Neurofibromatosis Therapeutic Acceleration Program (NTAP) initiatives are informed by a clear understanding of the challenges faced by drug companies who work in rare disorders.

Based on early engagement with industry partners, we are actively working to ensure the essential data and resources are in place to de-risk cNF therapeutic programs and remove obstacles that could otherwise delay new therapies reaching the market.

As well, we actively support collaboration between investigators and industry to help refine research strategies and access the best available resources.

Among the major clinical developments in the NF1 field has been the evaluation of the small molecule drug Selumetinib (AZD6244; Hydrogen Sulfate) for the treatment cutaneous neurofibroma.  In supporting the continued evaluation of Selumetinib for the treatment of cNF, NTAP partnered with AstraZeneca (the producer of Selumetinib), and the National Cancer Institute Pediatric Oncology Branch (NCI-POB) and Cancer Therapies Evaluation Program (CTEP) to open the study at Children’s Hospital Pennsylvania and Cincinnati Children’s Hospital.  These studies will provide additional clinical data that will be used by regulatory agencies. By ensuring all of the required primary and secondary endpoints (as well as exploratory endpoints) were met in a timely fashion, enrollment was completed in <6 months and the milestone for the primary endpoint triggering filing for review with the FDA was reached in September 2017.  This notable achievement was possible because of a collaborative spirit between academic, industry, public agency partners working together, and which has set the stage for the first approved medicine for patients with cutaneous neurofibroma.