Biotech case studies

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.

In 2023, a leading China-based biotech firm partnered with ICON to launch a global programme for a key asset. Building on a six-year strategic alliance and success in China, the client expanded into the US and Europe, prioritizing efficiency and cost control. ICON deployed a blended model—FSP in China and the US, and full-service support in Europe — to align with regional needs and strategic goals.

A small Asia-based biotech engaged ICON to manage its EU orphan drug designation submission for a treatment targeting Dercum’s disease. The designation was key to supporting a planned Phase 3 study and expanding into the EU, where the company had no existing presence.

An Asia Pacific based biotech was developing a therapeutic treatment for a neurodegenerative disorder. ICON Regulatory Consulting was engaged to support market entry.

A virtual biotech start-up acquired an immunomodulatory mechanism targeting inflammatory pathways. It had undergone phase 1 and small phase 2a studies. With urgency to advance development and limited resources, the client proposed two separate studies, posing challenges in complexity, timeline, and budget. The client turned to ICON to streamline development.

ICON stepped in when a biopharma company required urgent rescue assistance with two phase 3 trials for Hidradenitis Suppurativa (HS) and Psoriatic Arthritis (PsA).

A leading venture capital fund, focused on advancing breakthrough biopharma innovation, and sought to establish a
scalable operational framework to support its growing portfolio of early-stage companies.

A leading biotech company developing a groundbreaking non-opioid treatment for moderate-to-severe acute pain partnered with ICON to conduct a series of early-phase trials.

ICON conducted a multinational biosimilar phase 1 study for multiple sclerosis (MS). This trial was performed under high regulatory scrutiny and challenging pandemic conditions, with increased dosing targets requiring additional site activation.

A small US-based biotech company was in a race to be the first to market with their lead targeted oncology product.

ICON’s proactivity in risk mitigation, commitment to the study, project planning and communication contributed significantly to the ultimate success of the study.

ICON developed flexible, cost-effective CRF designs for a cell therapy immuno-oncology study, harmonising RECIST and iRECIST criteria while addressing re-treatment requirements and supporting sponsors in meeting study timelines.