetic Acid, Noncrystallizing Sorbitol Solution, Magnesium Aluminum Silicate, Polysorbate 80, Purified water. structure

Mechanism of action

12.1 Mechanism of Action Deflazacort is a corticosteroid prodrug, whose active metabolite, 21-desDFZ, acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. The precise mechanism by which deflazacort exerts its therapeutic effects in patients with DMD is unknown.

How supplied

16.1 How Supplied PYQUVI Oral Suspension 22.75 mg/mL is a white to off-white suspension. Supplied as 13 mL in a 30 mL bottle packaged with one bottle adapter and two 1 mL oral dispensers. NDC 73289-0089-1 16.2 Storage and Handling Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C to 30°C (59°F to 86°F). [See USP Controlled Room Temperature.] Discard any unused PYQUVI Oral Suspension remaining after 1 month of first opening the bottle. 16.1 How Supplied PYQUVI Oral Suspension 22.75 mg/mL is a white to off-white suspension. Supplied as 13 mL in a 30 mL bottle packaged with one bottle adapter and two 1 mL oral dispensers. NDC 73289-0089-1

Storage

16.2 Storage and Handling Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C to 30°C (59°F to 86°F). [See USP Controlled Room Temperature.] Discard any unused PYQUVI Oral Suspension remaining after 1 month of first opening the bottle.

Patient information

Advise the patients and/or caregivers to read the FDA-approved patient labeling if PYQUVI Oral Suspension is prescribed (Instructions for Use). Administration Warn patients and/or caregivers to not stop taking PYQUVI Oral Suspension abruptly or without first checking with their healthcare providers as there may be a need for gradual dose reduction to decrease the risk of adrenal insufficiency [see Dosage and Administration ( 2.3 ) and Warnings and Precautions ( 5.1 )] . PYQUVI Oral Suspension may be taken with or without food. Do not take PYQUVI Oral Suspension with grapefruit juice. PYQUVI Oral Suspension must be shaken well prior to measuring out each dose with the enclosed oral dispenser. The PYQUVI Oral Suspension dose may be placed in 3-4 ounces of juice (except grapefruit juice) or milk, mixed thoroughly, and immediately administered. Discard any unused PYQUVI Oral Suspension remaining after 1 month of first opening the bottle. Increased Risk of Infection Tell patients and/or caregivers to inform their healthcare provider if the patient has had recent or ongoing infections or if they have recently received a vaccine. Medical advice should be sought immediately if the patient develops fever or other signs of infection. Patients and/or caregivers should be made aware that some infections can potentially be severe and fatal. Warn patients who are on corticosteroids to avoid exposure to chickenpox or measles and to alert their healthcare provider immediately if they are exposed [see Warnings and Precautions ( 5.2 )] . Alterations in Cardiovascular/Renal Function Inform patients and/or caregivers that PYQUVI Oral Suspension can cause an increase in blood pressure and water retention. If this occurs, dietary salt restriction and potassium supplementation may be needed [see Warnings and Precautions ( 5.3 )] . Behavioral and Mood Disturbances Advise patients and/or caregivers about the potential for severe behavioral and mood changes with PYQUVI Oral Suspension and encourage them to seek medical attention if psychiatric symptoms develop [see Warnings and Precautions ( 5.5 )] . Decreases in Bone Mineral Density Advise patients and/or caregivers about the risk of osteoporosis with prolonged use of PYQUVI Oral Suspension, which can predispose the patient to vertebral and long bone fractures [see Warnings and Precautions ( 5.6 )] . Ophthalmic Effects Inform patients and/or caregivers that PYQUVI Oral Suspension may cause cataracts or glaucoma and advise monitoring if corticosteroid therapy is continued for more than 6 weeks [see Warnings and Precautions ( 5.7 )] . Vaccination Advise patients and/or caregivers to bring immunizations up-to-date according to immunization guidelines prior to starting therapy with PYQUVI Oral Suspension. Live-attenuated or live vaccines should be administered at least 4 to 6 weeks prior to starting PYQUVI Oral Suspension. Inform patients and/or caregivers that they may receive concurrent vaccinations with use of PYQUVI Oral Suspension, except for live-attenuated or live vaccines. [see Warnings and Precautions ( 5.8 )] . Serious Skin Rashes Instruct patients and/or caregivers to seek medical attention at the first sign of a rash [see Warnings and Precautions ( 5.9 )] . Drug Interactions Certain medications can cause an interaction with PYQUVI Oral Suspension. Advise patients and/or caregivers to inform their healthcare provider of all the medicines the patient is taking, including over-the-counter medicines (such as insulin, aspirin or other NSAIDS), dietary supplements, and herbal products. Inform patients and/or caregivers that alternate therapy, dosage adjustment, and/or special test(s) may be needed during the treatment. Manufactured by: Aucta Pharmaceuticals, Inc. Piscataway, NJ 08854 LB5122-01 02/2025

Label text from the FDA structured product label by Aucta Pharmaceuticals, Inc. (revised Jul 9, 2025). Long sections are shortened; the complete label is on DailyMed.

Active ingredients

Deflazacort in 1 product

Pyquvi NDC products (1)

NDC	Strength & form	Labeler	Type
73289-0089	Deflazacort 22.75 mg/mL Suspension	Aucta Pharmaceuticals, Inc.	ANDA

Frequently asked questions

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Who makes Pyquvi?

Pyquvi is listed by 1 labeler in the FDA NDC directory, including Aucta Pharmaceuticals, Inc..