FDA approves first generic of Emflaza oral suspension for DMD

Deflazacort oral suspension approved for patients 5 and older

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by Andrea Lobo |

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The U.S. Food and Drug Administration (FDA) has approved the first generic version of Emflaza (deflazacort) oral suspension for the treatment of people with Duchenne muscular dystrophy (DMD).

Called deflazacort oral suspension, the medication was approved for DMD patients 5 and older, a slightly older population than the original medication, which is approved for patients as young as 2.

The new generic medication, developed by Cranbury Pharmaceuticals, a wholly owned subsidiary of Tris Pharma, is already available to patients in the U.S.

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Generics usually sold at lower prices

Because generics are therapeutically equivalent to the brand-name medication but usually sold at lower prices, the approval may make deflazacort oral suspension more easily available to DMD patients in the country.

“Duchenne muscular dystrophy is a devastating rare disease, and with limited treatment options available there is a critical need for the greater accessibility that a generic therapy can bring,” Ketan Mehta, Tris Pharma’s founder and CEO, said in a press release.

“This FDA approval is a significant milestone for the patients, caregivers and physicians who may depend on this medication to treat DMD,” Mehta added.

DMD is caused by mutations in the DMD gene, which provides instructions for making a protein called dystrophin that acts like a “shock absorber” in muscle cells, helping protect muscles from damage during movements. The loss of functional dystrophin causes muscle wasting that worsens over time.

Emflaza and the generic medication belong to the class of corticosteroids, which are commonly prescribed to DMD patients to reduce inflammation, to help reduce muscle damage and preserve muscle strength and function.

“Tris Pharma founded Cranbury as part of the company’s continued commitment to develop high-quality medicines and make them accessible to individuals who need them,” said Janet Penner, Cranbury’s president. “The launch of deflazacort oral suspension aligns with this commitment, and we are proud to provide this critical therapy to those with DMD.”

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Emflaza initially approved in 2017 for DMD

Emflaza is available as tablets or as an oral suspension. It was initially approved by the FDA in 2017 for DMD patients 5 and older, becoming the first corticosteroid therapy approved for DMD. The indication was expanded in 2019 to include patients 2 and older.

The initial approval was based on data from a Phase 3 trial that compared Emflaza against the standard corticosteroid prednisone, given once daily for one year to boys with DMD, ages 5 to 15. Results showed the treatment was similar to prednisone and significantly better than a placebo at improving muscle strength and function for up to one year.

Cranbury’s generic version of deflazacort oral suspension was approved based on an abbreviated new drug application. This is the kind of application that’s normally used for generics, which generally does not require preclinical or clinical data demonstrating the drug’s safety and efficacy, but must scientifically demonstrate the generic medication performs in the same manner as the original product.

This was the second approved generic for deflazacort, following Aurobindo Pharma’s deflazacort oral tablets, approved in February for DMD patients 5 and older.