Marathon Pharmaceuticals, a U.S. research-based biopharmaceutical company focused developing of new treatments for rare diseases with manufacturing facilities in North Carolina, said the U.S. Food and Drug Administration (FDA) has granted approval of EMFLAZA (deflazacort) for the treatment of Duchenne muscular dystrophy in patients 5 years and older.

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

The company said the drug will be manufactured in tablet form by Alcami, a Wilmington-based contract development and manufacturing organization that also has a lab in Durham.

“We are in a new era in the treatment of Duchenne muscular dystrophy. 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,” said Timothy M. Cunniff, Pharm.D., executive vice president, Research & Development, Marathon Pharmaceuticals.

“This is only the first of our products to treat Duchenne muscular dystrophy and we are committed to serving this community of patients, researchers and advocates until a cure is found.”

The FDA granted EMFLAZA priority review, which is reserved for investigational medicines that may offer major advances in treatment over existing options. EMFLAZA is a new chemical entity in the United States that has never before been approved for the treatment of Duchenne muscular dystrophy anywhere in the world.

Alcami rebranded

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.

Barry Teator, NC Biotech writer, contributed to this story.