PUBLISHER: DelveInsight | PRODUCT CODE: 1376846
PUBLISHER: DelveInsight | PRODUCT CODE: 1376846
Various key players are currently investigating their drugs for hypoparathyroidism, such as Amolyt Pharma, Ascendis Pharma, Calcilytix Therapeutics (a subsidiary of BridgeBio), and others. The details of the country and therapy-wise market size have been provided below.
The Hypoparathyroidism report's drugs section includes an in-depth examination of marketed drugs and late-stage pipeline therapeutics (Phase III and Phase II) for hypoparathyroidism.
The drug chapters section contains useful information on various aspects of hypoparathyroidism clinical trials, including specific details such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. It also includes the most recent news updates and press releases on drugs that treat hypoparathyroidism.
TRANSCON PTH is an investigational prodrug of parathyroid hormone (PTH) and is under development as a once-daily hormone replacement therapy. It is designed to restore physiologic levels of PTH for 24 h each day. The company aims to provide a PTH replacement therapy that normalizes serum and urinary calcium and serum phosphate levels, addressing both the short-term symptoms and long-term complications of hypoparathyroidism and improving the quality of life of its patients.
Encaleret is an investigational small molecule antagonist of the calcium-sensing receptor (CaSR) studied in calcium homeostasis disorders, including autosomal dominant hypocalcemia type 1 (ADH1). Individuals with ADH1 have gain-of-function mutations in the CaSR, causing low serum calcium and a range of debilitating symptoms. ADH1 may also lead to relatively high calcium levels in the urine, a condition called hypercalciuria, which can impair kidney function and cause kidney stone formation. The drug is a potential first-in-class CaSR antagonist for ADH1, which was given Fast Track Designation by the US FDA in June 2021.
Natpara is a bio-engineered replica of the parathyroid hormone, which raises serum calcium by increasing renal tubular calcium reabsorption, intestinal calcium absorption, and bone turnover, which then releases calcium into the circulation. It was indicated as an adjunct to calcium and vitamin D to control hypocalcemia in patients with hypoparathyroidism, but in October 2022, Takeda decided to discontinue manufacturing NATPAR/NATPARA injection globally by the end of 2024 due to unresolved supply issues that were specific to the product.
Hypoparathyroidism is a rare condition in which the parathyroid glands fail to produce sufficient amounts of parathyroid hormone or the parathyroid hormone produced lacks biological activity. The treatment of hypoparathyroidism is directed toward the specific symptoms apparent in each individual and the lab tests. Treatment aims to raise calcium levels to relieve symptoms without causing abnormally high calcium levels in the blood (hypercalcemia) or the urine (hypercalciuria). The specific therapies used may vary depending on the disease severity, the specific symptoms present, an individual's age and overall health, personal preference, and additional factors.
Oral calcium supplements can increase calcium levels in the blood. However, calcium supplements can cause gastrointestinal side effects at high doses, such as constipation, in some people. Several different types of calcium supplements are available; some brands may work better for certain people. High doses of vitamin D, generally calcitriol, can help the body absorb calcium and eliminate phosphorus. Another form of vitamin D that may be used is ergocalciferol or cholecalciferol; outside the US, doctors use alpha calcidol. Ergocalciferol and cholecalciferol have a longer duration of action than calcitriol or alpha calcidol because the former two forms of vitamin D are stored in the body for a long time.
If the magnesium level is low and the patient experiences symptoms of hypoparathyroidism, the doctor may recommend taking a magnesium supplement. If the calcium levels remain low even with treatment, thiazide diuretics can help decrease the amount of calcium lost through the urine. However, some people with hypoparathyroidism, including people who inherited the condition, should not take thiazide diuretics.
Some individuals, especially those with severe symptoms due to low blood calcium levels, may require immediate relief through intravenous calcium therapy, even if their calcium levels are only mildly reduced. Intravenous therapy means a substance (e.g., calcium) is delivered into the bloodstream through an injection or infusion directly into a vein.
Recently, in January 2015, the US FDA approved the use of recombinant human parathyroid hormone (1-84) (rhPTH[1-84]), NATPARA, as a treatment for adult patients with chronic hypoparathyroidism who are uncontrolled with conventional therapy (calcium and activated vitamin D). However, this therapy was later discontinued due to a manufacturing defect.
Hypoparathyroidism is a rare condition in which the parathyroid glands fail to produce sufficient amounts of parathyroid hormone or lack biological activity. The secretion of the parathyroid hormone is inversely related to the concentration of ionized calcium in the extracellular fluid. The activity of the calcium-sensing receptor (CaSR), a G-protein coupled receptor, is affected by calcium concentration changes. As the calcium concentration in the extracellular fluid increases, this receptor is activated, and parathyroid cells decrease the secretion of parathyroid hormone. Conversely, the activity of the CaSR decreases, and parathyroid hormone secretion increases as calcium levels decline.
Parathyroid hormone (along with vitamin D and calcitonin, produced by the thyroid gland) plays a role in regulating the levels of calcium and phosphorus in the blood and in determining bone growth and bone cell activity. Due to a parathyroid hormone deficiency, individuals may exhibit abnormally low levels of calcium in the blood (hypocalcemia) and high levels of phosphorus (hyperphosphatemia).
The diagnostic biochemical hallmark of hypoparathyroidism is hypocalcemia in association with deficient production of PTH. It is thus readily distinguished from pseudohypoparathyroidism, a genetic disorder of PTH resistance in which the circulating PTH concentration is elevated. The diagnosis of hypoparathyroidism is also readily distinguished from secondary causes of hypocalcemia (e.g., vitamin D deficiency), in which the PTH level is also high. In hypoparathyroidism, circulating active vitamin D (1,25-dihydroxyvitamin D3) and bone turnover markers are usually in the lower normal range.
Unlike most other hormonal deficits, hypoparathyroidism is not treated by replacing the missing parathyroid hormone (PTH). The standard treatment consists of activated vitamin D (calcitriol) and calcium supplements; some may also need magnesium supplementation. Conventional therapy requires many pills to be taken throughout the day.
Diet recommendations usually include eating foods high in calcium, such as dairy products, breakfast cereals, fortified orange juice, and green, leafy vegetables, or avoiding foods high in phosphorus, such as meat, poultry, fish, nuts, whole grains, and beans. Conventional therapy with vitamin D and calcium may lead to calcium buildup in the kidneys. This buildup may lead to problems, including kidney stones and deposits of calcium, reduced kidney function, tissue damage, or even kidney failure.
Further details related to treatment and management are provided in the report…..
The Hypoparathyroidism epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Total Prevalent Cases of Hypoparathyroidism, Total Diagnosed Prevalent Cases of Hypoparathyroidism, Gender-specific Cases of Hypoparathyroidism, Age-specific Cases of Hypoparathyroidism, Type-specific Cases of Hypoparathyroidism, and Cause-specific Cases of Hypoparathyroidism, covering the United States, EU4 countries (Germany, France, Italy, Spain) and the United Kingdom, and Japan from 2019 to 2032.
In order to stay abreast of the latest trends in the market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research.
We have reached out to industry experts to gather insights on various aspects of hypoparathyroidism, including the evolving treatment landscape, patients' reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts included medical/scientific writers, professors, and researchers from prestigious universities in the US, Europe, the UK, and Japan.
Our team of analysts at DelveInsight connected with more than 10 KOLs across the 7 Major Markets (7MM). We contacted institutions such as Oregon Health & Science University and others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the hypoparathyroidism market, which will assist our clients in analyzing the overall epidemiology and market scenario.
We conduct qualitative and market intelligence analysis employing various methods, including SWOT analysis and Conjoint Analysis. Strengths, weaknesses, opportunities, and threats in disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are described in the SWOT analysis. These recommendations are based on the Analyst's evaluation of the patient burden, cost analysis, and the current and emerging therapy landscape. Conjoint Analysis compares the effectiveness and safety of numerous approved and emergent drugs depending on key criteria such as frequency of administration, designation, route of administration, and order of entry. To assess the success of therapy, several factors are evaluated.
Furthermore, the drug's safety is analyzed, in which acceptability, tolerability, and adverse events are closely monitored, and it establishes a firm grasp of the side effects of the drugs used in the trials. Furthermore, for each therapy, the rating is based on the route of administration, sequence of entrance and designation, chance of success, and addressable patient pool. These characteristics determine the ultimate weightage score and ranking of developing therapeutics.
Because newly authorized drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.
The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.