Both PTC Therapeutics and Sarepta Therapeutics expect to continue providing an uninterrupted supply of their Duchenne muscular dystrophy (DMD) therapies during the COVID-19 pandemic, representatives from the companies said in a Wednesday webinar hosted by Parent Project Muscular Dystrophy (PPMD).
PTC is the maker of Emflaza (deflazacort). Building on a statement issued in March, John Wing, PhD, senior medical science liaison at PTC, said in the webinar that the company does not anticipate COVID-19 will interfere with Emflaza’s supply.
“As of this update, we have more than a one-year supply of Emflaza in stock and ready for delivery. In addition, we currently have at least another year’s supply of the active pharmaceutical ingredient [deflazacort] ready for production,” Wing said. He added that most of the compounds used to make Emflaza are available in the U.S.
“We are closely monitoring shipping issues in the United States, which could have an impact of delivery of drugs to patients,” Wing said. “We will update our patients and heathcare providers immediately if challenges arise.”
Sarepta markets two therapies also for DMD: Vyondys 53 (golodirsen) and Exondys 51 (eteplirsen). Like PTC, Sarepta expects that COVID-19 will not significantly affect the supply of their approved treatments.
“With respect to supply, we are able to fully serve the community of patients on our approved therapies without interruption,” said Diane Berry, PhD, Sarepta’s senior vice president, global health policy, government and patient affairs. “We have inventory, we continue to manufacture, and we have a robust supply chain, and we have activated plans to minimize the potential for disruption.”
Both companies are offering support programs for people using their therapies. For people taking Emflaza, financial and educational support is available through PTC Cares, which can be accessed online or by calling 844–478–2227.
A similar program, SareptAssist, is available to people on Sarepta’s therapies, online or by phone at 888-727-3782.
In both programs, specialized case managers are available to help people navigate these challenging times.
“If there’s anything that you’re uncertain about or that you feel uncomfortable with, just call us,” said Dan Madden, executive director of patient services at Sarepta.
A particular topic of uncertainty in the current situation, which was discussed at length in the webinar, concerns home care and home infusions of therapies. Both Vyondys 53 and Exondys 51 are given via intravenous infusion (into the bloodstream). Getting these treatments at home requires the help of nurses, but such visits may increase the risk of infection with the new coronavirus.
“Balancing out having enough care and limiting exposure … it’s a struggle,” said Lisa Leesman, private duty nursing education specialist at Cincinnati Children’s Hospital Medical Center. Leesman said that nursing agencies like hers are doing what they can to minimize risk. This largely involves following guidelines from the Centers for Disease Control and Prevention (CDC), such as wearing appropriate personal protective equipment (PPE), including masks.
Yet, deciding how to mitigate risk is a highly individualized choice. According to Leesman, choices by patients and their families about home care during the pandemic vary substantially case-by-case. Some have decided to postpone home visits altogether, while others have limited visits to only certain times or select healthcare workers. “We’re supporting all of those decisions,” Leesman said.
Regardless of what choices are made, implementing appropriate preventive measures can be stressful, even scary, especially for young children. For instance, having nurses wear PPE — such as masks and goggles — can be intimidating without appropriate context, Leesman said.
Providing such context to children is important, she added. “Put the mask on the child; let the child play with that equipment a little bit.” Leesman also suggested a simple explanation along the lines of, “I’m wearing this because I want to make sure my germs don’t get near you, and I’m not sick, but I just want to be extra careful.” She said that any “element of play” can be especially helpful for young children.
The webinar speakers agreed that, especially in these uncertain times, communication at all levels is key.
“Caring for a loved one with Duchenne takes a team,” Wing said. “During these difficult times, you’re not alone. You should reach out to PPMD, you should reach out to PTC Cares, your healthcare providers and other people … We’re here for you.”
“We know this is a really stressful time for families, and it’s made even more stressful because we can’t interact and communicate in the ways we are used to,” Berry said. “We know weathering crises, uncertainty, managing transitions — these are not new for this community. You are pros at creating new normals every day, and your strength and resilience inspire us.”
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