FDA to hold Jan. 22 hearing on muscular dystrophy drug
Bay Ridge boy is part of clinical trial
The parents of a Bay Ridge boy suffering from a rare form of muscular dystrophy are bringing their fight to save his life to Washington D.C. next month.
Manni and Dayna Scarso said they will speak at a Food and Drug Administration (FDA) meeting in the nation’s capital on Jan. 22 to urge the agency to fast-track approval of a promising new drug called Eteplirsen to fight Duchenne Muscular Dystrophy, the disease that afflicts their six-year-old son Pietro.
Little Pietro is currently taking part in a clinical trial of Eteplirsen, receiving weekly infusions of the drug, and is already showing signs of significant progress, his father said.
“We’re on week 22 of a 96-week trial. He’s now able to climb stairs. He couldn’t do that before,” Manni Scarso, a broker of private jet sales, told reporters at a press conference in John Paul Jones Park in Bay Ridge on Dec. 4.
Duchenne Muscular Dystrophy, which predominantly hits young males, affects a patient’s motor skills and their ability to breathe. It afflicts one in every 3,500 boys in the U.S. A patient is usually confined to a wheelchair by the time he is 18 years old. Most patients do not live past the age of 35.
Pietro has more stamina after taking Eteplirsen, his parents said. “He used to play for a short time [and] would have to be picked up and carried because his legs would just give out. Now he has more energy, more stamina and more confidence,” Dayna Scarso said.
The FDA is expected to make a decision on whether to approve Eteplirsen a month after the Jan. 22 meeting. Medical experts are also expected to testify, the Scarsos said.
Pietro Scarso was diagnosed in 2012. His parents formed a nonprofit organization called Pietro’s Fight to raise money for research into a cure and to raise awareness of Duchenne Muscular Dystrophy. To date, Pietro’s Fight has raised $1 million through events like dinner-dances, 5K races, children’s amusement park visits and pajama days at schools.
Dayna Scarso, a stay-at-home mom, said she is thrilled with the progress she has seen in her son since he started taking Eteplirsen in July. The child has to travel to a hospital in Philadelphia to receive the infusion of the drug. “Duchene Muscular Dystrophy boys usually can’t jump with both feet. He can jump,” she said.
Samantha Altilio, a family friend who serves as executive director of Pietro’s Fight, said she saw the difference. Little Pietro insisted on showing her that he could jump. “Pietro got out of the car and said to me, ‘Samantha, look what I can do!’ It was great,” she said.
The drug, made by Sarepta Therapeutics, is giving families of patients hope, the Scarsos said.
State Sen. Marty Golden (R-C-Bay Ridge-Southwest Brooklyn) was at the press conference to show his support for the family. “We’re aiming for FDA approval in February. We want to make sure this becomes a priority for the FDA,” he said.
Golden said that while he understands that the FDA has to display caution in the approval process for new drugs, he believes families like the Scarsos can’t wait any longer. “There have been nine studies over the last four years,” he said.
The press conference was also held to announce the Second Annual PJs for PJ Fundraiser for Pietro’s Fight to take place on Dec. 17. Students in participating schools will wear pajamas to class to show solidarity with Pietro Scarso and collect donations for the organization.
Last year’s PJs for PJ event was successful, according to Altilio.
Golden said he plans to take part in the fundraiser. “I will be in my pajamas, by the way,” he told reporters.
For more information on the fundraiser, email [email protected].
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