Earlier this year, our CEO Leonardo Calzetti attended BioEurope Spring 2025 in Milan, one of the most important events in the biotech startups industry, bringing together innovators, investors and key leaders from around the world. This event served as an invaluable platform for discussing the challenges and opportunities for startups in Europe.
Among the key themes that emerged, one stood out clearly: the strategic value of expanding into the European market for U.S.-based companies, especially for those developing therapies for rare diseases.
Europe as a land of opportunity for U.S. biotech startups
In today’s highly dynamic global landscape, market diversification can provide strategic, operational and financial support for U.S. biotech startups. Expanding in Europe means accessing fertile ground for expansion, innovation and long-term value creation.
The European Union (EU) is the second-largest pharmaceutical market for orphan drugs, with a population of over 448 million people. This provides a significantly broader patient base compared to the U.S., enabling more extensive market penetration and increased sales potential. Beyond just numbers, the EU offers access to a more diversified healthcare ecosystem, with varied reimbursement models that allow companies to fine-tune their commercial strategies and mitigate the risks associated with a single-market focus.
Securing regulatory approvals from both the FDA and the EMA enhances a company’s global standing. Dual approvals not only reduce the risk of commercial failure but also elevate the company’s profile for IPOs, acquisitions, and strategic partnerships. A well-balanced US–EU pipeline is frequently seen by investors as a marker of maturity, operational strength and future scalability.
Incentives that matter: EMA’s support for rare disease innovation
Beyond market size and diversification, Europe offers a uniquely supportive regulatory environment, particularly for companies targeting rare diseases. The EMA encourages orphan drug development with a comprehensive package of financial and regulatory incentives.
- Extended market exclusivity, a longer runway for return on investment
One of the most compelling incentives is the ten years of market exclusivity for products granted Orphan Drug Designation (ODD). This protection, valid across all 27 EU Member States, ensures that no similar medicinal product for the same indication can be marketed for a decade. It acts as a commercial safeguard, improving predictability and enhancing investor confidence.
Market exclusivity can be extended to 12 years for companies that meet the requirements of a Pediatric Investigation Plan (PIP). Compared to the seven-year exclusivity available in the U.S., this is a meaningful strategic advantage.
- Centralized authorization procedure guarantees one application for all
The centralized authorization procedure administered by the EMA is another major benefit. It enables companies to submit a single marketing authorization application that, if approved, is valid in all EU Member States. This process significantly reduces administrative burden, regulatory complexity and time to market.
For rare diseases in particular, the EMA may also grant conditional marketing authorizations in cases of urgent medical need, allowing earlier access to the market when clinical data is promising but not yet complete. This approach can accelerate timelines without compromising safety and regulatory standards.
- Fee incentives reduce the cost of innovation
The EMA also offers substantial fee reductions for companies developing orphan drugs. These incentives cover key processes such as protocol assistance, marketing authorization applications and inspections.
Fee reductions are especially valuable for early-stage biotech startups, making it more feasible to continue development. Moreover, access to expert protocol assistance at a reduced cost improves alignment with regulatory expectations and increases the likelihood of successful approval.
A cross-border strategy for long-term growth
The insights shared at BioEurope Spring 2025 reaffirmed that for U.S. biotech startups—particularly those focused on rare diseases—securing an ODD in Europe is both a regulatory milestone and a strategic growth lever. It strengthens market positioning, attracts investment and provides robust, long-term support throughout the commercialization journey.
The EU is a strategic landscape. For U.S. biotech startups ready to scale globally, now is the time to explore the untapped potential Europe has to offer.