Company Award for Innovation

For a pioneering company using innovative methods to develop treatments for rare diseases.

The winner is selected on the basis of the following criteria:

  • Is the company developing treatments that address unmet medical needs?
  • Does the company work in therapeutic areas with small patient populations and in which there is currently no treatment available?
  • Are the company's treatments based on new, promising research or technology?
  • Does the company build partnerships with other stakeholders (i.e. academia/universities, entrepreneurs, clinicians) to explore research/therapies that could be applied to rare diseases?
  • Has the company's engagement with patients led to more relevant trial designs or safer and more adequate treatments?
  • Does the company's business model enable the acceleration of advances in rare disease therapies?
  • Does the company facilitate access to its treatments for all rare disease patients?


Previous Awardees:


HEALX logo

2022 Awardee: Lysogene

The EURORDIS Company Award for Innovation recognises Lysogene as a pioneering gene therapy company which has established itself among major players driving European-led innovation, in just over ten years. We are particularly encouraged by Lysogene’s promising advances towards delivering novel treatments in neuro degenerative and neuro developmental disease areas with high unmet medical needs. Lysogene’s continued readiness to advocate for early diagnosis and engage patients and caretakers as equal decision makers in the product development lifecycle also merits acknowledgement. The award also celebrates the remarkable personal journey of the company founder, as a mother, patient advocate and entrepreneur determined to change the landscape for severe rare diseases affecting children’s central nervous system.




2021 Awardee: Orchard Therapeutics

The EURORDIS Company Award for Innovation recognises Orchard Therapeutics’ leadership in harnessing the potential of hematopoietic stem cell gene therapy to change the course of severe inherited disorders.

EURORDIS particularly acknowledges the company for developing gene therapy for early onset metachromatic leukodystrophy (MLD), which addresses the urgent need of young patients and their families to treat a disease for which no approved therapeutic option previously existed.

2020 Awardee: Healx

HEALX logoThe Company Award for Innovation recognises Healx’s promising alternative model for drug discovery, integrating Artificial Intelligence and emerging technologies to identify treatments for rare diseases.

The innovative and ambitious Rare Treatment Accelerator programme pulls together the resources and expertise of diverse stakeholders to fast-track development and access to repurposed treatments for rare diseases.

This award places a spotlight on Healx’s significant investment to develop potentially life-changing medicines for people living with a rare disease, as well as the Company’s drive to promote an alternative framework to address rare diseases.





2019 Awardee: Chiesi

For Chiesi’s strong commitment to rare diseases and its support to policy development, as well as its strong pipeline for bringing to market a wide number of treatments, including products for extremely rare diseases.

This Award recognises Chiesi’s significant investment in Research and Development for rare diseases and its involvement at the European level in recent years. Chiesi’s longstanding, supportive relationship with EURORDIS is another reflection of this dedication, including its membership of ERTC since 2007 and its support to many of EURORDIS’ projects, including the Black Pearl Awards, the Membership Meeting and RareConnect. The Award also recognises the Company’s support to policy development, as well as its strong pipeline for bringing to market a wide number of treatments, including products for
extremely rare diseases.





2018 Awardee: Novartis

The EURORDIS Company Award for Innovation recognises Novartis for its longstanding track record in developing medicines for rare diseases. Through effective collaborations with the scientific, medical and patient communities, Novartis has a promising pipeline to address many rare diseases, including rare cancers. In 2017, Novartis received the first FDA approval for a gene therapy to treat cancer in children and young adults.

EURORDIS applauds the innovation that is the hallmark of the rare disease community. There are over 6000 rare diseases, an estimated 30 million people living with a rare disease in Europe and 300 million worldwide. The fact remains that few treatments are available for the majority of these diseases; many have no appropriate treatment or go undiagnosed. Continued innovation through effective collaboration is needed now for the millions of people worldwide who are living with a rare disease, for which Novartis is an excellent example.




2017 Awardee: GlaxoSmithKline

Since 2010, GSK has designated rare diseases as one of its coretherapeutic areas. In 2016, GSK brought to market an innovative gene therapy to treat patients with the rare disease adenosine deaminase severe combined immunodeficiency syndrome (ADASCID). “It is a huge privilege and a great honour, as well as humbling to accept this award on behalf of GSK. Since coming into the rare disease universe, I have felt overwhelmed by the welcome that you get, that everyone has a single-minded goal which is all around patient treatment and patient outcome. You don’t have to look very far to be inspired in this world. This award goes to all the people who have the courage, determination and focus, and who put in all their energy to make a change for all people living with severe rare disease. And of course to all the patients and families who drive us each day.”
Martin Andrews





2016 Awardee: Actelion, Switzerland

Established in 1997, Actelion focuses on the discovery, development and commercialization of innovative drugs for diseases with significant unmet medical needs. The Company has, in particular, made a difference for pulmonary arterial hypertension (PAH) in Europe and globally. Actelion’s pipeline reflects continued commitment to address unmet medical needs, and they have engaged with and supported patient organisations since their founding. An Emerald member of the EURORDIS Round Table of Companies (ERTC), Actelion has supported several key EURORDIS initiatives, including: the EurordisCare survey, EURORDIS Membership Meetings, and the Black Pearl Evening, which helps make possible EURORDIS’ actions to end isolation of people living with a rare disease, empower leaders of the rare disease patient community, and raise awareness of all rare diseases.






2015 Awardee: Pfizer, Inc.


Pfizer is one of the world’s premier pharmaceutical companies, and has demonstrated commitment to the rare disease cause - with 22 approved products to treat rare diseases worldwide including 4 in Europe. In 2010, Pfizer established its own Rare Disease Research Unit (RDRU), with the objective of taking an innovative and collaborative approach to the development of new medicines to create novel therapeutics across the spectrum of rare diseases. The current pipeline includes clinical and pre-clinical programmes in several rare diseases including sickle cell disease, haemophilia, muscular dystrophies, cystic fibrosis, and more. Through this award, EURORDIS recognises the role major pharmaceutical companies can play in the development of, and ensuring access to, innovative treatments for rare disease patients. The award also encourages companies to maintain a high level of corporate social responsibility by reassessing the value of medicines today so as to ensure that unmet medical needs are covered in the development of new treatments.




2014 Awardee: Orphan Europe


Orphan Europe, forming part of the Recordati group, has 25 years’ experience in bringing orphan medicinal products to the market. Seven orphan products produced by Orphan Europe have been authorised to date and this rich portfolio of successful treatments is supported by the robust pipeline of medicines in development. Orphan Europe’s support in the development of European Reference Networks EuroWilson, EPNET, E-IMD, E-HOD has helped to provide European guidelines for best care and treatment of rare disease patients. Strong alliances with patient organisations mark the central point of Orphan Europe’s platform for therapy development, working alongside patient organisations for cystinosis, renal genetic diseases (AIRG), metabolic diseases and porphyrias.
Orphan Europe also consistently supports networking and capacity-building for rare disease patient advocates via the EURORDIS Membership Meeting.




2014 Awardee: Sobi


Sobi, an international healthcare company dedicated to rare diseases, focuses on developing innovative treatments across four key therapeutic areas: haemophilia, inflammation/ autoimmune diseases, inherited metabolic diseases and oncology. Sobi is particularly sensitive to the need for targeted treatments that are accessible to paediatric populations. Sobi’s track record of creating successful dialogue with patient communities demonstrates their objective for a transparent and progressive rare disease framework. Sobi is represented on several European Union committees such as the Commission Expert Group on Rare Diseases (replacing the EUCERD) and the working group on Mechanism of Coordinated Access to Orphan Medicinal Products (MOCAOMP). This award recognizes the excellence and consistency of Sobi’s work in the rare disease community.




2013 Awardee: Prosensa


Prosensa has achieved several Orphan Drug designations and developed an impressive clinical portfolio in the short time since its founding in 2002. With the Company’s commitment to “develop innovative, RNA based therapeutics to fill unmet medical needs for patients with genetic diseases,” Prosensa has the potential to make a life changing difference for people living with rare diseases. Prosensa currently has several compounds in development for treating Duchenne Muscular Dystrophy (DMD), including a development in collaboration with GlaxoSmithKline for the development and commercialization of RNA based therapeutics for DMD. Prosensa has established partnerships with the patient community through strategic partnerships with muscular dystrophy focused patient organisations, including EURORDIS Members Duchenne Parent Project, Aktion Benni & Co e.v., and the AFM (French Muscular Dystrophy Association).




2013 Awardee: Celgene Corporation


Since its inception in 1986, Celgene’s leadership in the research, discovery, development and marketing of treatments for rare cancers has significantly improved conditions for rare disease patients. Their steadfast and significant reinvestment in research and development place it in a position to make a life-changing difference for thousands more rare disease patients in the future. The Company’s commitment to innovation is reflected in the more than 200 clinical trials underway worldwide using compound developed at Celgene. Celgene has created Celgene Patient Support® to assist patients worldwide in accessing products the Company has marketed in their respective countries. Celgene has a longstanding, supportive relationship with EURORDIS and other patients organisations in Europe and internationally. Most notably, the company supported  the pilot and growth of RareConnect: the Online Patients Communities Project, which enables people affected by rare diseases to form communities across languages and geographic barriers.





2013 Awardee: Genzyme, a Sanofi Company


Genzyme is a pioneer in researching, developing and marketing medicinal products for patients living with diseases. Now part of Sanofi, Genzyme, a Sanofi Company continues to offer hope to people living with genetic diseases, endocrine and cardiovascular diseases. Strategies to increase access to Genzyme products include free drug programs and humanitarian initiatives. Genzyme began this practice in 1999, establishing the Gaucher Initiative, a humanitarian partnership to provide the Company’s first product to Gaucher disease patients in developing countries. The Company is currently one of four co-funders of EURORDIS’ work in the EpiRare project, a three-year project designed to address the need for rare diseases registration throughout Europe. Genzyme is also a long-time sponsor of EURORDIS Membership Meetings and an Emerald Member of the EURORDIS Round Table of Companies.





2012 Awardee: CSL Behring


In recognition of the Company’s longstanding commitment to rare disease
patients, and most recently for its advances to benefit people living with primary immunodeficiency diseases and secondary immune-deficiencies.



EURORDIS Black Pearl Awards highlights