Rare Muscle Wasting Disease First Possible Cure Rejected by FDA

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BioMarin Pharmaceutical Inc. failed in convincing the Food and Drug Administration (FDA) with regard to the efficacy of its Kyndrisa drug in the treatment of Duchenne Muscular Dystrophy or DMD. Furthermore, BioMarin's drug did not win the approval of the FDA citing concerns on the potential long-term adverse effects, such as kidney damage and a reduction in the number of blood platelets.

Kyndrisa was regarded by some people as a potential treatment and would have been the first of its kind in the U.S. for the rare muscle-wasting condition had it not been rejected by the FDA.

"DMD is a progressive, degenerative genetic disorder that hampers muscle movement and affects one in 3,600 newborn boys. Most patients die by the age of 30. DMD has no cure.," according to a report from Reuters.

Meanwhile, another drug is up for review by the FDA. Sarepta Therapeutics Inc. is hoping that its DMD drug eteplirsen won't suffer the same fate as BioMarin's Kyndrisa. Both of the drugs work by helping in the production of dystrophin, which is a type of protein essential in the muscle fibers and could be the cause of DMD if lacking.

Heather Behanna, an analyst from Wedbush Securities, said that the two drugs could benefit a certain group of people with DMD accounting to about 1,800 boys in the United States and another 5,000 around the globe.

As for BioMarin Pharmaceutical Inc., it's not the end of the world for them as they still await the approval of Kyndrisa by the regulating bodies in Europe. And an approval this time around could soften the blow of the rejection by the FDA on its drug, especially, because Europe is a bigger market with a larger patient population.

DMD is a type of muscular dystrophy that usually affects the male population, although, girls can also suffer from the condition. One of the main signs exhibited by DMD patients includes progressive muscle weakness, according to the Mayo Clinic.

Other signs and symptoms, which typically appear between the ages of 2 and 3 years old, are frequent falls, difficulty getting up from a lying or sitting position, trouble running and jumping, waddling gait, walking on the toes, large calf muscles, muscle pain and stiffness and learning disabilities.

"Duchenne affects approximately one in every 3,500-5,000 male children, making it the most common fatal genetic disorder diagnosed in childhood," according to a report from PharmaFile. "There is currently no FDA-approved therapy designed specifically to treat Duchenne."