- Impact on patient quality of life quantified including long-term renal and cardiovascular complications
- Final outcomes of 6 year study into continuous use of rhPTH(1-84) shows improvements in key measurements of mineral homeostasis
Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) this week shared new data revealing the burden of chronic hypoparathyroidism on patients and caregivers, as well as potential long-term risks of renal and cardiovascular complications that patients treated with conventional therapy may experience. Six-year results from the open-label long-term safety and efficacy RACE study were also announced. Data was presented at the Endocrine Society’s 2019 Annual Meeting (ENDO) from 23–26 March in New Orleans, Louisiana, USA.
“Results from the Burden of Illness Global Survey in 13 countries reveal the extent of the impact hypoparathyroidism has on patients and their caregivers throughout their daily lives,” said John Bilezikian M.D., Professor of Medicine at the Columbia University Irving Medical Center, New York, NY, USA. “This offers important insights and enables us to take a more holistic approach in the management of these patients due to a greater understanding of the potential cardiovascular risks patients may face, along with other complications of the kidneys,” he added.
“We are committed to further investigating this rare condition and collecting more data on the long-term implications,” said John Germak, Global Medical Team Lead at Takeda. “Gaining valuable insights directly from patients enhances our understanding of the complex nature of the disease and its management. The data presented at ENDO supports this cause.”
Three abstracts from the Burden of Illness Global Survey were presented for the first time at ENDO. The multi-country self-reported survey, the first large study of its kind, looked at physical and mental health components to characterise the burden and impact of the disease. Results showed substantial symptom burden and health-related quality of life impact in patients with chronic hypoparathyroidism not adequately controlled with conventional therapy, and their caregivers.1,2,3
New data on the risk of chronic kidney disease (CKD) and its progression were also presented alongside a study investigating estimated glomerular filtration rate (eGFR) decline in patients with chronic hypoparathyroidism. Furthermore, results from a new study looking at the risk of cardiovascular conditions in patients with chronic hypoparathyroidism – an area of limited prior research – were shared.4,5,6,7
Final results from the six-year RACE study, with efficacy endpoints of calcium dose reduction, reduction in calcitriol dose and normalization or maintaining serum calcium, reported data in key measurements of mineral homeostasis in chronic hypoparathyroidism patients treated with recombinant human parathyroid hormone rhPTH (1-84). In addition, results from this study reported a safety profile consistent with previous clinical trials results. 8
Takeda presented a total of eight abstracts at the congress further building on the company’s knowledge and experience of the disease.
All abstracts are available on the ENDO 2019 website at https://www.abstractsonline.com/pp8/#!/5752
About the Data
Burden of Illness Global Patient and Caregivers Survey1,2,3
The survey data showed patients experience substantial symptom burden including physical fatigue (97%), muscle cramps (86%), tingling (84%), brain fog (77%), anxiety (78%) and depression (76%). The magnitude of symptom severity correlated with the impact on the reduction in patients’ health-related quality of life.
Patients also reported an impact on their lifestyle through a symptom diary, showing an effect on sleep (78%), the ability to exercise (84%), ability to work (75%) as well as an impact on family relationships (63%). In turn, caregivers reported a major impact on their relationship with spouse/partner, family and friends.
Long-term Complications Associated with Chronic Hypoparathyroidism4,5,6,7,13
Four studies were presented. Results from a study investigating the risk of CKD and its progression, a retrospective controlled cohort study comparing risks in chronic and non-hypoparathyroidism patients, showed that patients with chronic hypoparathyroidism on conventional therapy had a significantly increased risk of developing CKD stage 3 and greater, as well as progression to end stage renal disease. In addition, eGFR – a key indicator of kidney function13 – was examined and an association with chronic hypoparathyroidism and increased rate of eGFR decline over time was observed in the subset of cohorts with laboratory data available. Moreover, hypoparathyroid patients had an increased risk of nephrolithiasis and nephrocalcinosis compared with matched controls of patients without hypoparathyroidism.
In another retrospective cohort study, to compare the risk of cardiovascular (CV) conditions in patients with chronic hypoparathyroidism and non-hypoparathyroidism patients, the database analysis showed that chronic hypoparathyroidism patients had an increased risk of experiencing a new occurrence of each of the cardiovascular conditions studied, as well as the composite cardiovascular endpoints of cerebrovascular disease, coronary artery disease, peripheral artery disease and heart failure, compared to patients without hypoparathyroidism.
These studies, presented for the first time at ENDO, employed a systematic way to examine risk associations between long-term complications and chronic hypoparathyroidism. Although considered hypothesis generating and further research is warranted, the findings provide valuable new data and additional learnings for this rare condition.
RACE 6-year Long-term Safety and Efficacy of rhPTH (1-84)8
Six-year results from the open-label RACE study presented at ENDO showed that treatment with rhPTH (1-84) in patients with chronic hypoparathyroidism had a safety profile consistent with previous clinical studies, and impacted key measurements of mineral homeostasis, notably of urinary calcium. Albumin-corrected serum calcium levels remained within the target range, stable urinary calcium excretion, serum phosphate, serum creatinine, and eGFR was observed and doses of oral calcium and calcitriol were reduced by >40% and >70% compared to baseline.
About Hypoparathyroidism
Hypoparathyroidism is a rare endocrine disease which occurs when inadequate levels of parathyroid hormone (PTH) are secreted by the parathyroid glands in the neck, resulting in a mineral imbalance in the body.9,10,11 Chronic hypoparathyroidism is diagnosed in patients with a low concentration of calcium in the blood and inappropriately low PTH levels; for postsurgical hypoparathyroidism, the features of hypoparathyroidism must persist for at least 6 months after surgery to be considered to be chronic.10,12 Chronic hypoparathyroidism can have a significant impact on patients’ health-related quality of life, as well as leading to serious long-term complications.
About NATPARA® (parathyroid hormone) for Injection in the U.S.
NATPARA, available as 25, 50, 75, and 100 mcg per dose strength, is a parathyroid hormone indicated as an adjunct to calcium and vitamin D to control hypocalcemia in patients with hypoparathyroidism.14 Because of the potential risk of osteosarcoma, NATPARA is recommended only for patients who cannot be well-controlled on calcium supplements and active forms of vitamin D alone. NATPARA was not studied in patients with hypoparathyroidism caused by calcium-sensing receptor mutations. NATPARA was not studied in patients with acute post-surgical hypoparathyroidism.
About NATPAR® for Injection in Europe
NATPARA is approved in Europe under the tradename NATPAR®. NATPAR is indicated as adjunctive treatment of adult patients with chronic hypoparathyroidism who cannot be adequately controlled with standard therapy alone.15 Takeda is authorised to market NATPAR in the 28 Member States of the European Union, as well as in Iceland, Liechtenstein and Norway. NATPAR is currently commercially available in Germany, Greece, Austria, Denmark and Norway.
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