{"id":36162,"date":"2020-06-08T13:58:00","date_gmt":"2020-06-08T11:58:00","guid":{"rendered":"https:\/\/www.ipsen.com\/press-releases\/ipsen-demonstrates-continued-commitment-to-rare-diseases-with-eight-abstracts-accepted-at-endo-2020-published-in-the-journal-of-the-endocrine-society\/"},"modified":"2024-07-23T14:02:35","modified_gmt":"2024-07-23T12:02:35","slug":"ipsen-demonstrates-continued-commitment-to-rare-diseases-with-eight-abstracts-accepted-at-endo-2020-published-in-the-journal-of-the-endocrine-society","status":"publish","type":"press_release","link":"https:\/\/www.ipsen.com\/press-releases\/ipsen-demonstrates-continued-commitment-to-rare-diseases-with-eight-abstracts-accepted-at-endo-2020-published-in-the-journal-of-the-endocrine-society\/","title":{"rendered":"Ipsen demonstrates continued commitment to rare diseases with eight abstracts accepted at ENDO 2020 published in the Journal of the Endocrine Society"},"content":{"rendered":"

Data published evaluates fibrodysplasia ossificans progressiva (FOP) natural disease progression
\nThe Natural History Study (NHS) is the first protocol-specified, longitudinal
\nevaluation of FOP disease progression
\nPARIS, FRANCE, 08 June 2020 \u2013\u00a0<\/b>Ipsen (Euronext: IPN; ADR: IPSEY), shared data from the company\u2019s growing Rare Diseases Therapeutic Area, with the publication of eight abstracts in the\u00a0Journal of the Endocrine Society<\/em>. The data include a first-of-its kind study titled, \u201cA Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP): 12-Month Outcomes,\u201d highlighting one-year data on the natural progression of FOP and the impact of heterotopic ossification (HO) on patients\u2019 physical functioning over time.1\u00a0Ipsen and its partners also shared several studies detailing findings from health economics and outcomes research on the management of acromegaly.
\nFOP is an ultra-rare, severely disabling genetic bone disease that affects approximately 1.36 per million individuals worldwide.2,3\u00a0FOP is characterized by formation of bone in soft and connective tissues, known as heterotopic ossification (HO).4\u00a0Sporadic episodes of painful soft tissue swelling called \u2018flare-ups\u2019 can precede HO.4\u00a0HO is permanent, cumulative and severely impacts physical function over time with most patients\u2019 movement becoming extremely limited, often confining them to a wheelchair by their twenties.4
\nThe prospective Natural History Study (NHS) of FOP is the first global, multicenter, longitudinal study designed to measure disease progression over three years.1\u00a0Participant selection was representative of the FOP community worldwide and includes 114 patients with FOP who harbor a documented ACVR1 mutation, of those 99 (55 male \/ 44 female; mean age of group: 17.4 years) had both baseline and 12-month assessments and were included in the analysis. Ninety-three of the 99 participants had evaluable whole-body computed tomography and were included in the HO analysis.1
\nThe analysis showed that over the course of 12 months, 40% (37\/93) of participants had new HO.1\u00a0Of the NHS participants with new HO, 65% (24\/37) reported at least one flare-up (mean rate of 2.3 flare-ups\/year).1\u00a060% (56\/93) of NHS participants did not experience new HO over 12 months.1\u00a0Of those participants, 43% (24\/56) reported at least one flare-up (mean rate of 1.8 flare-ups\/year).1\u00a0CAJIS and FOP-PFQ were not sufficiently sensitive to assess FOP disease progression over 12 months.
\n\u201cThe results show that evaluating HO may be a viable way to measure disease progression and potential treatment effect,\u201d said study co-investigator and co-author, Mona Al Mukaddam, M.D., MS of the Center for Research in FOP and Related Disorders, Perelman School of Medicine, University of Pennsylvania. \u201cBy examining the natural course of FOP, we hope to better understand the physical burden of HO as well as the link between flare-ups and disease progression.\u201d
\nAdditional abstracts published in the\u00a0Journal of the Endocrine Society<\/em>\u00a0included data on the management of patients living with FOP or acromegaly.5-11
\n\u201cOur research reinforces Ipsen\u2019s commitment to actively listen to and work with patients, physicians, nurses, patient association groups, and other key stakeholders to best address the complexity and challenges of living with rare diseases such as FOP or acromegaly,\u201d said Jim Roach, M.D., Senior Vice President and Global Head, Rare Diseases Therapeutic Area at Ipsen. \u201cThese diseases impact the quality of life both for patients and their caregivers and often place economic and societal burdens due to absences from work and high healthcare costs. Enhancing our understanding of these diseases will hopefully allow us to develop solutions that meaningfully impact patients.\u201d<\/p>\n

Overview of Ipsen data accepted by ENDO 2020:<\/b><\/p>\n\n\n\n\n\n\n\n\n\n\n
Medicine\/Disease<\/b><\/p>\n

\u00a0<\/b><\/td>\n

Abstract Number\/Title<\/b><\/td>\nStudy Authors<\/b><\/td>\n<\/tr>\n
FOP<\/td>\nOR29-05 \u2013\u00a0A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP): 12-Month Outcomes<\/b>1<\/b><\/a><\/td>\nMona Al Mukaddam, Robert J. Pignolo, Genevi\u00e8ve Baujat, Matthew A. Brown, Carmen De Cunto, Maja Di Rocco, Edward C. Hsiao, Richard W. Keen, Kim-Hanh Le Quan Sang, Andrew Strahs, Rose Marino, and Frederick S. Kaplan<\/td>\n<\/tr>\n
SUN-344 \u2013\u00a0Patients with Fibrodysplasia Ossificans Progressiva Have an Increased Prevalence of Cardiac Conduction Abnormalities<\/b>5<\/b><\/a><\/td>\nSamuel Kou, Carmen De Cunto, Genevi\u00e8ve Baujat, Kelly L. Wentworth, Donna Grogan, Matthew A. Brown, Maja Di Rocco, Richard Keen, Mona Al Mukaddam, Frederick S. Kaplan, Robert J. Pignolo, and Edward C. Hsiao<\/td>\n<\/tr>\n
Palovarotene<\/td>\nMON-348 \u2013\u00a0Surgical Management of Bilateral Hip Fracture in a Patient with Fibrodysplasia Ossificans Progressiva Treated with Palovarotene<\/b>6<\/b><\/a><\/td>\nSukhmani Singh, Joseph Kidane, Kelly L. Wentworth, Daria Motamedi, Saam Morshed, and Edward C. Hsiao<\/td>\n<\/tr>\n
Acromegaly<\/td>\nSUN-296 \u2013\u00a0Acromegaly Significantly Impacts Employees\u2019 Health Benefit Costs and Increases Work Absenteeism<\/b>7<\/b><\/a><\/td>\nKevin C.J. Yuen, Kathryn A. Munoz, Richard Alan Brook, John D. Whalen, Ian A. Beren, and Antonio Ribeiro-Oliveira<\/td>\n<\/tr>\n
MON-141 \u2013\u00a0Healthcare Services Utilization and Costs Associated with the Management of Patients Living with Acromegaly<\/b>8<\/b><\/a><\/td>\nAntonio Ribeiro-Oliveira, Kathryn A. Munoz, Richard Alan Brook, Ian A. Beren, John D. Whalen, and Kevin C.J. Yuen<\/td>\n<\/tr>\n
MON-306 \u2013\u00a0Acromegaly Comorbidity Costs, Quality of Life, and Mortality: Lifetime Comparisons for Controlled Acromegaly, Uncontrolled Acromegaly, and the General US Population<\/b>9<\/b><\/a><\/td>\nMelanie D. Whittington, Jonathan D. Campbell, R. Brett McQueen, Kathryn A. Munoz, John D. Whalen, and Antonio Ribeiro-Oliveira<\/td>\n<\/tr>\n
Somatuline\u00ae\u00a0Autogel\/Depot<\/td>\nMON-301 \u2013\u00a0Long-Acting SSA Treatment Patterns in Sweden From 2005 to 2017: A Nationwide Study<\/b>10<\/b><\/a><\/td>\nDaniel S. Olsson, Daniel Granfeldt, \u00c5se Bj\u00f6rstad, Antonio Ribeiro-Oliveira, Anna Jonasson, John D. Whalen<\/td>\n<\/tr>\n
MON-LB47 \u2013\u00a0An International Simulated Use Study (PRESTO) to Evaluate Nurse Preferences Between the Lanreotide Autogel New Syringe and Octreotide Long-Acting Release Syringe<\/b>11<\/b><\/a><\/td>\nDaphne T. Adelman, Xuan-Mai Truong-Thanh, Marion Feuilly, Aude Houchard, and David Cella<\/td>\n<\/tr>\n<\/table>\n

For further information:<\/u><\/b><\/p>\n\n\n\n\n\n
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Media<\/b>
\nChristian Marcoux, M.Sc.
\nSenior Vice President, Global Communications
\n+33 (0)1 58 33 67 94
\nchristian.marcoux@ipsen.com<\/b><\/a><\/p>\n

Financial Community<\/b>
\nEugenia Litz
\nVice President, Investor Relations
\n+44 (0) 1753 627721
\neugenia.litz@ipsen.com<\/b><\/a><\/td>\n

Kelly Blaney
\nVice President, Global Communications
\n+44 (0) 7903 402275
\nkelly.blaney@ipsen.com<\/b><\/a><\/p>\n

\u00a0<\/u><\/p>\n

Myriam Koutchinsky
\nInvestor Relations Manager
\n+33 (0)1 58 33 51 04
\nmyriam.koutchinsky@ipsen.com<\/b><\/a><\/td>\n

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References<\/b><\/p>\n

    \n
  1. Al Mukaddam M et al. A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP): 12-Month Outcomes.\u00a0Journal of the Endocrine Society.\u00a0<\/em>2020; DOI:\u00a0https:\/\/doi.org\/10.1210\/jendso\/bvaa046.254<\/b><\/a><\/li>\n
  2. Lilijesthrom M & Bogard B. The Global Known FOP Population. Presented at the FOP Drug Development Forum. Boston, MA; 2016.<\/li>\n
  3. Baujat et al. Prevalence of fibrodysplasia ossificans progressiva (FOP) in France: an estimate based on a record linkage of two national databases.\u00a0Orphanet Journal of Rare Diseases.<\/em>\u00a02017; 12:123.<\/li>\n
  4. The Medical Management of Fibrodysplasia Ossificans Progressiva: Current Treatment Considerations, IFOPA. Accessed: May 2020. Available:\u00a0http:\/\/fundacionfop.org.ar\/wp-content\/uploads\/2019\/05\/GUIDELINES-May-2019.pdf<\/b><\/a><\/li>\n
  5. Kou S et al. Patients with Fibrodysplasia Ossificans Progressiva Have an Increased Prevalence of Cardiac Conduction Abnormalities.\u00a0Journal of the Endocrine Society<\/em>. 2020; DOI:\u00a0https:\/\/doi.org\/10.1210\/jendso\/bvaa046.238<\/b><\/a><\/li>\n
  6. Singh S et al. Surgical Management of Bilateral Hip Fracture in a Patient with Fibrodysplasia Ossificans Progressiva Treated with Palovarotene.\u00a0Journal of the Endocrine Society<\/em>. 2020; DOI:\u00a0https:\/\/doi.org\/10.1210\/jendso\/bvaa046.371<\/b><\/a><\/li>\n
  7. Yuen K et al. Acromegaly Significantly Impacts Employees\u2019 Health Benefit Costs and Increases Work Absenteeism.\u00a0Journal of the Endocrine Society<\/em>. 2020; DOI:\u00a0https:\/\/doi.org\/10.1210\/jendso\/bvaa046.375<\/b><\/a><\/li>\n
  8. Ribeiro-Oliveira A et al. Healthcare Services Utilization and Costs Associated with the Management of Patients Living with acromegaly.\u00a0Journal of the Endocrine Society<\/em>. 2020; DOI:\u00a0https:\/\/doi.org\/10.1210\/jendso\/bvaa046.383<\/b><\/a><\/li>\n
  9. Whittington M et al. Acromegaly Comorbidity Costs, Quality of Life, and Mortality: Lifetime Comparisons for Controlled Acromegaly, Uncontrolled Acromegaly, and the General US Population.\u00a0Journal of the Endocrine Society<\/em>. 2020; DOI:\u00a0https:\/\/doi.org\/10.1210\/jendso\/bvaa046.423<\/b><\/a><\/li>\n
  10. Olsson D et al. Long-Acting SSA Treatment Patterns in Sweden From 2005 to 2017: A Nationwide Study.\u00a0Journal of the Endocrine Society<\/em>. 2020; DOI:\u00a0https:\/\/doi.org\/10.1210\/jendso\/bvaa046.818<\/b><\/a><\/li>\n
  11. Adelman D et al. An International Simulated Use Study (PRESTO) to Evaluate Nurse Preferences Between the Lanreotide Autogel New Syringe and Octreotide Long-Acting Release Syringe.\u00a0Journal of the Endocrine Society<\/em>. 2020; DOI:\u00a0https:\/\/doi.org\/10.1210\/jendso\/bvaa046.2163<\/b><\/a><\/li>\n
  12. Brue F et al. The risks of overlooking the diagnosis of secreting pituitary adenomas.\u00a0Orphanet Journal of Rare Diseases.<\/em>\u00a02016;11:135\u201352<\/li>\n
  13. Somatuline Autogel 60mg SmPC. Accessed: May 2020. Available:\u00a0https:\/\/www.medicines.org.uk\/emc\/product\/4808\/smpc<\/b><\/a><\/li>\n<\/ol>\n

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