A Valley boy diagnosed with a rare and fatal childhood disease is now being allowed access to an experimental drug.
Denise and Rickey Miller's 21-month-old son Woodrow has Niemann-Pick Disease Type C. It's a form of childhood Alzheimer's that eventually takes away the child's ability to walk, talk, swallow, and breathe.
For months now, Woodrow's family has been fighting to get their child access to Adrabetadex, an experimental drug that had been undergoing clinical research by Mallinckrodt Pharmaceuticals.
However, Mallinckrodt decided to stop research on the drug after clinical trials showed no promising results.
Families of children who are taking the medication have been fighting back, saying their children are proof the drug works.
The pharmaceutical company did provide children already getting the medication access to it until later this year when they planned to stop all production of the drug.
However, Woodrow was not one of those children. Denise and Rickey have been on a campaign to get their child access to Adrabetadex before it is too late for him.
Statistically, Denise said, her son only has one more year left to live without any treatment. The Millers have already started to see their son losing motor functions and getting weaker by the day.
ABC15 has learned Mallinckrodt Pharmaceuticals has reached an agreement with another company, Mandos, LLC., to acquire Adrabetadex.
The acquisition, if approved, would allow Mandos, LLC to continue clinical research and development of Adrabetadex. This means children with Niemann-Pick Disease will have access to the drug for at least another two years.
ABC15 spoke to Woodrow's family Tuesday night. Denise said the company had agreed to give Woodrow access to the medication. The family left for Rush University Medical Center in Chicago Tuesday night to start his treatments. Woodrow is scheduled to get his first dose of Adrabetadex on Friday.
It is important to note, there is no promise that this medication will save his life as Niemann-Pick Type C is a fatal disease.
Denise feels, however, it gives them hope that Woodrow will live a longer and healthier life.
Mallinckrodt officials issued the following statement Tuesday night:
"Mallinckrodt Pharmaceuticals has reached an agreement to divest and transfer the Investigational New Drug (IND) application for experimental drug adrabetadex (VTS-270) to Mandos, LLC (“Mandos”). Additionally, Mallinckrodt plans to work with Mandos in an effort to secure production of additional drug supply for children who are suffering from the ultra-rare and devastating Niemann-Pick Type C1 disease (“NPC1”).
The planned supply is expected to allow access to adrabetadex for potentially up to two years for patients who are eligible to continue treatment under expanded access or other future potential development programs. The decision to plan for additional drug supply comes after Mallinckrodt’s careful consideration of the needs and concerns of children with NPC1 and the ability to make drug supply available for the continuous treatment of NPC1 patients in the near-term, as allowable.
Under the agreement, Mallinckrodt will transfer sponsorship of its IND application for adrabetadex, which is filed with the U.S. Food and Drug Administration, to Mandos at closing. The agreement and the closing of the transaction is subject to approval of the U.S. Bankruptcy Court for the District of Delaware as part of Mallinckrodt’s Chapter 11 filing.
Mallinckrodt announced in January of this year that it would discontinue the development of adrabetadex following a comprehensive analysis of clinical data that did not show clear evidence of benefit for NPC1 patients. However the Company is aware of and acknowledges that some parents and treating physicians report observing benefit in children being treated with adrabetadex.
“Recognizing both the critical need for new treatment options for Niemann-Pick disease and the concerns of the NPC patient community – who we have worked closely with since acquiring adrabetadex in 2018 – MNK has worked judiciously to identify an entity that would seek to find a path forward for the drug,” said Steven Romano, M.D., Executive Vice President and Chief Scientific Officer at Mallinckrodt. “We are hopeful Mandos will further advance both the understanding of the disease and the potential to develop the drug as a future treatment option for NPC patients.”
Mallinckrodt’s team would like to thank the patients, families, treating physicians and NPC community for their support over the last several clinical study years. We have come to know these families and the community personally, and hope this announcement provides some initial comfort as potential options are considered for the program’s path forward. If the transaction is approved, Mallinckrodt will assist Mandos in the closure of the existing clinical program and fully transition the expanded access over the course of the next several months in support of Mandos and the patient community. The Company will provide additional updates on the progress of the approval of the transaction and the agreement with Mandos at the close of the transaction."