One local family is celebrating the U.S. Food and Drug Administration (FDA)’s recent approval of Exondys 51 (also known as Eteplirsen), the first drug approved to treat patients with Duchenne Muscular Dystrophy.
The disease – known for short as DMD – is a recessive X-lined form of muscular dystrophy that affects one in every 3,500 to 5,000 boys, including Bay Ridge’s own Pietro Scarso, a seven-year-old boy who was diagnosed at just three years old.
Currently, the second grader is in his 62nd week of trials for the drug which, with help from manufacturer Sarepta Therapeutics, supplements the patient’s absent gene, Exon 51, and slows down the progression of the disease while research and trials continue towards an ultimate cure.
“The boys in the trials such as our son Pietro, in his 62nd week, are walking longer, getting in and out of their beds, they are stronger, and can do so much more than they would be able to do without this drug,” said Pietro’s parents, Manni and Dayna Scarso – co-founders of local organization Pietro’s Fight – in a statement.
Since its inception in 2012, Pietro’s Fight has raised more than $1 million towards finding a cure.
“The FDA did their job in making sure that Eteplirsen was a safe drug prescribed to these children,” said Samantha Altilio, executive director of Pietro’s Fight. “It took 115 days for the FDA to come to a decision, it took courage from these boys and their parents, and it took support from the community and the nation to get this achieved.”
That decision – announced by the FDA on Sunday, September 19 – comes less than one week before Pietro’s Fight’s fourth annual “Fight to the Finish Line,” a 5k race (and one of the organization’s five annual fundraisers) that has kicked off for the last four years from the Veteran’s Memorial Pier at Bay Ridge Avenue and Shore Road.
It will do the same this Saturday, September 24, starting with a special press conference to celebrate the good news at 9 a.m.
“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval.”
“We have been waiting for this day for a long time, when the boys without the Exon 51 gene can get the treatment they need in the fight against Duchenne Muscular Dystrophy,” said State Senator Marty Golden, a longtime advocate for the local organization. “I salute the Scarso family and all you have done over the past four years, and I am proud to have been a partner with you in this mission. This community has and will continue to embrace and stand with Pietro’s Fight and the DMD community to further advance research and raise awareness.”