Partnering with Industry
Removing obstacles and risks industry associates with therapeutic discovery for rare disease
Biopharmaceutical and biotechnology companies face unique challenges – and opportunities - in developing therapies for rare disorders. We understand this. Thus, at NTAP we like to engage early with industry partners and provide biopharmaceutical and biotechnology partners with the essential data and resources needed to de-risk cutaneous neurofibroma and plexiform therapeutic programs and remove obstacles that could otherwise delay new therapies from reaching the market.
NTAP does the following to de-risk your industry efforts to discover therapeutics:
- Provides access to essential clinical data
- Helps define research approaches that are focused and yield rapid answers
- Removes bureaucratic, intellectual property, and legal obstacles
- Supports access to accurate and sufficient patient pipelines
As a nonprofit research accelerator, our only agenda is to advance the development of NF1 therapies. Our complete toolkit of expertise and access is built to help you move from lab to trial to market. We reduce obstacles by supporting collaboration between investigators and industry to help refine research strategies and access the best available resources.
NTAP Partners with AstraZeneca and the First NF1 Drug Is Approved: A Case Study
Among the major industry breakthroughs we supported in the NF1 field has been the approval of the small molecule drug selumetinib (Koselugo) for the treatment of pediatric patients (2 years of age and older) with NF1 and symptomatic, inoperable pNFs.
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 increase enrollment at Children’s Hospital Pennsylvania and Cincinnati Children’s Hospital. NTAP also supported key natural history studies that are used by regulatory agencies including the FDA in the evaluation of selumetinib. Collectively, this work led to the approval of selumetinib for the treatment of plexiform neurofibroma in adolescents in 2020.
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 plexiform neurofibroma.