Mark Kelly, Kyrsten Sinema, and Debbie Lesko advocate for access to lifesaving drug for Arizona child

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This week Rep. Debbie Lesko and Senators Kyrsten Sinema and Mark Kelly joined forces to help a desperate Arizona family.

The leaders sent a bipartisan letter to Acting Commissioner of the Food and Drug Administration (FDA) Janet Woodcock and leadership of pharmaceutical companies to advocate for continued access to a lifesaving drug for their 22-month old constituent, Woodrow Miller. The little boy has a rare disease called Niemann-Pick Disease Type-C. The disease, some consider a form of childhood Alzheimer’s, will eventually take away Miller’s ability to walk, talk, swallow, eat, and breathe, as it weakens their muscles, brain, hearing, sight, and motor skills. Currently, it is fatal 100 percent of the time. “There are roughly 100 kids in the United States with this disease, 500 in the world,” Denise Miller, Woodrow’s mother, told ABC15.

There is a drug that the Miller family hopes can help him. They hope an experimental drug called Adrabetadex could give them more time with their son. The family has heard testimony from other parents that the drug has prolonged the lives of their children. 

However, the pharmaceutical company Mallinckrodt Pharmaceuticals decided to stop producing the drug, reported ABC15, because clinical trials did not show promising results. The company had decided about 40 children who are already using the drug until October 2021 before cutting off access. 

Lesko heard about the issue knew she needed to help. “After speaking with Woodrow’s mother, Denise, I sent a letter to the FDA advocating for access to this lifesaving drug. I also spoke directly with Secretary of Health and Human Services Xavier Becerra about Woodrow’s condition and the importance of him gaining access,” said Lesko. “While I am so encouraged that Woodrow is currently receiving this treatment, the fight is not over. I will continue to do what I can to encourage the parties involved to continue providing this lifesaving treatment to Woodrow and other children affected by this horrible disease.”

After months of advocacy and intervention by the offices of Lesko, Sinema, and Kelly, the Woodrow’s gained access to adrabetadex through a clinical trial and the FDA’s Expanded Access Program. Mallinckrodt reached an agreement with Mandos, LLC, to transfer the Investigational New Drug application for adrabetadex and allow its continued utilization through the Expanded Access Program.

“Arizona families like the Millers are in heart-breaking situations, and like any parents, are seeking solutions to help access treatments their child needs,” stated Senator Sinema. “We are pleased the companies and stakeholders like the FDA, at our urging, were able to find a solution to ensure that the Millers will be able to access lifesaving treatment for Woodrow, and we’ll continue supporting Arizona families asking for access to similar lifesaving care.”

Sen. Kelly commented, “I’ve lived through the experience of having to be an advocate for a loved one facing a serious medical condition. When I spoke with Woodrow’s mom, we talked about the challenges of searching for and accessing the best care, just as their family is doing now. No family should have to jump through hoops to get their child access to the care they need. I’m so glad that we were able to get Woodrow access to this critical treatment he needs.”

The letter advocates for the swift approval of this agreement and continued work by all parties to find a solution for Woodrow and other families affected by NPC. Read the full letter here.