FDA Approves Cranbury’s Generic Version of Deflazacort to Treat Duchenne Muscular Dystrophy

Sharon Hesterlee, PhD

According to a new announcement, the FDA has approved a new generic version of deflazacort (Emflaza; PTC Therapeutics), a treatment indicated for patients 5 years of age and older with Duchenne muscular dystrophy (DMD). The new generic, which allows for greater access and improved cost-effectiveness, is from Cranbury Pharmaceuticals, a wholly-owned subsidiary of Tris Pharma.¹

"We were pleased to see a generic option for deflazacort, which has been available for much longer in Europe at generic prices. As corticosteroids like deflazacort represent standard-of-care for Duchenne, the wider availability of deflazacort will provide options for some families who may have previously had challenges getting the brand name version, Emflaza, approved," Sharon Hesterlee, PhD, executive vice president and chief research officer of the Muscular Dystrophy Association, told NeurologyLive^®.

Deflazacort oral suspension, a corticosteroid indicated for DMD, has been on the market since 2017 under the name Emflaza and manufactured by PTC Therapeutics. It has several noted warnings and precautions, including alterations in endocrine function, immunosuppression and increased risk of infection, alterations in cardiovascular/renal function, gastrointestinal perforation, behavioral and mood disturbances, and effects on bones, among others. It is contraindicated in patients with known hypersensitivity to deflazacort or to any of the inactive ingredients.

"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, founder and chief executive officer at Tris Pharma, said in a statement.¹ "This FDA approval is a significant milestone for the patients, caregivers and physicians who may depend on this medication to treat DMD. Our portfolio of differentiated, high-quality generic medications has always been core to our business at Tris, and I look forward to continuing to expand our generic offerings under our new subsidiary, Cranbury Pharmaceuticals."

READ MORE: Final Readout of FIREFISH Study Showcases Risdiplam’s Pronounced Effects in Spinal Muscular Atrophy

Since its approval, deflazacort has been recommended as a once-daily 0.9 mg/kg tablet administered orally. Dosage of the therapy must be decreased gradually if the drug has been administered for more than a few days. Patients are also to administer live attenuated or live vaccines at least 4 to 6 weeks prior to starting deflazacort. The therapy can be taken with or without food, and patients are prohibited from taking the therapy with grapefruit juice.

Janet Penner

"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," Janet Penner, president at Cranbury, said in a statement.¹ "The launch of deflazacort oral suspension aligns with this commitment, and we are proud to provide this critical therapy to those with DMD. As a tenured member of the Tris team, I am honored to take on the role of president of our new subsidiary and have the opportunity to provide important generic medicines to patients with a range of disorders and diseases."

This was the second approved generic for deflazacort, following Aurobindo Pharma’s approval in early February. Aurobindo, an Indian-based company, gained clearance to market deflazacort tablets in doses of 6 mg, 18 mg, 30 mg, and 36 mg, which was bioequivalent and therapeutically equivalent to the reference listed drug strengths in its original approval.²

Deflazacort was originally approved in patients with DMD aged 5 and older but was later expanded in 2019 to include children age 2 to 5 years. At the time, it made deflazacort the only FDA-approved treatment for patients with DMD age 2 and older, regardless of disease-causing genetic mutation. The original approval was based on data from a phase 3 randomized, double-blind, placebo-controlled study of 196 patients with DMD aged 5 to 15 over a 52-week period that compared treatment with deflazacort with prednisone and placebo.^3,4

REFERENCES
1. Cranbury Pharmaceuticals receives US FDA approval for first generic version of Emflaza oral suspension (deflazacort) for Duchenne muscular dystrophy. News release. June 12, 2024. Accessed June 12, 2024. https://www.trispharma.com/cranbury-pharmaceuticals-receives-u-s-fda-approval-for-first-generic-version-of-emflaza-oral-suspension-deflazacort-for-duchenne-muscular-dystrophy/
2. Aurobindo Pharma receives USFDA approval for deflazacort tablets, 6 mg, 18 mg, 30 mg, and 36 mg. News release. Aurobindo Pharma. February 12, 2024. Accessed June 12, 2024. https://www.aurobindo.com/api/uploads/disclosureofeventsinformation/LetterToSEsPressReleaseUSFDAproductAproval12022024.pdf
3. PTC Therapeutics receives FDA approval for the expansion of the Emflaza® (deflazacort) labeling to include patients 2-5 years of age. News release. PTC Therapeutics. June 7, 2019. Accessed June 12, 2024. www.prnewswire.com/news-releases/ptc-therapeutics-receives-fda-approval-for-the-expansion-of-the-emflaza-deflazacort-labeling-to-include-patients-2-5-years-of-age-300863988.html.
4. Griggs RC, Miller JP, Greenberg CR, et al. Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology. 2016;87(20):2123-2131.