Saving a lean biotech more than $4 million with strategic regulatory support

A US-based clinical stage biotech company had several 505(b)(2) NDA assets in development for rare diseases. This very small biotech operated with a lean team which did not have the necessary regulatory expertise to manage regulatory strategy and submissions. ICON has supported the client with strategic regulatory consulting across their entire portfolio since 2015. During that time ICON has enabled submissions for orphan drug designation (ODD), Fast Track designation, FDA meetings, investigational new drug (IND) filings and IND maintenance.

With a stretched team and limited regulatory knowledge the sponsor needed support advancing their lead asset, a drug for a serious neurological condition with unmet medical need, from pre-IND stage to new drug application (NDA) filing. The sponsor’s goal was to submit their first ever NDA by the end of the second quarter of 2025. Achieving this would require avoiding potential delays and additional study requirements during the submission process. Designation as an orphan drug would be a key element of success, potentially saving the sponsor from additional studies and an expensive Prescription Drug User Fee Act (PDUFA) fee.

ICON Regulatory Consulting Services partnered with the sponsor to provide strategic and operational support from planning for the pre-IND meeting with the FDA to final submission of the NDA filing. ICON collaborated with the sponsor to develop and execute an effective regulatory strategy. Supports included:

• Authoring ODD request
• Composing type B and C meeting requests and briefing packages
• Preparing for and debriefing after FDA meetings
• Formulating module 1, 2 and 3 documents for initial IND
• Assembling and publishing of the initial IND eCTD
• IND maintenance
• Preparing and writing pre-NDA meeting package
• Developing key messaging for module 2 clinical summaries and pivotal Clinical Study Report
• Compiling module 1, 2 and 3 documents for 505(b)(2) NDA, including orphan drug exclusivity request and product labelling
• Compiling and publishing of the NDA eCTD

The sponsor achieved their goal of submitting their first NDA by the end of June 2025 and obtained FDA agreement to a reduced clinical package to support their NDA. This avoided the need for a costly phase 3 efficacy and safety study and accelerated the time to submission. The treatment obtained ODD and therefore was exempt from Pediatric Research Equity Act (PREA) requirements to conduct pediatric studies. Orphan drug designation also meant that the sponsor was not required to pay a a $4 million PDUFA fee for review.

The sponsor continues to partner with ICON to advance their lead asset through Canadian and EU regulatory submissions processes and for regulatory support developing other pipeline products to address unmet medical needs.