EU Orphan Designation & SME Strategy for US Biopharma

Strategic Situation 04

Preserving Asset Value Through EU Orphan Designation and SME Strategy for a US Biopharma

Strategic Situation 04

Orphan Strategy · SME Status · EU Market Entry · Health Authority Engagement

Situation

A US-based biopharma establishing its European presence required strategic guidance on EU orphan designation filing, SME status optimisation, and submission infrastructure for EU regulatory engagement. The company needed both strategic regulatory advice and practical support establishing its European regulatory presence.

Contribution

Provided EU orphan designation strategy and filing support, advised on SME status eligibility and benefits, established EU submission portal infrastructure, and guided health authority engagement strategy for initial European regulatory interactions.

Business consequence

Established European regulatory infrastructure and designation status to support long-term asset value, partnering readiness and future EU development flexibility.

How we supported the client

Provided guidance on the benefits and incentives of SME status in the EU for the orphan designation application process

Offered strategic oversight and guidance on the EU orphan designation application process and best practices for navigating the submission process

Assisted in the submission of the Orphan Designation including publishing through our partner network

Assisted in preparing and submitting the application through the appropriate EU portals, including IRIS (a secure online platform for handling product-related scientific and regulatory procedures with the European Medicines Agency (EMA))

Strategically laid out options when and where to go for scientific advice in Europe by weighting national versus central advice based on client`s needs and planned development program

Wrote the scientific advice package
/ orphan designation as well as reviewed

Advised on opportunities of acceleration options in Europe

Submitted a request for a SME
(micro, small and medium-sized enterprises)

Strategic
Regulatory
Advice

Interactions with Health Authorities

Submission
Management

Partnering with industry experts

Client quote

Our partners at Therainnova have been an integral part of our global development team, providing critical EU regulatory insights that have added significant clarity and validation of our overall clinical development strategy

Theresa Matkovits, Ph.D.

Chief Development Officer, Matinas Biopharma