Data published evaluates fibrodysplasia ossificans progressiva (FOP) natural disease progression
The Natural History Study (NHS) is the first protocol-specified, longitudinal
evaluation of FOP disease progression
PARIS, FRANCE, 08 June 2020 – Ipsen (Euronext: IPN; ADR: IPSEY), shared data from the company’s growing Rare Diseases Therapeutic Area, with the publication of eight abstracts in the Journal of the Endocrine Society. The data include a first-of-its kind study titled, “A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP): 12-Month Outcomes,” highlighting one-year data on the natural progression of FOP and the impact of heterotopic ossification (HO) on patients’ physical functioning over time.1 Ipsen and its partners also shared several studies detailing findings from health economics and outcomes research on the management of acromegaly.
FOP is an ultra-rare, severely disabling genetic bone disease that affects approximately 1.36 per million individuals worldwide.2,3 FOP is characterized by formation of bone in soft and connective tissues, known as heterotopic ossification (HO).4 Sporadic episodes of painful soft tissue swelling called ‘flare-ups’ can precede HO.4 HO is permanent, cumulative and severely impacts physical function over time with most patients’ movement becoming extremely limited, often confining them to a wheelchair by their twenties.4
The prospective Natural History Study (NHS) of FOP is the first global, multicenter, longitudinal study designed to measure disease progression over three years.1 Participant 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
The analysis showed that over the course of 12 months, 40% (37/93) of participants had new HO.1 Of the NHS participants with new HO, 65% (24/37) reported at least one flare-up (mean rate of 2.3 flare-ups/year).1 60% (56/93) of NHS participants did not experience new HO over 12 months.1 Of those participants, 43% (24/56) reported at least one flare-up (mean rate of 1.8 flare-ups/year).1 CAJIS and FOP-PFQ were not sufficiently sensitive to assess FOP disease progression over 12 months.
“The results show that evaluating HO may be a viable way to measure disease progression and potential treatment effect,” 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. “By 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.”
Additional abstracts published in the Journal of the Endocrine Society included data on the management of patients living with FOP or acromegaly.5-11
“Our research reinforces Ipsen’s 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,” said Jim Roach, M.D., Senior Vice President and Global Head, Rare Diseases Therapeutic Area at Ipsen. “These 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.”

Overview of Ipsen data accepted by ENDO 2020:

For further information:

References

1. Al Mukaddam M et al. A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP): 12-Month Outcomes. Journal of the Endocrine Society. 2020; DOI: https://doi.org/10.1210/jendso/bvaa046.254
2. Lilijesthrom M & Bogard B. The Global Known FOP Population. Presented at the FOP Drug Development Forum. Boston, MA; 2016.
3. Baujat et al. Prevalence of fibrodysplasia ossificans progressiva (FOP) in France: an estimate based on a record linkage of two national databases. Orphanet Journal of Rare Diseases. 2017; 12:123.
4. The Medical Management of Fibrodysplasia Ossificans Progressiva: Current Treatment Considerations, IFOPA. Accessed: May 2020. Available: http://fundacionfop.org.ar/wp-content/uploads/2019/05/GUIDELINES-May-2019.pdf
5. Kou S et al. Patients with Fibrodysplasia Ossificans Progressiva Have an Increased Prevalence of Cardiac Conduction Abnormalities. Journal of the Endocrine Society. 2020; DOI: https://doi.org/10.1210/jendso/bvaa046.238
6. Singh S et al. Surgical Management of Bilateral Hip Fracture in a Patient with Fibrodysplasia Ossificans Progressiva Treated with Palovarotene. Journal of the Endocrine Society. 2020; DOI: https://doi.org/10.1210/jendso/bvaa046.371
7. Yuen K et al. Acromegaly Significantly Impacts Employees’ Health Benefit Costs and Increases Work Absenteeism. Journal of the Endocrine Society. 2020; DOI: https://doi.org/10.1210/jendso/bvaa046.375
8. Ribeiro-Oliveira A et al. Healthcare Services Utilization and Costs Associated with the Management of Patients Living with acromegaly. Journal of the Endocrine Society. 2020; DOI: https://doi.org/10.1210/jendso/bvaa046.383
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. Journal of the Endocrine Society. 2020; DOI: https://doi.org/10.1210/jendso/bvaa046.423
10. Olsson D et al. Long-Acting SSA Treatment Patterns in Sweden From 2005 to 2017: A Nationwide Study. Journal of the Endocrine Society. 2020; DOI: https://doi.org/10.1210/jendso/bvaa046.818
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. Journal of the Endocrine Society. 2020; DOI: https://doi.org/10.1210/jendso/bvaa046.2163
12. Brue F et al. The risks of overlooking the diagnosis of secreting pituitary adenomas. Orphanet Journal of Rare Diseases. 2016;11:135–52
13. Somatuline Autogel 60mg SmPC. Accessed: May 2020. Available: https://www.medicines.org.uk/emc/product/4808/smpc

Attachment

• Ipsen-ENDO-2020-8-June-2020