FDA Interactions
REiNS outcome measures have shaped FDA regulatory decisions for neurofibromatosis therapeutics, including the first-ever NF1 drug approval.
First NF1 Drug Approval
REiNS criteria underpinned the landmark FDA approval of selumetinib — the first drug approved for NF1.
Endpoint Standardization
FDA now references REiNS-recommended endpoints when reviewing NF clinical trial designs.
Pediatric Considerations
REiNS recommendations address age-specific measures, aligning with FDA pediatric labeling requirements.
Open Access Policy
All REiNS publications are open-access, supporting FDA reviewers and sponsors in accessing the evidence base.
Regulatory Milestones
Selumetinib (Koselugo) FDA Approval
The FDA approved selumetinib for pediatric patients with NF1 and symptomatic, inoperable plexiform neurofibromas. REiNS volumetric MRI criteria were used as the primary endpoint in the SPRINT trial that led to approval.
REiNS Criteria in FDA Drug Approval
REiNS imaging criteria were specifically cited in the FDA's basis for approval of selumetinib, marking the first time NF-specific response criteria developed by REiNS were used to support regulatory approval.
FDA Oncology Center of Excellence Collaboration
REiNS representatives met with FDA representatives to discuss the use of REiNS outcome measures and response criteria in NDA submissions and IND applications for NF therapeutics.
Pediatric Rare Disease Priority Review
REiNS collaborated with FDA in discussions around the pediatric rare disease designation for NF1 therapeutics, providing input on endpoint acceptability for plexiform neurofibroma trials.
First REiNS Recommendations Published
The inaugural REiNS supplement in Neurology established standardized response criteria, initiating dialogue with regulatory bodies about acceptable endpoints for NF clinical trials.
Working with Regulatory Agencies
REiNS actively engages with the FDA and other regulatory agencies to ensure that consensus recommendations are aligned with regulatory requirements for endpoint acceptability. The collaboration welcomes dialogue with sponsor companies designing NF clinical trials. For regulatory consultation inquiries, contact reinscollaboration@gmail.com.