Member Spotlight / News - March 31, 2023

EUCOPE Member Spotlight: Q&A with Avanzanite Bioscience

Every month, EUCOPE spotlights a member company and the great work they’re doing to advance the life sciences industry and drive innovation to serve patients better. In March, we spoke with Adam Plich, Founder and CEO of Avanzanite Bioscience B.V.

Adam Andrzej Plich is the Founder & CEO of Avanzanite Bioscience B.V. (Avanzanite), an Amsterdam-based specialty pharmaceutical company offering end-to-end commercialization and distribution partnerships with research-based biopharmaceutical originators. Prior to founding Avanzanite, his impressive industry experience includes pioneering the European Pricing and Market Access at Teva Pharmaceuticals and running a successful boutique advisory firm consulting for many emerging biotechs setting up European operations.

Tell us about your organisation and its mission and how you drive innovation internally?

We established Avanzanite to address a critical market need in the European orphan drug landscape which is due to the complexity and cost of streamlining business operations across 30+ countries given a limited number of patients and an increasingly challenging market access environment. The result of these unique market dynamics leaves emerging biopharmaceutical companies unmotivated or ill-equipped to establish European operations or enter certain countries.

As a result, there are an increasing number of biopharmaceutical companies either opting not to launch their new orphan medicines in Europe or exiting the market altogether. These dynamics are particularly damaging to emerging biopharmaceutical originators that are the driving force of global pharmaceutical innovation, especially for rare diseases.

Ultimately, the patients are the ones paying the price by missing out on promising therapies. It’s a shame and unfair. Every day, thousands of Europeans are diagnosed with life-altering and rare medical conditions. Yet many lack access to the latest innovations in medicines simply because of where they live. Correcting this systemic inequity needs to be addressed now, as the number of medical innovations that could help patients is expected to dramatically increase in the coming years.

At Avanzanite, we passionately believe that everyone in Europe – and beyond – should be able to benefit from approved orphan medicines. We have developed a market-driven solution, a first-in-class biopharmaceutical commercialization platform specifically addressing rare diseases in Europe, to close the inequity gap.

NOBODY SHOULD BE LEFT BEHIND. That’s why we founded Avanzanite.

How do your organisation’s activities help patients now and into the future?

Avanzanite was founded to capitalize on two primary objectives, one economic, and one humanitarian. The economic goal is to offer end-to-end commercialization and distribution partnership with research-based biopharmaceutical originators to unlock the full value of their orphan medicines in European markets; this is the unique expertise and core competency of the Avanzanite team through its decades of operating experience navigating this area. Equally important, the Company’s humanitarian goal stems from our commitment to enabling access to novel medicines for patients who suffer from orphan diseases regardless of where in Europe they live.

Avanzanite is dedicated to finding hidden gems, late-stage or approved medicines, and supporting the underappreciated heroes of the rare disease space. Derived from the word ‘Tanzanite’ – one of the rarest and most underappreciated gemstones on earth. This strongly relates to our mission. For example, we just signed an exclusive distribution deal with Advicenne, a Paris-based biotech, for an approved medicinal product for a rare paediatric kidney disease. We will be making this drug available to patients in several European countries. Starting with Germany and Greece in record time: in a matter of weeks rather than months. Patients have been waiting for this medicine in these countries already now for two years and we are closing the gap as soon as possible. We are also now preparing a launch for another orphan medicine currently under review by the European Medicines Agency across 26 European Union countries to ensure patients would benefit from this treatment in every corner of the continent.

We also just celebrated our first annual Knights for Rare event, where we spotlighted Acanthamoeba Keratitis, a rare disease close to our hearts. Avanzanite is dedicated to supporting the people who make a difference in the everyday lives of patients, as well as R&D experts who spend their careers working to improve quality of life. We want to bring these people into the spotlight and help them make a bigger impact, raise awareness, and reach more patients across Europe.

What do you see as the biggest challenge facing the life sciences industry today?

As many as 80% of orphan drug launches fail to meet sales goals due to the challenges of the dynamic European marketplace, which means they are not getting to patients in need. In many countries in both Western and Central Eastern Europe, patients are never getting access to approved orphan drugs or are waiting on average 18–36 months for final reimbursement decisions.

This issue will only worsen as the development, research, and approvals of orphan drugs have dramatically increased in recent years and will continue to grow. For example, there are over 800 investigational orphan medicines with open IND status by the FDA; some estimates report that 40% of the current global pipeline is in orphan drugs.

However, once these biotechs complete R&D, they may decide not to launch their products at all. Just in 2021–22, there are several medicines for rare diseases which have not been launched in Europe or in many countries.

There are multiple reasons behind this. First, the investments to set up infrastructure across various markets in Europe is huge. The task is complex and often a distraction for the biotechs whose major skills are in R&D. That applies equally to those based in the US as well as in Europe. Secondly, biotechs struggle to identify patients as there are so few of them. It takes significant investment and unique knowledge to identify a rare disease population and reach them with treatments. Third, with few patients, and huge infrastructure costs, many biotechs face challenging pricing & reimbursement negotiations with national authorities.

All of this requires highly experienced talent. With many companies setting up operations in Europe at the same time, the talent bench is becoming thinner and thinner – and “throwing more money at the problem” is not a substitute for the people that actually “get” what is required to make orphan drugs available in Europe. This is one of Avanzanite’s biggest strengths. Our network has proven indispensable and our people are dedicated to bringing access to every patient possible.

With 50% of all orphan drugs being developed by single-asset companies, we may face a situation where there are innovative medicines – already approved! – for numerous rare and very rare diseases but they may not find patients in Europe. The situation for ultra-orphan medicines, ATMPs, and other niche or overlooked drugs is even worse.

What are the major health policy issues and themes that you are most focused on in 2023?

Avanzanite aims to have a pipeline of around 30 rare orphan treatments over the next 8 years. This is our focus in 2023 and the coming years. We rely on our deep contact base and our network of country managers and field experts. The more we scale, the faster we can reach patients across Europe and the more efficiently and effectively we can help our partners unlock revenue potential that is otherwise missed.

In this context, the key health policy issue that we are most focused on is pricing & reimbursement regulations for orphan drugs at country levels. Our ultimate objective is to contribute to the discussions on this topic so that we can sustainably make our portfolio available to patients across all countries in Europe, including the small and mid-size countries where the inequity gap is the largest.

Since our corporate launch in November 2022, we have been working to identify ultra-orphan drugs, ATMPs, and any other niche or overlooked medicines for rare diseases as the most critical health policy areas. As mentioned earlier, these medicines face particularly unique challenges across many European Member States and something should be done about this. While we aim to contribute to the dialogue with the national and European authorities in this field, we will also continue our efforts to address these challenges through our commercial solution.

What attracted you to join EUCOPE and how can we help you achieve your business goals?

We have always felt we belong to EUCOPE and share its mission and values. The association represents the heroes we highly respect and partner with like R&D innovators that invest significant capital in scientific areas where no one else will. Many of these biotechs develop breakthrough orphan drugs. Yet they often do not reach patients in need and their revenue potential is hampered. It’s a shame and unfair. At Avanzanite, we believe patients with rare diseases should have access to the latest medical innovations regardless of where they live. We also believe biotechs deserve attractive returns on their risky and lengthy investments. Avanzanite’s founding mission is to tackle these critical issues in the European orphan drug space with our unique business model. By joining EUCOPE we hope to share this vision and take every opportunity to collaborate with its members.

For more information about EUCOPE membership

Visit our Benefits of Membership Page, or schedule an introductory call with our Business Development Manager Dante Di Iulio