Ascendis Pharma A/S

Ascendis Pharma A/S works on 3 rare diseases tracked on Trial Friend, including Achondroplasia, Hypoparathyroidism, Turner Syndrome, with 10 active clinical trials (8 recruiting) and 3 FDA-approved rare disease drugs.

Ascendis Pharma is a biopharmaceutical company applying its TransCon technology platform to develop long-acting prodrug therapies for rare endocrine diseases. The company markets Skytrofa for pediatric growth hormone deficiency, Yorvipath for hypoparathyroidism, and Yuviwel for achondroplasia.

Type
Rare Disease Specialist
Ticker
ASND
Headquarters
Copenhagen, Denmark
Founded
2007
10
Active Rare Disease Trials
3
Approved Rare Disease Drugs
3
Rare Diseases in Portfolio
19
Years Active

Focus areas at Ascendis Pharma A/S

As a rare disease specialist, Ascendis Pharma A/S has active clinical trial programs and drug development efforts across 3 rare diseases, including Achondroplasia, Hypoparathyroidism, Turner Syndrome. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.

The clinical trials section below shows active and recruiting rare disease studies sponsored by Ascendis Pharma A/S, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.

Ascendis Pharma A/S is headquartered in Copenhagen, Denmark, founded in 2007, publicly traded under the ticker symbol ASND. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.

Ascendis Pharma A/S Drug Pipeline

Ascendis Pharma A/S has 10 active clinical trials across 3 development stages, with 8 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.

Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.

Understand Ascendis Pharma A/S's pipeline
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4
Phase 24 trials
Recruiting
Recruiting
Active Not Recruiting
Active Not Recruiting
3
Phase 33 trials
Recruiting
Recruiting
3
Other3 trials
Recruiting

Ascendis Pharma A/S Clinical Trials (10)

Active and recruiting clinical trials sponsored by Ascendis Pharma A/S, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.

Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.

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RECRUITINGRecently updatedNCT07857460

German Hypoparathyroidism Registry

HypoparathyroidismPseudohypoparathyroidism

Chronic hypoparathyroidism is a rare condition in which the parathyroid glands do not make enough parathyroid hormone (PTH). PTH helps control the levels of calcium and phosphate in the blood. When there is not enough PTH, calcium levels can become too low and phosphate levels can become too high. This may cause symptoms such as tingling, muscle cramps, weakness, or other health problems. The most common cause of chronic hypoparathyroidism is surgery in the neck area. It can happen after thyroid surgery, surgery on the parathyroid glands, or other operations in the neck. At the moment, most patients are treated with calcium and vitamin D tablets. These treatments help control calcium levels, but they cannot fully replace the normal effects of PTH in the body. A medicine that contains a form of human PTH is available for some patients, but it is not yet the standard treatment for everyone. This registry collects medical information and follows patients over time. Its aim is to better understand patients' symptoms, possible long-term health problems, treatments, and overall health. A group of patients who have had their thyroid removed but do not have hypoparathyroidism will also be included for comparison.

Ages 18 Years+1 location
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RECRUITINGPHASE3Recently updatedNCT07221851

Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders

Intervention: Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector

Turner SyndromeShort Stature Homeobox Gene MutationIdiopathic Short StatureSmall for Gestational Age at Delivery

This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and \<18 years with open growth plates. The purpose of the study is to see how well treatment with once-weekly lonapegsomatropin works compared to treatment with daily somatropin. Approximately 186 participants will be distributed equally (1:1), to receive either lonapegsomatropin for 2 years or somatropin for 1 year followed by lonapegsomatropin for 1 year. This trial will be conducted in the United States, France, Germany, Italy, Romania, Spain and South Korea.

Ages 2 Years - 17 Years51 locations
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RECRUITINGPHASE3Recently updatedNCT07081997

A Phase 3 Randomized Clinical Trial to Investigate the Safety and Efficacy of Palopegteriparatide at Doses Greater Than 30 μg/Day in Adult Participants With Hypoparathyroidism

Intervention: Palopegteriparatide Experimental Arm, Palopegteriparatide Control Arm

HypoparathyroidismEndocrine System DiseasesParathyroid Diseases

This trial has a treatment duration of 78 weeks and will include adult participants already on treatment with palopegteriparatide at doses at or greater than 30 mcg/day. All participants will receive subcutaneous palopegteriparatide during the trial and will be individually and progressively titrated to an optimal dose at pre-specified dose levels. The primary purpose of the trial is to provide additional evidence of treatment effect and safety of palopegteriparatide at doses greater than 30 mcg/day in adults with hypoparathyroidism. The trial will be conducted in the US.

Ages 18 Years+8 locations
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RECRUITINGRecently updatedNCT07264634

A Study to Assess the Amount of Palopegteriparatide in Breast Milk of Lactating Females Requiring YORVIPATH® (Palopegteriparatide)

Intervention: Palopegteriparatide

This is an observational, opportunistic lactation study to be conducted in lactating female participants who are currently receiving therapeutic doses of YORVIPATH as part of their usual care and who have chosen to breastfeed their infant(s). The potential transfer of palopegteriparatide into breast milk will be assessed.

Ages 18 Years - 50 Years1 location
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RECRUITINGPHASE3Recently updatedNCT07706764

A Study to Test the Effects and Safety of Palopegteriparatide in Adolescents With Long-term Hypoparathyroidism

Intervention: Palopegteriparatide

This trial will enroll adolescents between ages of ≥12 and \<18 years with clinically diagnosed hypoparathyroidism . The purpose of the study is to see how well treatment with once-daily palopegteriparatide works and how safe it is. At least 12 participants will receive palopegteriparatide for 234 weeks. This trial will be conducted in Europe.

Ages 12 Years - 18 Years3 locations
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RECRUITINGPHASE2Updated a few months agoNCT06732895

A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.

Intervention: Navepegritide, Placebo for navepegritide

The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia. What will be measured is Annualized Growth Velocity after a 52-week treatment period.

Ages 12 Years - 17 Years5 locations
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RECRUITINGPHASE2No updates in a whileNCT06079398

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

Intervention: Navepegritide, Placebo for Navepegritide

This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to \< 2 years at the time of randomization.

Ages 0 Years - 2 Years18 locations
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Ascendis Pharma A/S FDA-Approved Drugs (3)

Medications developed or marketed by Ascendis Pharma A/S that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.

Drug NameBrand NameRare DiseasesApproval Date
LONAPEGSOMATROPIN-TCGD
Recombinant Human Growth Hormone [EPC]
Skytrofa
subcutaneous
—Aug 25, 2021
PALOPEGTERIPARATIDEYorvipath
subcutaneous
Aug 9, 2024
NAVEPEGRITIDEYuviwelFeb 27, 2026

Ascendis Pharma A/S Trial Locations

Ascendis Pharma A/S clinical trials are running at 110 sites in 19 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.

United States
55▼
France
9▼
Germany
7▼
South Korea
7▼
Italy
6▼
Spain
5▼
Denmark
3▼
Ireland
3▼
United Kingdom
3▼
Romania
2▼
Canada
2▼
Poland
1▼

Rare Disease Focus Areas (3)

Diseases targeted by Ascendis Pharma A/S's clinical trial and drug development programs

Patient Resources

Organizations and resources related to Ascendis Pharma A/S's rare disease focus areas

Frequently Asked Questions About Ascendis Pharma A/S

Common questions about Ascendis Pharma A/S's rare disease programs, clinical trials, and treatments.