FDA SEEKS LARGER TRIAL OF PROMISING DRUG TO TREAT MUSCULAR DYSTROPHY

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By Miriam Raftery

April 21, 2014 (Washington D.C.) --The U.S. Food and Drug Administration, or FDA, is on the verge of approving the first-ever drug treatment for a severe form of muscular dystrophy.  Duchenne muscular dystrophy is a deadly disease that typically  leaves its victims in wheelchairs by their teens and dead in their 20s, according to the U.S. Human Genome Institute.  This disease strikes one in every 3500 boys worldwide.

There has never been a cure or an effective treatment to slow down the progression of the disease, only treating symptoms such as trouble breathing. But now a new drug called eterplirsen made by Sarepta Therapeutics has shown dramatic results in a clinical trial.  The test was small, with only 12 boys—but  all 12 had muscle deterioration  virtually halted after taking the drug.

Most striking is the case of two brothers. One is running and playing after participating in the clinical trial. His brother, who did not, is now in a wheelchair and is now having difficulty feeding himself. 

Despite these dramatic results,  and a lack of side effects, the FDA has been reluctant thus far to approve the drug without more testing and information. A separate trial that used a similar technology, though somewhat different treatment, failed, the Washington Post reports. 

But patients families say waiting could cost them their children’s lives, so they have been  lobbying in Washington and filed a White House petition asking that the drug be approved, also posting  online videos of boys benefiting from eterplirsen.

Duchenne muscular dystrophy is a mutation in a gene that produces dystrophin, a protein essential for muscle function. The eterplirsen drug works by bypassing the mutation and restoring the gene’s ability to produce dystrophin.  This can transform Duchenne into a much milder form of muscular dystrophy called Becker.   

Jerry Mendell, a pediatric expert at Nationwide children’s Hospital in Ohio, was the lead investigator on the clinical trial.  He calls the results “unequival,” adding, “Since the day the kids were started on eteplirsen, they basically have never changed again…I see it as a game changer.”

Only about 13 percent of Duchenne patients have the gene mutation that would allow them to benefit from eteplirsen, but Sarepta is developing medications to treat other gene mutations for muscular dystrophy patients. The disease is hereditary,

THE FDA has now asked for a larger clinical trial with up to 100 patients involving a winder range of ages, in hopes of confirming the promising results and verifying the drug’s safety. 

The treatment is not a cure, since it halts the progression of the disease, but does not reverse it.  So time is of the essence for families with children stricken by Duchenne Muscular Dystrophy.

Even if approved, however, the new drug could be among the most expensive ever to come to market, according to the Washington Post, raising questions over just how accessible the treatment would be for families with limited financial means.

 

 


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