Deflazacort is a Small Molecule owned by PTC Therapeutics, and is involved in 12 clinical trials, of which 11 were completed, and 1 is planned.

Deflazacort is a glucocorticoid receptor agonist. The drug candidate binds to the glucocorticoid receptor and enhances the corticosteroids function. Muscle wasting and loss of walking ability are associated with DMD. Glucocorticoids stabilize and improve muscle strength and walking. By prolonging independent ambulation the drug candidate improves the symptoms of the conditions.

The revenue for Deflazacort is expected to reach a total of $1.5bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Deflazacort NPV Report.

Deflazacort is currently owned by PTC Therapeutics.

Deflazacort Overview

Deflazacort (MP-104, Emflaza) is an oxazoline derivative of prednisolone with anti-inflammatory and immunosuppressive activity. It is formulated as tablets and suspension for oral route of administration. Emflaza is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 5 years of age and older.

Deflazacort was under development for the treatment of limb-girdle muscular dystrophy 2I (LGMD2I) and Duchenne muscular dystrophy (DMD) in patients 2 to 5 years of age.

PTC Therapeutics Overview

PTC Therapeutics discovers, develops, and markets medicines for the treatment of rare disorders. Its marketed products include Evrysdi (risdiplam) for spinal muscular atrophy, Translarna (ataluren) and Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy. The company’s pipeline candidates target the treatment of Huntington’s disease; children with inherited mitochondrial disease; leiomyosarcoma; acute myelogenous leukemia; and aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics works in collaboration with various biotechnology and pharmaceutical companies for advancing its pipeline candidates. The company operates through its subsidiaries in the US and Europe. PTC Therapeutics is headquartered in South Plainfield, New Jersey, the US.

The company reported revenues of (US Dollars) US$538.6 million for the fiscal year ended December 2021 (FY2021), an increase of 41.4% over FY2020. The operating loss of the company was US$374.4 million in FY2021, compared to an operating loss of US$431.8 million in FY2020. The net loss of the company was US$523.9 million in FY2021, compared to a net loss of US$438.2 million in FY2020. The company reported revenues of US$217.1 million for the third quarter ended September 2022, an increase of 31.2% over the previous quarter.

Quick View – Deflazacort

Report Segments
  • Innovator (NME)
Drug Name
  • Deflazacort
Administration Pathway
  • Oral
Therapeutic Areas
  • Genetic Disorders
  • Musculoskeletal Disorders
Key Companies
Highest Development Stage
  • Marketed

GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

To create this model, GlobalData takes into account factors including patent law, known and projected regulatory approval processes, cash flows, potential applicable patients, drug margins, company expenses, and pricing estimates. Combining these data points with GlobalData’s world class analysis creates high value models that companies can use to help in evaluation processes for each drug or company.

The rNPV method integrates the probability of a drug reaching a clinical stage into the cash flow at that time, which provides a more accurate rNPV, as it considers the probability that the drug never makes it through the clinical pathway to commercialization. GlobalData’s rNPV model uses proprietary likelihood of approval (LoA)and phase transition success rate(PTSR) data for the indication in the highest development stage, which can be found on GlobalData’s Pharmaceutical Intelligence Center.