Advancing treatments for rare diseases together

rare disease

27Feb

EspeRare, an efficient model for addressing rare diseases

rare disease, Esperare, conférence, drug repositioning

The UK-based organisation Findacure invited EspeRare to present its innovative model at a conference on drug repositioning. This annual event brings together in London major stakeholders in the field (patient groups, clinicians, researchers and life sciences professionals)  on the occasion of World Rare Disease Day. This is a unique opportunity for all these actors to discuss the latest advances in drug repositioning as a source of future treatments for rare diseases.

Caroline Kant, EspeRare's CEO, will explain the philanthropic venture model and how it accelerates the development of treatments for rare diseases. Click here for details on her presentation.

Picture credit: Barbara Asboth

26Sep

Rimeporide receives Orphan Drug designation from USA health authorities

Rimeporide, orphan drug, USA, orphan drug designation, orphan disease, rare disease, Duchenne, Duchenne Muscular Dystrophy, DMD

The Food and Drug Administration (FDA), the US Department of Health and Human Services, has granted an Orphan Drug Designation (ODD) for Rimeporide for the treatment of Duchenne muscular dystrophy (DMD). See here for more details.

ODD is designed to promote the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases. In particular, this ODD grants Rimeporide a 7-year data exclusivity in the US.

Rimeporide already received ODD in DMD from the European Medicines Agency in 2015. Further to this designation in Europe, EspeRare has been able to conduct a phase Ib clinical trial of Rimeporide on young DMD patients. This clinical trial will be completed by the end of 2017.

07Jul

EspeRare renews hope for children suffering from a disabling skin disease

XLHED, rare disease, NFED, re-launch

EspeRare is currently looking to re-launch the development of ER004 (formerly known as EDI200) as a treatment for babies affected by X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED).

XLHED is a serious rare disease that is life-threatening, particularly in the first years of life. Infants affected by this genetic disorder cannot sweat and are at risk of sudden death due to hyperthermia and severe respiratory issues. See here for more information on the disease.

ER004 is the first and only therapy for this disease. ER004 was developed until 2015 by Edimer Pharmaceuticals (US). Unfortunately its development was stalled following a clinical setback as the treatment, when administered to newborn babies, did not seem to provide therapeutic benefits.

In the meantime, Prof. Holm Schneider, a German medical expert, renewed hope for XLHED patients. When he administered ER004 in-utero to 3 babies (including Maarten & Linus, the babies in the picture), he could show that these patients were subsequently able to sweat normally. These results highlight the strong potential for this therapy, when administered during pregnancy, to address the most debilitating aspects of XLHED. See here for more information on Maarten and Linus.

Using Prof. Holm Schneider’s promising new in-utero approach, EspeRare is now restarting the development of this promesing therapy. Currently, the main hurdle to overcome is to find the right regulatory and funding path forward. Once those challenges are addressed, clinical development in Europe is set to restart as early 2018.

Importantly and in-line with our patient-centered approach, EspeRare is partnering with XLHED patient associations, such as the American organisation NFED, to better understand the patients needs and their expectations from a therapy. See here for news from NFED.

Together with our partners, we are working very hard to ensure a way forward for this ground-breaking treatment. We hope to be able to share more exciting news on this program soon!