RARE Daily

FDA Grants Fast Track to Amolyt’s Experimental Hypoparathyroidism Candidate

May 2, 2024

Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation to Amolyt Pharma’s lead therapeutic peptide candidate, eneboparatide, in phase 3 development for the treatment of hypoparathyroidism.

“We believe FDA’s granting of Fast Track designation to eneboparatide reflects the agency’s recognition of the significant unmet needs that persist among patients suffering from hypoparathyroidism,” stated Thierry Abribat, founder and CEO of Amolyt Pharma. “We look forward to maintaining a constructive dialog with the agency as we work to bring new hope to patients suffering from this rare but challenging endocrine disorder as efficiently as possible.”

Hypoparathyroidism is a rare endocrine condition defined by a deficiency of parathyroid hormone (PTH) that results in decreased calcium and elevated phosphorus levels in the blood. Approximately 80 percent of the estimated 80,000 people in the U.S. and 110,000 in the European Union with hypoparathyroidism are women. Despite available treatments, patients experience persistent, life-altering symptoms and often develop complications and comorbidities that diminish quality of life and create segments of the patient population with specific clinical needs. Clinical manifestations of hypoparathyroidism impact many tissues and organ systems, in particular, the kidneys and bone.

“The current standard of care treatment, oral calcium and vitamin D supplementation, seldom controls the life altering symptoms and complications of hypoparathyroidism, with many patients at risk of declining kidney function and diminished bone quality,” said Mark Sumeray, chief medical officer. “In studies to date, eneboparatide has been shown to normalize mean serum calcium and mean urinary calcium excretion while restoring balanced bone turnover. Building upon findings from our successful phase 2 clinical trial, we are working diligently to execute our ongoing CALYPSO phase 3 study and look forward to topline data in 2025.”

Eneboparatide is an investigational therapeutic peptide designed to bind with high affinity to a specific conformation of the parathyroid hormone receptor to produce sustained and stable levels of calcium in the blood and thereby manage the symptoms of hypoparathyroidism, and to limit urine calcium excretion by restoring calcium reabsorption by the kidney, with the goal of consequently preventing progressive decline in kidney function and the development of chronic kidney disease. Eneboparatide is also designed to have a short half-life to potentially preserve bone integrity, an important potential benefit, since the majority of patients are peri- and postmenopausal women with an increased risk of developing osteoporosis.

CALYPSO is a phase 3 multicenter, randomized, placebo-controlled, double-blind study designed to evaluate the efficacy and safety of eneboparatide in patients with chronic hypoparathyroidism. Approximately 165 patients treated with standard of care will be randomized in a 2:1 ratio to receive eneboparatide or placebo. The primary efficacy endpoint is the proportion of patients that achieve albumin-adjusted serum calcium within the normal range and independence from standard of care after 24 weeks of treatment.

The key secondary efficacy endpoints include normalization of 24-hour urinary calcium in patients with hypercalciuria at baseline and assessment of patient-reported outcomes that reflect symptoms associated with physical and cognitive function and impact on quality of life. Additional exploratory endpoints assess bone quantity and quality using DXA scanning and high resolution peripheral quantitative CT scanning. After the initial 24-week placebo-controlled period, all patients will be treated with eneboparatide in an open-label extension phase for an additional 28 weeks.

The FDA’s Fast Track process is designed to facilitate the development and expedite the review of new drugs to treat serious conditions with unmet medical needs, with the goal of introducing new treatment options to patients faster.

A drug that receives FDA Fast Track designation is eligible for more frequent meetings and written communication with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval, and eligibility for Accelerated Approval and Priority Review, if relevant criteria are met.

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