Sinema, Kelly, Lesko Advocate for Continued Access to Lifesaving Drug for Arizona Family

May 24, 2021

WASHINGTON – Arizona Senators Kyrsten Sinema and Mark Kelly and Congresswoman Debbie Lesko (AZ-08) sent a bipartisan letter to the Food and Drug Administration (FDA) and relevant leadership of pharmaceutical companies advocating for continued access to a lifesaving drug for 22-month old Arizona boy, Woodrow Miller. A recent decision from Mallinckrodt Pharmaceuticals was announced that provided initial relief for families like the Millers, but will require longer-term solutions.
“Arizona families like the Millers are in heart-breaking situations, and like any parents, are seeking solutions to help access treatments their child needs. 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 foraccess to similar lifesaving care,” said Sinema.
“I’ve lived through the experience of having to be an advocate for a loved one facing a serious medical condition,” said Senator Mark Kelly. “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.”
“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 Congresswoman Debbie 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.”

Woodrow Miller was diagnosed with Niemann-Pick Disease Type C (NPC) soon after birth. NPC is a rare, progressive genetic disorder with no known cure. For months, Woodrow’s mother, Denise Miller, has sought access to the experimental drug adrabetadex, currently manufactured by Mallinckrodt Pharmaceuticals. Due to a decision by Mallinckrodt to discontinue the manufacture of the drug and related trials, children with NPC had been told they would lose access to this critical treatment in October 2021, and newer patients like Woodrow were denied even short-term access.
After months of advocacy and intervention by families like the Millers, dedicated researchers and clinicians, and the offices of Sinema, Kelly, and Lesko, Woodrow finally gained access to adrabetadex. Thankfully, Mallinckrodt also reached an agreement with Mandos, LLC, to transfer the Investigational New Drug application for adrabetadex and allow families to continue receiving the treatment through the FDA Expanded Access Program. The letter advocates for the swift approval of this agreement and continued work by all parties to find a more permanent solution for Woodrow and other families affected by NPC.
Read the full letter HERE.