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With a long-standing heritage in rare endocrine diseases, HRA Pharma Rare Diseases was established to continue to ensure that life-saving treatments are accessible to patients on a global scale.


Today, HRA Pharma Rare Diseases, the specialised affiliate business of Laboratoire HRA Pharma SAS, is proud to celebrate its successful one year anniversary. The dedicated unit is a result of HRA Pharma’s over 17 year commitment to the rare disease community, which brings valuable solutions to market and enhances the lives of patients, their families and healthcare teams who care for them. HRA Pharma Rare Diseases has a portfolio of products in the global market, including Metopirone®, Ketoconazole HRA™, and Lysodren® with plans to continue its wider global expansion underway.

Since taking off in 2003 with the rights to sell Lysodren®, HRA Pharma Rare Diseases became a separate legal entity in July 2019. To date, Lysodren® is the only FDA & EMEA approved treatment for the aggressive late-stage of Adrenal Cortical Carcinoma (ACC) and is considered a cornerstone of care for this particular cancer. To enable the safe and correct administration of this treatment, HRA Pharma successfully developed Lysosafe®. This service enables healthcare professionals to measure patients’ mitotane plasma levels and is a crucial tool for patients’ monitoring and safety. 

HRA Pharma continued its dedication to rare endocrine patients with the launch of Metopirone® and Ketoconazole HRA™ in 2011 and 2014 respectively, after direct requests from endocrinologists to make these vital products available to their patients. Both are used to treat endogenous Cushing’s Syndrome, a debilitating and potentially life-threatening rare disease associated with significant morbidities and increased risk of mortality. HRA Pharma Rare Diseases has become the market leader in the treatment of Cushing’s Syndrome and continues to cooperate with academic institutions to further invest and support different research and development projects. 

HRA Pharma Rare Diseases has forged strong relationships with organisations worldwide that support rare endocrine diseases and partnered with healthcare practitioners to help develop these lifesaving initiatives. As it enters its second year, HRA Pharma Rare Diseases is eager to broaden these community relationships and establish a wider global presence. Having established a strong presence in Europe, HRA Pharma Rare Diseases looks to expand its global reach and strengthen its presence in key international markets, such as the US, Asia and the Middle East, to ensure that no patient is left behind.

Frédérique Welgryn, General Manager, HRA Pharma Rare Diseases, says, “At HRA Pharma Rare Diseases, we are dedicated to serving the forgotten rare disease community and are proud to be celebrating our one year anniversary. Thanks to our long heritage and strong relationships within the community, we’ve developed initiatives that tackle the patients’ unmet needs. We are committed to enabling worldwide access to treatments that provide the long-term management of rare and ultra-rare diseases and will continue to do so through our global expansion plans.”



About HRA Pharma Rare Diseases:

Established in July 2019, HRA Pharma Rare Diseases was created as a result of the company’s heritage in rare diseases and its close partnerships with the community. HRA Pharma Rare Diseases has a portfolio of medicines that address Cushing’s syndrome (CS) and Adrenal Cortical Carcinoma (ACC). Now well established in Europe, the company continues to grow, with future plans to expand its geographical reach worldwide.


About HRA Pharma:

HRA Pharma is one of the fastest growing consumer healthcare companies in the world and has a strong focus on prescription to over the counter switches. Created in 1996, the objective of HRA Pharma was to provide innovative solutions to medical needs that had not been addressed previously by large pharmaceutical groups.

HRA Pharma is number one in emergency contraception, scar and blister care in Europe after growing the brand with new products and expanding into new markets, with its Rare Disease affiliate being the market leader in the treatment of Cushing’s Syndrome.  

HRA Pharma aims to more than double in size by 2022 by using a combination of swift switching, new products, geographic expansion and smart acquisitions. Currently, HRA Pharma has direct operational presence in 11 European countries and partnerships that have extended its products' availability to over 90 countries worldwide. 

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Last Updated: 01-Jul-2020