PYQUVIT (deflazacort oral suspension) (Deflazacort Oral)

PYQUVIT (deflazacort) is an FDA-approved oral corticosteroid specifically indicated for Duchenne Muscular Dystrophy (DMD) in pediatric and adult patients. Manufactured by Aucta Pharmaceuticals, Inc., it received orphan drug designation due to the rare and severe nature of DMD. This neurological therapeutic agent helps slow disease progression and preserve muscle function in this devastating genetic disorder.

Manufacturer / TM Owner

Aucta Pharmaceuticals, Inc.

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Indications & Clinical Uses

PYQUVIT is used to treat Duchenne Muscular Dystrophy, a progressive X-linked genetic disorder characterized by severe muscle weakness and degeneration. The drug is indicated for both pediatric and adult DMD patients to help slow the rate of decline in muscle strength and function. Deflazacort, the active ingredient, is a corticosteroid that works by reducing inflammation and modulating immune responses within muscle tissue, thereby slowing muscle degeneration. The target patient population includes males with confirmed DMD diagnosis, typically beginning treatment in early childhood to maximize therapeutic benefit. By preserving muscle function and delaying loss of ambulation, PYQUVIT helps maintain quality of life and independence in DMD patients, addressing a critical unmet medical need in this rare neuromuscular disorder affecting approximately 1 in 3,500 to 5,000 male births.

Side Effects

Common side effects of PYQUVIT include increased appetite, weight gain, mood changes, sleep disturbances, and gastrointestinal upset. Serious adverse events may include immunosuppression leading to increased infection risk, hyperglycemia, hypertension, osteoporosis with fracture risk, and adrenal insufficiency with prolonged use. Patients require regular monitoring including blood glucose levels, blood pressure, bone density assessments, and growth parameters in pediatric patients. Ophthalmologic examinations are recommended to screen for cataracts. Baseline and periodic laboratory assessments including complete blood counts and metabolic panels are essential to detect early complications and guide dose adjustments.

Precautions & Warnings

PYQUVIT is contraindicated in patients with systemic fungal infections or known hypersensitivity to deflazacort. Caution is warranted in patients with active infections, diabetes, hypertension, osteoporosis, or psychiatric disorders. Pregnancy category C; use only if benefits outweigh risks. Pediatric patients require careful monitoring for growth suppression and developmental effects. Patients with hepatic or renal impairment may require dose adjustments. Live vaccines should be avoided during treatment. Abrupt discontinuation is contraindicated; gradual tapering is necessary to prevent adrenal crisis. Patients should be counseled on infection prevention and maintaining adequate calcium and vitamin D intake.

Avoid Interactions

PYQUVIT may interact with CYP3A4 inhibitors and inducers, affecting deflazacort metabolism. Concurrent use with NSAIDs increases gastrointestinal ulceration risk. Live vaccines should be avoided due to immunosuppression. Deflazacort may reduce efficacy of certain antidiabetic agents, requiring glucose monitoring and potential dose adjustments. Concurrent use with other immunosuppressants increases infection risk. Patients should avoid grapefruit juice, which may inhibit metabolism. Inform healthcare providers of all medications, supplements, and herbal products before starting PYQUVIT.

Frequently Asked Questions

PYQUVIT (deflazacort) is used to treat Duchenne Muscular Dystrophy (DMD) in pediatric and adult patients. It helps slow the progression of muscle weakness and degeneration associated with this rare genetic disorder. By preserving muscle function and delaying loss of mobility, PYQUVIT improves quality of life and independence in DMD patients.

PYQUVIT is administered orally as tablets. The dosage is individualized based on patient weight, age, and clinical response, typically ranging from 0.75 to 1.5 mg/kg/day divided into one or two daily doses. Patients should take PYQUVIT consistently as prescribed by their healthcare provider, with or without food.

Common side effects include increased appetite, weight gain, mood changes, insomnia, and gastrointestinal upset. Some patients may experience facial puffiness or acne. Most side effects are manageable with dose adjustment or supportive care, though serious effects like immunosuppression require medical attention.

PYQUVIT is manufactured by Aucta Pharmaceuticals, Inc., a pharmaceutical company specializing in rare disease treatments and orphan drugs.

Yes, PYQUVIT has received FDA orphan drug designation for the treatment of Duchenne Muscular Dystrophy. This designation recognizes DMD as a rare disease affecting fewer than 200,000 individuals in the United States. Orphan drug status provides incentives for development and commercialization of treatments for rare diseases.

Published by

GNH India Pharmaceuticals Limited

Licensed pharmaceutical supplier

Last updated: 17 Jun 2026

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