Log in
Forgot password ?
Become a member for free
Sign up
Sign up
New member
Sign up for FREE
New customer
Discover our services
Dynamic quotes 
  1. Homepage
  2. Equities
  3. France
  4. Euronext Paris
  5. Ipsen
  6. News
  7. Summary
    IPN   FR0010259150


SummaryMost relevantAll NewsAnalyst Reco.Other languagesPress ReleasesOfficial PublicationsSector newsMarketScreener Strategies

Ipsen : What working in rare disease has taught me about supporting better FOP clinical practice

04/27/2021 | 10:19am EDT

By Dr Rolf Morhart

My journey to help make an impact

I first became interested in pediatrics during a 3-year stay abroad in a South African hospital in the late 1970s. It was there that I realized how much I value the open nature of young people and it was this experience that inspired me to devote my career to this area of medicine. So, when I returned to Germany, I started to train as a pediatrician and eventually took over the position of Chief Physician in adolescent and children's medicine at Garmisch-Partenkirchen Clinic1. My career then evolved to working within the rare disease field, with a particular focus on the ultra-rare disease Fibrodysplasia Ossificans Progressiva (FOP) which through a genetic mutation (ALK2/ACVR1) leads to new bone formation where bone typically does not form or should not exist including in muscle and connective tissue2. This progressively restricts movement and most patients become immobilized and confined to a wheelchair by their third decade of life, requiring lifelong assistance for daily activities.

Although in recent years progress has been made, there are still a lot of unknowns in FOP. As soon as I became aware of the disease, I was determined to help make a positive difference and together with the International Clinical Council (ICC) on FOP, we have helped consolidate best practices, via a global voice, for clinical care and clinical research for those living with FOP3. But there is still more to be done. In light of FOP Awareness Day, and the positive progress made in this field, I would like to share some learnings from my experience of working in this area.

Access to the right professionals is key

It is estimated that 400 million people worldwide are affected by one of 6,000-8,000 rare diseases4, but treatments are even rarer with only a few hundred existing. FOP is an ultra-rare disease affecting 1.36 per million people worldwide, which when compared to many other diseases, is lacking in knowledge and information, even within the teams who work in the rare disease space. Throughout my career of working in FOP I recognize a specific expertise is required to guide our thought-process and help us make the best decision for the person and their unique experience living with FOP. This expertise goes beyond clinical and scientific knowledge but is about listening to the person, their experiences and challenges and how you can support with not just the management of their FOP as it progresses, but also where we can help in other ways where their life is impacted by their condition.

The rarity of diseases like FOP pose a challenge for accessing the right level of care, for example those living with FOP in Germany frequently don't have an expert in their area, so they are forced to travel in order to get an evaluation and proper management of their disease with the appropriate clinician. Accessing clinical teams with the right level of expertise is critical to all people living with rare diseases, but is of paramount importance in FOP to prevent harm or trigger disease progression which can be caused by routine diagnostic tests including biopsies, inappropriate therapies like operations to remove heterotopic bone or invasive medical interventions. On a practical level people living with FOP should avoid injections and surgery as this can trigger a flare-up of their disease and increase the chances of muscle/connective tissue turning to bone.

As we are becoming more active in teaching in this field, we are hoping to reduce this gap, and create more opportunities for those living with FOP to find the right healthcare professionals (HCPs), ideally someone locally, to support them through their diagnosis and beyond. I feel proud to have had the opportunity to meet and work with many inspirational colleagues and leaders in the advocacy community across my career, to increase awareness and education for FOP.

There has been great progress, but we need to continue pushing new research and screening standard of care

Although some challenges are yet to be overcome, there has been major progress in the rare disease field in the last 30 years due to medical improvements, such as genetic disease assessment. For FOP in particular, a key development was the discovery of the mutation in the FOP gene, ALK2/ACVR1. This scientific achievement made it possible to facilitate greater research and fill the gap in how and why new bone was being formed outside of the human skeleton. This has enabled researchers and others in the field to undertake more work, increasing our knowledge through further research and clinical trials exploring potential therapeutic options.

On a national level, we are also starting to see countries prioritize rare disease care and incorporate more specific assessments as part of their standard of care. Within the last month Brazil has introduced newborn screening as part of the newborn assessment to look for the sign of unusually shaped big toes at birth, a sign of FOP. This will certainly have a huge impact on FOP management in Brazil. Incorporating screening protocols like this in our countries will make important strides in earlier diagnosis and widespread recognition across healthcare teams.

Support is vital

While increased clinical research and overall disease awareness/education are vital for those living with rare conditions like FOP, it is not the only form of support that is needed. Family support is of upmost importance and can range from day-to-day challenges to key milestones like diagnosis and treatment.

Currently, there are several institutions in the field dedicated to supporting those living with FOP and rare diseases in general; patient organization groups like International Fibrodysplasia Ossificans Progressiva Association (IFOPA) have been invaluable in their support to the FOP ecosystem. In my opinion the IFOPA is a true leader in rare diseases worldwide for their work supporting those living with FOP. In addition to raising funds, supporting research, holding regular webinars on range of topics (such as mental health or COVID-19's impact on FOP), they also offer many useful resources to help individuals learn about FOP and are key in informing treatment guidelines5. They are making a strong and positive impact through their work. I truly hope their approach can be multiplied and spread across different healthcare fields and rare disease areas as the support patient organizations provide is invaluable.

What I would like up-and-coming HCPs to know

Support depends on more than one factor (family, patient organization and HCPs) - it is crucial we, HCPs, play our part in making a difference in the lives of those living with rare diseases like FOP. You may not encounter many people living with rare diseases in your clinic across your career, but no matter how rare the scenario or unique the case, the knowledge of these diseases shouldn't be.


Ipsen SA published this content on 27 April 2021 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 27 April 2021 14:18:04 UTC.

© Publicnow 2021
All news about IPSEN
04:30aIPSEN  : launches Perspectives Matter around World Kidney Cance...
06/14ISPRM 2021 : New Ipsen analysis highlights potential treatment gap in adults liv..
06/11IPSEN  : Finds Potential Treatment Gap Among Spasticity Patients in US
06/11IPSEN  : ISPMR 2021 – 11 June 2021.pdf
06/07IPSEN  : Exelixis Say Phase 3 Trial of Cabozantinib Drug in Differentiated Thyro..
06/07IPSEN  : Exelixis COSMIC-311 ASCO – 7 June 2021.pdf
06/05IPSEN  : World Environment Day 2021
05/31TODAY'S ANALYST RECOMMENDATIONS : Advanced Micro Devices, AstraZeneca, Bodycote,..
05/31IPSEN  : US FDA Accepts Ipsen's Approval Request For Ultra-Rare Genetic Disorder..
05/31IPSEN : Ex-dividend day for final dividend
More news
Sales 2021 2 737 M 3 259 M 3 259 M
Net income 2021 542 M 645 M 645 M
Net Debt 2021 83,7 M 99,7 M 99,7 M
P/E ratio 2021 13,8x
Yield 2021 1,13%
Capitalization 7 469 M 8 899 M 8 892 M
EV / Sales 2021 2,76x
EV / Sales 2022 2,62x
Nbr of Employees 5 703
Free-Float 41,9%
Duration : Period :
Ipsen Technical Analysis Chart | MarketScreener
Full-screen chart
Technical analysis trends IPSEN
Short TermMid-TermLong Term
Income Statement Evolution
Mean consensus OUTPERFORM
Number of Analysts 17
Average target price 91,03 €
Last Close Price 90,30 €
Spread / Highest target 25,1%
Spread / Average Target 0,81%
Spread / Lowest Target -16,9%
EPS Revisions
Managers and Directors
David G. Loew Chief Executive Officer & Director
Aymeric Le Chatelier Chief Financial Officer & Executive Vice President
Marc M. P. de Garidel Non-Executive Chairman
Aidan Murphy Executive Vice President-Technical Operations
Howard Mayer Executive Vice President-Research & Development
Sector and Competitors
1st jan.Capitalization (M$)
IPSEN33.52%9 087
JOHNSON & JOHNSON4.48%433 009
ROCHE HOLDING AG12.33%333 736
PFIZER, INC.6.76%219 989
NOVARTIS AG1.83%212 691
ABBVIE INC.7.82%204 052