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    Muscular dystrophy drug launch halts after pricing concerns

    Marathon Pharmaceuticals is temporarily pausing the launch of its drug to treat a rare form of muscular dystrophy (MD) after concerns over its $89,000 price tag.

    FDA recently approved deflazacort (Emflaza) to treat Duchenne muscular dystrophy in patients aged 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.

    However, patients were outraged after finding out the price, which is 50 to 70 times higher than the price of delfazacort in Canada and Europe, according to Fox News. Senator Bernie Sanders (I-Vt.) and Representative Elijah Cummings (D-Md.) warned they would launch an investigation over hearing about the drug's  high cost in the United States.

    Related: FDA approves first drug for Duchenne muscular dystrophy

    "We are pausing our commercialization efforts in order to meet with Duchenne community leaders and explain our commercialization plans, review their concerns, discuss all options, and move forward with commercialization..." wrote Jeff Aronin, chairman and CEO of Marathon Pharmaceuticals in an open letter to patients.

    Emflaza is the second approval for the disease in a few months, after FDA granted accelerated approval of eteplirsen injection (Exondys 51, Sarepta Therapeutics) last fall.

    “We are in a new era in the treatment of Duchenne muscular dystrophy. For the first time, patients in the US with Duchenne will have widespread access to an FDA approved medicine that is indicated for all genetic forms of the condition,” said Timothy M. Cunniff, PharmD, executive vice president of research & development for Marathon Pharmaceuticals, in a company statement.

    FDA granted Emflaza priority review, which is reserved for investigational medicines that may offer major advances in treatment over existing options. “This is the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use in the US,” FDA said in a statement.

    Related: First therapy approved for rare blood cancer

    “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,” said Pat Furlong, founding President and CEO of Parent Project Muscular Dystrophy (PPMD), in the Marathon statement.

    Marathon Pharmaceuticals conducted 17 new pre-clinical and clinical studies and exclusively licensed 2 additional clinical studies to support the New Drug Application submissions to the FDA.

    The effectiveness of Emflaza was established in a multicenter, randomized, double-blind, placebo-controlled, 52-week study conducted in the United States and Canada. The study population consisted of 196 male pediatric patients, aged 5 to 15 years, who were ambulatory and non-ambulatory—one of the largest clinical studies ever conducted in this patient population.

    Patients were randomized to therapy with deflazacort (0.9 mg or 1.2 mg/kg/day), an active comparator or placebo. After 12 weeks, placebo patients were re-randomized to receive either deflazacort or the active comparator for an additional 40 weeks. A comparison to placebo was made after 12 weeks of treatment. 

    The change in average muscle strength score between Baseline and Week 12 was significantly greater for the deflazacort 0.9 mg/kg/day dose group, the recommended dose, than for the placebo group. In addition, increases in muscle strength continued through the 52-week duration of the study, demonstrating persistence of the treatment effect observed at Week 12. 

    Read more: FDA green lights first treatment for rare spinal disease

    Christine Blank
    Contributing Editor Christine Blank is a freelance writer based in Florida.

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