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Newly Approved Drug for Duchenne Muscular Dystrophy Made in NC


-- Alcami photo

A treatment for Duchenne muscular dystrophy approved by the U.S. Food and Drug Administration will be manufactured in Wilmington, N.C.

The drug, the steroid deflazacort, will be marketed as EMFLAZA by Marathon Pharmaceuticals of Northbrook, Ill. The company said the drug will be manufactured in tablet form by Alcami, the Wilmington-based contract development and manufacturing organization that also has a lab in Durham. 

Duchenne, a severe form of muscular dystrophy, is a rare and fatal genetic disease that affects about 15,000 people in the United States, mostly boys and young men.

The FDA approved EMFLAZA for use by patients 5 years old and older. It's a treatment, not a cure.

“We are in a new era in the treatment of Duchenne muscular dystrophy,” Timothy Cunniff, Pharm.D., executive vice president of research and development for Marathon, said in a news release. “For the first time, patients in the U.S. with Duchenne will have widespread access to an FDA-approved medicine that is indicated for all genetic forms of the condition. We are pleased that this development will help patients with this disease stay stronger longer.”

The FDA granted EMFLAZA priority review, which is reserved for investigational medicines that may offer major advances in treatment over existing options. However, soon after the approval, Marathon drew criticism for its plan to price the long-used generic steroid at $89,000 for a year’s supply. Marathon also characterized the U.S. FDA approval as a new drug, though it's actually a new indication in the U.S. for a long-established steroid.

Old drug offers new option for Duchenne families in U.S.

“We are pleased to learn that the FDA has approved EMFLAZA,” said Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy, the largest nonprofit organization in the United States focused solely on Duchenne muscular dystrophy. “While steroids are considered standard of care for Duchenne patients, there has been no steroid specifically approved for Duchenne. The FDA approval of EMFLAZA provides options for our families when making crucial decisions about care with their providers. We hope that this approval gives more families access to this important medication."

Marathon said it conducted 17 preclinical and clinical studies and exclusively licensed two additional clinical studies to support New Drug Application submissions for EMFLAZA to the FDA.

“EMFLAZA is an important new drug with proven benefit in boys with Duchenne muscular dystrophy, increasing muscle strength and physical function, which is important for both patients and caregivers,” said Robert Griggs, M.D., a leading neurologist and an early investigator of deflazacort at the University of Rochester Medical Center. “In my own research and in the clinic, deflazacort keeps boys walking longer. By undertaking the research needed to secure FDA approval of EMFLAZA, we now know more about the drug, its dosing and possible interactions. These are all advances in care for patients with Duchenne.”

Joel Wood, president of the Foundation to Eradicate Duchenne, said the treatment has had a positive effect on his son, James, who has Duchenne.

“Today’s approval makes it possible for all patients living with Duchenne to get access to this new treatment option,” Wood said. “That’s an important step forward. Marathon’s commitment to research – conducting the research needed for FDA approval and its ongoing clinical programs – means continued progress in the treatment of Duchenne.”

Duchenne muscular dystrophy is caused by mutations in the dystrophin gene. Without dystrophin, a structural protein, muscles deteriorate and are unable to work properly. 

The disorder occurs in about one in 5,000 live male births and typically results in the inability to walk by the teen years or earlier, and severe respiratory and cardiac complications that lead to death generally between the late teens and early twenties.

Marathon, a privately held company, develops treatments for rare diseases with a focus on patients who currently have no treatment options. The company’s pipeline of new medicines includes treatments for rare neurological and movement disorders.

Alcami a new brand

Alcami was formed in 2016 by Wilmington-based AAIPharma Services Corp. and Wisconsin-based Cambridge Major Laboratories following their merger in 2013. The rebranded company provides a full range of services to support pharmaceutical and biotechnology clients, from early-stage development and scale up to integrated manufacturing and commercialization.

Over the last 30 years, Alcami and its predecessor companies have supported more than 500 Investigational New Drug filings and over 50 New Drug Applications, Abbreviated New Drug Applications and New Animal Drug Applications.

In addition to its Wilmington and Durham facilities, Alcami has operations in St. Louis, Germantown, Wis., Edison, N.J., Charleston, S.C., and Weert, The Netherlands. The sites collectively employ about 1,000 people.

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