Dear Steve and Elissa,
I am writing on behalf of the Niemann-Pick Type C (NPC) Therapy Accelerator in regard to the adrabetadex program. Specifically, we would like to set up a time to speak with Mallinckrodt (MNK) about the future of adrabetadex, and discuss possibilities for allowing the NPC community to gain access to the investigational product and the related data which has been collected over the years. Below are some of the questions that we have outlined to help prepare for the meeting:
- Is Mallinckrodt willing to donate data from the clinical trials to the NPC community to ensure its curation and access for future research/clinicians/families. We are in talks with the International Niemann-Pick Disease Registry about being a secure repository for this information. Other options would include the NIH or the Critical Path Institute.
- In recent communications, you mentioned hope for a path forward and the possibility of transitioning the asset to another entity or it would go back to the NIH. Can we discuss the potential options and what would need to happen to pursue the options in terms of intellectual property, liability, technology transfer for manufacturing and similar transitional considerations
- On the February 3, 2021 webinar, MNK said the IND would revert back to NIH in 6 months if no one else pursued it. Please provide the start and end date for this 6-month period?
- Would MNK consider reverting the IND back to the NIH sooner?
- The NPC community would like to have a dialogue with the FDA and discuss the potential of a path forward. Since Mallinckrodt is the IND-holder, we would respectfully request that you request a meeting for us to join you to speak with the FDA.
- In your conversations with the FDA, to better understand what additional work FDA might expect in future studies (if any), what were the major gaps in the data expectations of the regulator and specifically whether there were issues FDA identified when comparing to historical controls like the Natural History Data and other controlled data that is available?
- Who was Mallinckrodt’s manufacturer/supplier of adrabetadex and would you put us in contact with them so we can explore if ongoing access might be feasible from a supply chain perspective?
- What amount and form of adrabetadex supply is left? What is the shelf life of each batch or formulation (i.e. formulated liquid, powder, etc… with different manufacturing dates)? Will the remaining supply extend beyond October 2021 if an alternate path forward is achieved?
- If the NPC community found a path forward to support extended access, would you be willing to help us manufacture another batch given the time-sensitive nature of the process and the desire to prevent gaps in patients’ treatment?
Thank you and we are happy to work with your administrators to coordinate this meeting. The NPC Therapy Accelerator team includes:
The NPC Therapy Accelerator Team