Sens. Kelly, Sinema and Rep. Lesko Advocate for Continued Access to Lifesaving Drug for Arizona Child with Rare Health Condition

Today U.S. Senators Mark Kelly (D-AZ) and Kyrsten Sinema (D-AZ) and Congresswoman Debbie Lesko (AZ-08) sent a bipartisan letter to Acting Commissioner of the Food and Drug Administration (FDA) Janet Woodcock and relevant leadership of pharmaceutical companies advocating for continued access to a lifesaving drug for their 22-month old constituent, Woodrow Miller.  

“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.”

“Arizona families like the Millers are in heart-breaking situations, and like any parents, are seeking solutions to help access treatments their child needs,” said Senator Kyrsten 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.” 

“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 Type C1 (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. 

After months of advocacy and intervention by the offices of Kelly, Sinema, and Lesko, Woodrow finally gained access to adrabetadex through the FDA’s Expanded Access Program. However, due to a decision by Mallinckrodt to discontinue the manufacture of the drug and related trials in October 2021, Woodrow, and many other children with NPC, will potentially lose access to this critical treatment. 

Thankfully, Mallinckrodt has 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. 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.