This section highlights FDA-related milestones and regulatory updates for drugs developed by Ascendis Pharma A/S (ASND).
Over the past two years, Ascendis Pharma A/S has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
TransCon, TransCon, TransCon, SKYTROFA®, YORVIPATH, and TransCon. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
TransCon CNP FDA Regulatory Timeline and Events
TransCon CNP is a drug developed by Ascendis Pharma A/S for the following indication: In Children with Achondroplasia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- TransCon CNP
- Announced Date:
- May 6, 2026
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma A/S announced new data from a subgroup analysis showing that children with achondroplasia ≥5 years of age at enrollment treated with once-weekly TransCon CNP (navepegritide) in its pivotal ApproaCH Trial demonstrated significantly greater annualized growth velocity (AGV) compared to placebo at Week 52, and sustained these growth improvements through up to two years of treatment.
AI Summary
Ascendis Pharma reported subgroup results from the pivotal ApproaCH Trial showing that children with achondroplasia who were at least 5 years old at enrollment and treated once weekly with TransCon CNP (navepegritide) had significantly greater annualized growth velocity (AGV) than placebo at Week 52. These larger growth gains were sustained through up to two years of treatment, indicating a durable benefit for this age group in the trial.
Safety in the ≥5 subgroup was similar to the overall study population: a low rate of injection-site reactions (all mild), no symptomatic hypotension, and no acceleration of bone age. Most adverse events were mild or moderate, and none led to treatment discontinuation or withdrawal. A slide presentation with the detailed data is available on Ascendis Pharma’s Investor Relations website. These results strengthen the evidence for TransCon CNP as a treatment option to improve growth outcomes in school-age children with achondroplasia.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- April 6, 2026
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma A/S announced that YUVIWEL (navepegritide; developed as TransCon® CNP) was granted orphan drug exclusivity by the U.S. Food & Drug Administration (FDA) and that YUVIWEL is now commercially available in the United States.
AI Summary
Ascendis Pharma announced that YUVIWEL (navepegritide; TransCon CNP) was granted orphan drug exclusivity by the U.S. Food & Drug Administration to increase linear growth in pediatric patients 2 years and older with achondroplasia who have open growth plates. YUVIWEL is now commercially available in the United States, and revenue has been recognized after treatment began for initial patients.
YUVIWEL is a prescription medicine approved under accelerated approval based on improved annualized growth velocity; continued approval may depend on confirmatory trials showing clinical benefit. Serious side effects can include low blood pressure; common side effects include injection site reactions such as redness, itching, swelling, bruising, pain, and blistering. Caregivers should tell the child’s healthcare provider about medical conditions, medications, pregnancy or breastfeeding plans, and kidney problems before starting treatment.
Ascendis leaders and a parent in the achondroplasia community expressed hope that this once-weekly option will help children gain growth and confidence. Please see full U.S. Prescribing Information for details.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- March 16, 2026
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma announced new data from its pivotal ApproaCH Trial showing that children with achondroplasia treated with once-weekly TransCon CNP (navepegritide) maintained consistent improvements in growth through Week 104, with further improvement in body proportionality during the second year of treatment.
AI Summary
Ascendis Pharma reported Week 104 data from its pivotal ApproaCH Trial showing that children with achondroplasia treated once weekly with TransCon CNP (navepegritide) maintained consistent improvements in linear growth through two years of treatment. The company also reported further improvement in body proportionality during the second year, suggesting the therapy may help not only height but also more balanced skeletal proportions over time.
The ApproaCH Trial was a randomized, double‑blind, placebo‑controlled study in 84 children ages 2–11, using TransCon CNP 100 µg/kg once weekly versus placebo for 52 weeks, followed by an open‑label extension in which all participants received TransCon CNP through Week 104. These results support a sustained growth effect with continued treatment and indicate additional gains in body proportionality during the second year.Read Announcement
- Drug:
- TransCon CNP
- Announced Date:
- February 27, 2026
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma A/S announced that the U.S. Food & Drug Administration (FDA) has granted approval under the FDA's Accelerated Approval Program for YUVIWEL® (navepegritide; developed as TransCon® CNP), the first and only once-weekly treatment indicated to increase linear growth in children 2 years of age and older with achondroplasia with open epiphyses and the only one to provide continuous systemic exposure to CNP over the weekly dosing interval.
AI Summary
Ascendis Pharma announced the FDA has approved YUVIWEL (navepegritide; TransCon CNP) under the Accelerated Approval Program. YUVIWEL is the first and only once‑weekly treatment approved to increase linear growth in children aged 2 years and older with achondroplasia and open growth plates. As a once‑weekly prodrug of C‑type natriuretic peptide (CNP), it is designed to provide continuous systemic exposure of active CNP across the weekly dosing interval to counteract overactive FGFR3 signaling.
The FDA’s approval was based on a clinical package including three randomized, double‑blind, placebo‑controlled trials and up to three years of open‑label extension data; the pivotal ApproaCH trial is reported in JAMA Pediatrics. Ascendis expects U.S. availability in early Q2 2026 and will offer patient support and financial assistance. YUVIWEL’s accelerated approval is based on improved annual growth velocity and may require confirmatory trials for continued approval. Common risks include injection‑site reactions and possible low blood pressure.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- November 25, 2025
- Target Action Date:
- February 28, 2026
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma announced that Prescription Drug User Fee Act (PDUFA) goal date extended by three months to February 28, 2026
AI Summary
Ascendis Pharma said the U.S. Food and Drug Administration notified the company that information submitted on November 5, 2025, related to a post‑marketing requirement for TransCon CNP (navepegritide) in children with achondroplasia, was considered a major amendment to the New Drug Application. Because of that designation, the FDA extended the Prescription Drug User Fee Act (PDUFA) target action date by three months, moving it to February 28, 2026.
Ascendis said it has responded to all outstanding FDA requests, including a revised protocol for the post‑marketing study that was the primary topic at its late‑cycle meeting. The company added it will work closely with the FDA to finalize the post‑marketing elements with the goal of bringing the therapy to U.S. patients as soon as possible.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- November 25, 2025
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma announced that the U.S. Food & Drug Administration (FDA) notified the Company that information submitted on November 5, 2025, related to the post-marketing requirement, in response to the FDA's ongoing review of the New Drug Application (NDA) for TransCon CNP (navepegritide) for children with achondroplasia, constituted a major amendment to the NDA.
AI Summary
Ascendis Pharma said the U.S. Food & Drug Administration notified the company that information submitted on November 5, 2025, related to the post‑marketing requirement for TransCon CNP (navepegritide) constituted a major amendment to its New Drug Application (NDA). Because this submission was deemed a major amendment, the FDA extended the Prescription Drug User Fee Act (PDUFA) target action date by three months to February 28, 2026.
Ascendis said it has responded to outstanding FDA requests, including providing a revised protocol for the post‑marketing study discussed at a late‑cycle meeting. The company said it will work closely with the FDA to finalize the post‑marketing requirements and remains focused on advancing TransCon CNP toward availability for children with achondroplasia in the U.S.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- November 17, 2025
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma A/S announced that pivotal Week 52 results from its randomized double-blind, placebo-controlled ApproaCH Trial of investigational once-weekly TransCon® CNP (navepegritide) in children with achondroplasia have been published in JAMA Pediatrics, a journal of the American Medical Association.
AI Summary
Ascendis Pharma announced that pivotal Week 52 results from the randomized, double‑blind, placebo‑controlled ApproaCH trial of once‑weekly TransCon CNP (navepegritide) in children with achondroplasia were published in JAMA Pediatrics. The trial enrolled 84 children ages 2–11 randomized 2:1 to TransCon CNP 100 µg/kg/week or placebo in a 52‑week double‑blind period, followed by an open‑label extension through Week 104. TransCon CNP is an investigational prodrug of C‑type natriuretic peptide designed to provide continuous CNP exposure and inhibit the overactive FGFR3 pathway in achondroplasia.
The publication reports that TransCon CNP produced a significantly higher annualized growth velocity at Week 52 versus placebo (the primary endpoint). Treated children also showed improved body proportionality and lower‑limb alignment, plus numerical gains in health‑related quality of life. Benefits occurred without accelerated bone age or negative effects on spinal curvature. Safety and tolerability were similar to placebo, with most adverse events mild or moderate, low rates of injection site reactions, and no symptomatic hypotension or bone fractures observed.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- June 9, 2025
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma A/S announced Week 26 interim analysis results from its ongoing COACH Trial, the first clinical trial to evaluate combination treatment with once-weekly investigational TransCon CNP (navepegritide) and once-weekly TransCon hGH (lonapegsomatropin) in children with achondroplasia.
AI Summary
Ascendis Pharma A/S shared promising Week 26 interim analysis results from its ongoing COACH Trial—the first study investigating the combined use of once‐weekly TransCon CNP (navepegritide) and once‐weekly TransCon hGH (lonapegsomatropin) in children with achondroplasia. In the trial, treatment‐naïve children experienced a mean annualized growth velocity (AGV) of 9.14 cm/year and an improvement in ACH height Z-score of +0.53. Those previously treated with TransCon CNP showed a mean AGV of 8.25 cm/year, with a Z-score gain of +0.44. The combination treatment also demonstrated accelerated improvements in body proportionality that aligned with the increase in linear growth. Importantly, safety and tolerability findings were consistent with the monotherapy profiles for each treatment, with most treatment-emergent adverse events being mild. These interim results suggest that adding TransCon hGH may boost the benefits of TransCon CNP in managing growth disorders in achondroplasia.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- June 2, 2025
- Target Action Date:
- November 30, 2025
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma A/S announced that Prescription Drug User Fee Act (PDUFA) goal date of November 30, 2025 to complete its review.
AI Summary
Ascendis Pharma A/S announced that the U.S. FDA has accepted its New Drug Application (NDA) for TransCon CNP (navepegritide) as a treatment for children with achondroplasia. The FDA has granted this application a Priority Review designation, meaning the agency views the drug as a promising option that may offer significant improvements in safety or effectiveness compared to current therapies.
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of November 30, 2025, to complete its review of TransCon CNP. This target date reflects the agency’s aim to expedite the evaluation process for treatments addressing serious conditions. Ascendis Pharma looks forward to working with the FDA as they continue the review process for TransCon CNP, which is administered once weekly and designed to provide sustained exposure to its active compound.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- June 2, 2025
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma A/S announced that the U.S. Food & Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for TransCon CNP (navepegritide) for the treatment of children with achondroplasia
AI Summary
Ascendis Pharma A/S announced that the U.S. Food & Drug Administration (FDA) has accepted its New Drug Application (NDA) for TransCon CNP (navepegritide), granting it Priority Review for the treatment of children with achondroplasia. This designation speeds up the evaluation process for drugs that could provide significant improvements in treating serious conditions. The PDUFA goal date is set for November 30, 2025, meaning the FDA plans to complete its review by that time.
TransCon CNP is an investigational, once-weekly prodrug of C-type natriuretic peptide designed to deliver continuous exposure to active CNP. This approach aims not only to promote growth but also to address other clinical challenges associated with achondroplasia. With a safety and tolerability profile similar to placebo and evidence of multiple benefits beyond linear growth, Ascendis hopes this therapy could fulfill a significant unmet need for children facing this rare genetic condition.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- May 13, 2025
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma A/S announced new data showing improvements in growth and bone morphometry from Week 52 of its pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia.
AI Summary
Ascendis Pharma A/S announced promising new data at Week 52 of its pivotal ApproaCH Trial for TransCon CNP (navepegritide) in children with achondroplasia. In this double-blind, placebo-controlled study of 84 children aged 2-11 years, participants receiving TransCon CNP showed significantly improved annualized growth velocity compared to those on placebo. The therapy, given once weekly as an investigational prodrug of C-type natriuretic peptide (CNP), provides continuous exposure to active CNP, which targets growth plates and skeletal muscles.
Moreover, the trial’s bone morphometry analysis revealed enhancements in lower limb alignment, proportional growth, and increased spinal canal dimensions versus placebo. These results suggest that TransCon CNP may offer benefits beyond linear growth by also improving bone structure in children with achondroplasia. Researchers presented these positive findings during sessions at the ESPE & ESE 2025 congress, marking an encouraging step forward in treating the condition.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- March 31, 2025
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma announced that it has submitted its New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) for TransCon CNP (navepegritide) for the treatment of children with achondroplasia.
AI Summary
Ascendis Pharma has submitted its New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) for TransCon CNP (navepegritide), a promising treatment for children with achondroplasia. The submission is based on strong clinical data from three randomized, double-blind, placebo-controlled trials, along with up to three years of open-label extension data. This investigational prodrug of C-type natriuretic peptide is administered once each week to provide continuous exposure of active CNP to crucial tissues, including growth plates and skeletal muscles.
Preliminary results from the pivotal ApproaCH Trial indicate that TransCon CNP not only increases growth velocity but may also reduce health burdens such as abnormal leg bowing and muscle weakness. Ascendis Pharma is optimistic that these benefits and a favorable safety profile will help the treatment emerge as a best-in-class option for achondroplasia. Future regulatory steps include an EU Marketing Authorisation Application planned for Q3 2025.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- September 16, 2024
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma announced topline data from the pivotal double-blind placebo-controlled ApproaCH Trial of TransCon CNP (navepegritide), which included 84 children with achondroplasia (ages 2-11 years) randomized 2:1 (TransCon CNP:placebo).
AI Summary
Ascendis Pharma announced promising topline data from its pivotal double‐blind, placebo‐controlled ApproaCH Trial of TransCon CNP (navepegritide) in 84 children aged 2–11 with achondroplasia. In this trial, patients were randomized in a 2:1 ratio to receive TransCon CNP or placebo for 52 weeks. The results showed that children receiving TransCon CNP had an annualized growth velocity (AGV) significantly higher than those on placebo. Overall, the least squares mean treatment difference in AGV at Week 52 was 1.49 cm/year (p<0.0001). Notably, children aged 5–11 years experienced an even greater improvement with a 1.78 cm/year difference (p<0.0001). In addition to superior linear growth, the treatment demonstrated benefits in other growth parameters and was generally well tolerated, with low and mild injection-site reactions. These findings support the potential of once-weekly TransCon CNP as an effective and safe treatment option for children with achondroplasia.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- September 16, 2024
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma A/S announced topline data from the pivotal double-blind placebo-controlled ApproaCH Trial of TransCon CNP (navepegritide), which included 84 children with achondroplasia (ages 2-11 years) randomized 2:1 (TransCon CNP:placebo).
AI Summary
Ascendis Pharma announced topline results from its pivotal ApproaCH Trial evaluating TransCon CNP (navepegritide) in 84 children aged 2-11 with achondroplasia. In this double-blind, placebo-controlled study, participants were randomized 2:1 to receive either TransCon CNP or placebo. The data showed that children receiving TransCon CNP achieved a higher annualized growth velocity compared to those on placebo. For the overall group, the least squares mean treatment difference at Week 52 was 1.49 cm per year, and for children aged 5-11 years, the difference was even greater at 1.78 cm per year. Additional favorable outcomes were noted in height Z-scores and other growth parameters. TransCon CNP was well-tolerated, with only a low frequency of mild injection site reactions, suggesting that it may offer a safe, effective, and convenient treatment option for achondroplasia in children.
Read Announcement- Drug:
- TransCon CNP
- Announced Date:
- June 24, 2024
- Indication:
- In Children with Achondroplasia
Announcement
Ascendis Pharma announced the oral presentation of data from its Phase 2 ACcomplisH Trial of TransCon CNP (navepegritide) in children aged 2 to 10 years old with achondroplasia by Ravi Savarirayan, M.D., Murdoch Children's Research Center (Australia), during ICCBH 2024, the International Conference on Children's Bone Health being held in Salzburg, Austria.
AI Summary
Ascendis Pharma announced an oral presentation at the International Conference on Children's Bone Health (ICCBH) 2024 in Salzburg, Austria. During the event, Dr. Ravi Savarirayan from Murdoch Children's Research Center, Australia, presented data from the Phase 2 ACcomplisH Trial evaluating TransCon CNP (navepegritide) in children aged 2 to 10 years old with achondroplasia.
The trial showed that after one year of treatment at a 100μg/kg/week dose, patients experienced significant improvements in well-being and physical functioning compared to those given a placebo. These gains were noted to extend beyond what could be explained by the increases in linear growth alone, suggesting an additional direct treatment effect of TransCon CNP. This finding highlights the potential of once-weekly TransCon CNP to not only promote growth but also enhance quality of life for young patients with achondroplasia.
Read Announcement
TransCon hGH (Lonapegsomatropin) FDA Regulatory Timeline and Events
TransCon hGH (Lonapegsomatropin) is a drug developed by Ascendis Pharma A/S for the following indication: Pediatric Growth Hormone Deficiency (GHD).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- TransCon hGH (Lonapegsomatropin)
- Announced Date:
- April 8, 2026
- Indication:
- Pediatric Growth Hormone Deficiency (GHD)
Announcement
Ascendis Pharma A/S announced new data demonstrating TransCon hGH accelerated TransCon CNP's benefits beyond linear growth with substantial improvements in arm span, spinal canal dimensions, and lower limb alignment.
AI Summary
Ascendis Pharma reported Week 52 COACH trial data showing that adding TransCon hGH to TransCon CNP accelerated benefits beyond linear growth in children with achondroplasia. The company said the combination produced substantial improvements in arm span, spinal canal dimensions, and lower limb alignment.
The combination therapy produced unprecedented arm‑span gains, a result described as highly meaningful to the achondroplasia community. Compared with TransCon CNP alone, the combo showed enhanced increases in spinal canal dimensions, which could be important for reducing neurological risk.
In the treatment‑naïve cohort, tibiofemoral angle (TFA) improved, indicating better straightening of the legs, while children on long‑term TransCon CNP monotherapy maintained TFA within the normal range in COACH. The company said the results are promising for improving daily function and quality of life.
Read Announcement- Drug:
- TransCon hGH (Lonapegsomatropin)
- Announced Date:
- January 8, 2026
- Indication:
- Pediatric Growth Hormone Deficiency (GHD)
Announcement
Ascendis Pharma A/S announced topline results from Week 52 of COACH, the first Phase 2 clinical trial to evaluate combination therapy with once-weekly TransCon CNP (navepegritide) and once-weekly TransCon hGH (lonapegsomatropin) in children with achondroplasia.
AI Summary
Ascendis Pharma reported topline Week 52 results from COACH, the Phase 2 trial testing once-weekly TransCon CNP (navepegritide) plus once-weekly TransCon hGH (lonapegsomatropin) in children with achondroplasia. Combination therapy produced durable growth through one year without compromising safety or tolerability. For the TransCon CNP treatment‑naïve cohort, mean annualized growth velocity (AGV) was 8.80 cm/year with an ACH height Z‑score improvement of +1.02, described as roughly triple the efficacy seen with TransCon CNP alone. The cohort previously treated with TransCon CNP showed mean AGV of 8.42 cm/year, a 3.28 cm/year increase from baseline and a Z‑score gain of +0.86 (1.28 to 2.15).
Beyond taller stature, children showed improved body proportionality and arm span (arm span >84th percentile for achondroplasia), and AGV exceeded the 97th percentile for average‑stature children. Bone age aligned with chronological age. Safety was consistent with each monotherapy: generally well tolerated with mostly mild treatment‑emergent adverse events and few injection‑site reactions. All participants completed 52 weeks and remain on study treatment.
Read Announcement- Drug:
- TransCon hGH (Lonapegsomatropin)
- Announced Date:
- December 16, 2024
- Indication:
- Pediatric Growth Hormone Deficiency (GHD)
Announcement
Ascendis Pharma announced positive Week 26 topline results from New InsiGHTS, its Phase 2 randomized, open-label, active-controlled trial in the U.S. to investigate the safety, tolerability, and efficacy of once-weekly TransCon hGH (lonapegsomatropin; approved for pediatric growth hormone deficiency) compared to daily somatropin in prepubertal children with Turner syndrome.
AI Summary
Ascendis Pharma announced positive Week 26 topline results from its U.S. Phase 2 New InsiGHTS trial. This study compared once-weekly TransCon hGH (lonapegsomatropin) with daily somatropin in 49 prepubertal children with Turner syndrome, aged 1 to 10 years old. Across all three TransCon hGH starting dose cohorts, the treatment showed a safety and tolerability profile comparable to that of daily somatropin. In addition, the annualized height velocity—a key measure of growth—was similar between the two treatment groups, regardless of the dose.
These promising results mark the first clinical trial of TransCon hGH outside pediatric growth hormone deficiency and lay the foundation for a planned basket trial in 2025 to support label expansion. Ascendis also anticipates reporting Week 52 data for further assessment of long-term benefits.
Read Announcement- Drug:
- TransCon hGH (Lonapegsomatropin)
- Announced Date:
- December 12, 2024
- Target Action Date:
- July 27, 2025
- Indication:
- Pediatric Growth Hormone Deficiency (GHD)
Announcement
Ascendis Pharma announced that he FDA set a Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025.
AI Summary
Ascendis Pharma recently received positive news from the FDA regarding its supplemental Biologics License Application for TransCon hGH to treat adult growth hormone deficiency (GHD). The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025, for its review of the application. This decision is significant as it moves the company closer to expanding the approved use of SKYTROFA®, which is already marketed for pediatric GHD, to include adults. The initiative aims to address the significant unmet needs in treating adult GHD, a condition that can lead to serious health issues, including increased body fat and decreased muscle mass, along with higher healthcare costs. With the upcoming review milestone, Ascendis Pharma hopes that the trial results will support broader treatment options for adults affected by this under-treated condition.
Read Announcement- Drug:
- TransCon hGH (Lonapegsomatropin)
- Announced Date:
- December 12, 2024
- Indication:
- Pediatric Growth Hormone Deficiency (GHD)
Announcement
Ascendis Pharma announced that the U.S. Food & Drug Administration (FDA) has accepted for review its supplemental Biologics License Application (sBLA) in adult growth hormone deficiency (GHD) for TransCon hGH (lonapegsomatropin-tcgd; marketed as SKYTROFA® for pediatric GHD).
AI Summary
Ascendis Pharma announced that the U.S. Food & Drug Administration (FDA) has accepted its supplemental Biologics License Application (sBLA) for TransCon hGH in the treatment of adult growth hormone deficiency (GHD). TransCon hGH, known as lonapegsomatropin-tcgd, is currently marketed as SKYTROFA® for pediatric GHD, and this acceptance marks a step toward expanding its use to adults. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025, for review of the application.
Ascendis Pharma views this milestone as a key move to address the significant unmet need among adults with GHD—an undertreated condition associated with multiple health challenges and high healthcare costs. The decision to review the sBLA was based on robust clinical trial results demonstrating both the drug’s effectiveness and safety in adults.
Read Announcement- Drug:
- TransCon hGH (Lonapegsomatropin)
- Announced Date:
- September 30, 2024
- Indication:
- Pediatric Growth Hormone Deficiency (GHD)
Announcement
Ascendis Pharma A/S announced it has submitted a supplemental Biologics License Application (sBLA) to the U.S. Food & Drug Administration (FDA) for TransCon hGH (marketed as SKYTROFA® (lonapegsomatropin-tcgd) in the U.S. for pediatric growth hormone deficiency) for the treatment of adults with growth hormone deficiency (GHD).
AI Summary
Ascendis Pharma A/S has submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for its long‐acting growth hormone therapy, TransCon hGH. Marketed as SKYTROFA® (lonapegsomatropin‐tcgd) in the U.S. for pediatric growth hormone deficiency, the company now seeks to expand its use for treating adults with growth hormone deficiency (GHD). This sBLA submission marks an important effort to address the needs of adult patients by potentially offering a continuous exposure growth hormone treatment option. The application demonstrates Ascendis Pharma’s commitment to advancing its innovative TransCon technology platform, reflecting its focus on developing therapies that have a meaningful impact on patient care. The FDA’s review will establish whether TransCon hGH can safely and effectively benefit adults with GHD, potentially broadening the therapeutic options available for hormone deficiencies.
Read Announcement
TransCon PTH FDA Regulatory Timeline and Events
TransCon PTH is a drug developed by Ascendis Pharma A/S for the following indication: Adult Hypoparathyroidism.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- TransCon PTH
- Announced Date:
- November 7, 2025
- Indication:
- Adult Hypoparathyroidism
Announcement
Ascendis Pharma A/S (NA announced that a new pooled analysis showed sustained and clinically meaningful improvements in renal function in adults with hypoparathyroidism treated with TransCon PTH (palopegteriparatide) through Year 3 of the Company's Phase 2 PaTH Forward and Phase 3 PaTHway trials.
AI Summary
Ascendis Pharma reported a pooled analysis from its Phase 2 PaTH Forward and Phase 3 PaTHway trials showing sustained, clinically meaningful improvements in kidney function through Year 3 in adults with hypoparathyroidism treated with TransCon PTH (palopegteriparatide). The pooled analysis included 139 patients. At Year 3, 70.3% of treated patients had eGFR gains of ≥5 mL/min/1.73 m2. Mean eGFR rose by about 9.8 mL/min/1.73 m2 in PaTH Forward and 8.8 mL/min/1.73 m2 in PaTHway. The biggest gains occurred in the first six months and were larger in patients who started with lower eGFR.
Patients also showed reduced 24-hour urinary calcium that remained in the normal range. By Year 3, ≥91% were independent of conventional therapy and ≥84% had normal blood calcium. TransCon PTH was generally well tolerated, with most adverse events mild or moderate and no treatment-related discontinuations. The findings were presented at Kidney Week 2025.
Read Announcement- Drug:
- TransCon PTH
- Announced Date:
- July 14, 2025
- Indication:
- Adult Hypoparathyroidism
Announcement
Ascendis Pharma announced new data from Week 156 of its Phase 3 PaTHway Trial confirming that long-term treatment with TransCon PTH (palopegteriparatide) continued to provide a durable response in adults with hypoparathyroidism regardless of its cause (post-surgical, autoimmune, genetic, or idiopathic), including improvements in biochemistries, kidney function, and quality of life.
AI Summary
Ascendis Pharma recently announced Week 156 results from its Phase 3 PaTHway Trial, showing that long-term treatment with TransCon PTH (palopegteriparatide) delivers a durable response in adults with hypoparathyroidism. The data confirm that the treatment continues to improve key health markers—helping normalize serum calcium levels, enhance kidney function, and boost quality of life regardless of whether the hypoparathyroidism is post-surgical, autoimmune, genetic, or idiopathic.
Patients in the study maintained normal biochemical markers and demonstrated significant improvements in kidney function as measured by eGFR. The study also noted a reduction in dependence on conventional treatments, reflecting the potential of TransCon PTH to offer meaningful long-term benefits. These results support the promise of TransCon PTH as a lasting treatment option for a wide range of hypoparathyroidism cases.
Read Announcement- Drug:
- TransCon PTH
- Announced Date:
- July 7, 2025
- Indication:
- Adult Hypoparathyroidism
Announcement
Ascendis Pharma A/S announced two oral presentations at ENDO 2025, the annual meeting of the Endocrine Society being held July 12-15, 2025, in San Francisco. Dr. Aliya Khan will present new data from Week 156 of the Company's Phase 3 PaTHway Trial demonstrating the safety and efficacy of long-term treatment with TransCon® PTH (palopegteriparatide) in adults with hypoparathyroidism – including maintenance of serum and urine biochemistries within normal levels and sustained improvement in renal function.
AI Summary
Ascendis Pharma A/S announced two oral presentations at ENDO 2025, the annual meeting of the Endocrine Society in San Francisco from July 12-15, 2025. One key presentation will be by Dr. Aliya Khan, who will share new data from Week 156 of the Phase 3 PaTHway Trial. This data highlights the long-term safety and efficacy of TransCon® PTH (palopegteriparatide) for adults with hypoparathyroidism.
Dr. Khan’s presentation focuses on how the treatment maintains serum and urine biochemistries within normal levels and shows sustained improvement in renal function. This advancement suggests that TransCon® PTH can help manage the condition effectively over time, offering hope for improved quality of life in patients with hypoparathyroidism. The presentations at ENDO 2025 further underline the potential of Ascendis Pharma’s innovative therapies in the field of endocrine disorders.
Read Announcement- Drug:
- TransCon PTH
- Announced Date:
- May 12, 2025
- Indication:
- Adult Hypoparathyroidism
Announcement
Ascendis Pharma A/S announced new data from Week 214 of its Phase 2 PaTH Forward Trial showing that long-term treatment with TransCon PTH (palopegteriparatide) continued to provide a durable response in adults with hypoparathyroidism.
AI Summary
Ascendis Pharma A/S recently presented Week 214 data from its Phase 2 PaTH Forward Trial at the ESPE & ESE 2025 congress. The study showed that long-term treatment with TransCon PTH (palopegteriparatide) provided a durable response in adults with hypoparathyroidism. Nearly all patients maintained normal albumin-adjusted serum calcium levels, and 93% stayed independent from conventional therapy. The trial highlighted that bone turnover markers rose quickly, peaked by Week 26, and then remained stable above baseline levels. Improvements in skeletal dynamics were evident, as bone mineral density stayed within normal ranges, and most patients experienced a significant increase in kidney function, measured by eGFR. Additionally, long-term use of TransCon PTH was well-tolerated, with most side effects being mild or moderate and no new safety concerns noted.
Read Announcement- Drug:
- TransCon PTH
- Announced Date:
- May 14, 2024
- Target Action Date:
- August 14, 2024
- Indication:
- Adult Hypoparathyroidism
Announcement
Ascendis Pharma announced that Prescription Drug User Fee Act (PDUFA) goal date extended by three months for further review of submission to August 14, 2024
AI Summary
Ascendis Pharma announced a delay in the FDA review process for its recent submission. The Prescription Drug User Fee Act (PDUFA) goal date has been extended by three months, moving to August 14, 2024. This extension means that the FDA will take additional time to thoroughly evaluate the details of the company’s application. The delay gives both the agency and Ascendis Pharma extra room to ensure that every aspect of the submission meets the necessary standards. Investors and other stakeholders should note that the decision process is being handled with extra care, and further updates will be awaited as the review continues. This move reflects the FDA’s commitment to a careful, detailed review and demonstrates the challenges inherent in the drug approval process.
Read Announcement- Drug:
- TransCon PTH
- Announced Date:
- May 14, 2024
- Indication:
- Adult Hypoparathyroidism
Announcement
Ascendis Pharma announced that the U.S. Food and Drug Administration (FDA) notified the Company that information submitted in response to the FDA's ongoing review of the New Drug Application (NDA) for TransCon PTH (palopegteriparatide) for adults with hypoparathyroidism constituted a major amendment to the NDA.
AI Summary
Ascendis Pharma recently received important news from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for TransCon PTH. The FDA informed the company that the additional information it submitted in response to their ongoing review of the NDA is considered a major amendment to the application. This update centers on the treatment for adults with hypoparathyroidism, and the FDA’s decision marks a significant step in evaluating the drug's safety and efficacy data.
The major amendment designation suggests that the agency viewed the newly provided data as substantial enough to have a meaningful impact on the overall review process. This development reflects the FDA's continued scrutiny of TransCon PTH, and it will likely inform further discussions and decisions as the review process progresses.
Read Announcement- Drug:
- TransCon PTH
- Announced Date:
- May 13, 2024
- Indication:
- Adult Hypoparathyroidism
Announcement
Ascendis Pharma announced 2-year results from a post-hoc analysis of the Company's Phase 3 PaTHway Trial demonstrating significant and sustained improvements in renal function in adults with chronic hypoparathyroidism treated with TransCon PTH (palopegteriparatide).
AI Summary
Ascendis Pharma released encouraging 2-year results from a post-hoc analysis of its Phase 3 PaTHway Trial. The study focused on adults with chronic hypoparathyroidism and examined the effects of TransCon PTH (palopegteriparatide) on kidney function. The analysis showed that patients experienced significant and sustained improvements in renal function, a key factor in overall health for those with the condition. These results suggest that TransCon PTH is not only effective in replacing the missing hormone but also in supporting kidney health over a prolonged period. The positive impact on renal function could make TransCon PTH a vital treatment option for patients, potentially improving their long-term quality of life. These findings add to the growing body of evidence supporting the benefits of TransCon PTH in managing chronic hypoparathyroidism.
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SKYTROFA® FDA Regulatory Events
SKYTROFA® is a drug developed by Ascendis Pharma A/S for the following indication: For the Once-Weekly Treatment of Children and Adolescents with Growth Hormone Deficiency.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- SKYTROFA®
- Announced Date:
- July 28, 2025
- Indication:
- For the Once-Weekly Treatment of Children and Adolescents with Growth Hormone Deficiency
Announcement
Ascendis Pharma announced that the U.S. Food & Drug Administration (FDA) has approved SKYTROFA® (lonapegsomatropin-tcgd; developed as TransCon hGH) for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD), a rare disorder resulting from decreased or total loss of growth hormone production.
AI Summary
On July 28, Ascendis Pharma announced that the U.S. Food & Drug Administration approved SKYTROFA® (lonapegsomatropin-tcgd) to replace natural growth hormone in adults with growth hormone deficiency, a rare disorder caused by decreased or no hormone production. SKYTROFA is a once-weekly prodrug that delivers sustained release of active human growth hormone.
The FDA’s decision was based on data from the Phase 3 foresiGHt study, which compared weekly TransCon hGH against placebo and daily somatropin in adults with GHD. Adult GHD is linked to abnormal body composition, dyslipidemia, insulin resistance, higher cardiovascular risk, and reduced quality of life.
Physicians believe a once-weekly injection may boost real-world adherence and improve outcomes. Ascendis’s CEO noted that expanding SKYTROFA to adults is the first of many planned label expansions, supporting the company’s Vision 2030 goal to become a leader in endocrinology rare diseases.
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YORVIPATH FDA Regulatory Timeline and Events
YORVIPATH is a drug developed by Ascendis Pharma A/S for the following indication: For Hypoparathyroidism in Adults.
This drug is approved by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- YORVIPATH
- Announced Date:
- December 19, 2024
- Indication:
- For Hypoparathyroidism in Adults
Announcement
Ascendis Pharma A/S announced that YORVIPATH® (palopegteriparatide; developed as TransCon PTH) is now commercially available by prescription in the United States.
AI Summary
Ascendis Pharma A/S announced that YORVIPATH® (palopegteriparatide) is now commercially available by prescription in the United States. This breakthrough treatment is designed for adults with hypoparathyroidism, a condition caused by a lack of parathyroid hormone (PTH). YORVIPATH is a prodrug of PTH and is given as a once-daily injection, providing a steady release of active hormone over a 24-hour period. Notably, it is the only FDA-approved treatment that directly addresses the root cause of the disorder in adults. Ascendis Pharma has also established a dedicated U.S. support team to help patients, caregivers, and physicians navigate the treatment process. Their program offers clinical education, help with prior authorizations, instruction on injection techniques, and financial assistance, ensuring that patients have broad and affordable access to this innovative therapy.
Read Announcement- Drug:
- YORVIPATH
- Announced Date:
- September 11, 2024
- Indication:
- For Hypoparathyroidism in Adults
Announcement
Ascendis Pharma announced that the United States Food & Drug Administration (FDA) has granted Orphan Drug exclusivity to YORVIPATH® (palopegteriparatide, developed as TransCon PTH), providing seven years of market exclusivity for YORVIPATH in the United States for the treatment of hypoparathyroidism in adults.
AI Summary
Ascendis Pharma announced that the U.S. Food & Drug Administration (FDA) has granted Orphan Drug exclusivity to YORVIPATH® (palopegteriparatide, developed as TransCon PTH) for the treatment of hypoparathyroidism in adults. This exclusivity provides the drug with seven years of market protection in the United States, meaning no similar drug can compete in this indication during that time.
YORVIPATH is designed as a prodrug of parathyroid hormone (PTH [1-34]) and is administered once daily to provide continuous exposure over a 24-hour period. The orphan exclusivity confirms that YORVIPATH is unique in addressing this rare condition, which currently affects an estimated 70,000 to 90,000 people in the U.S. This milestone underlines the potential of YORVIPATH to offer a meaningful treatment option for patients with this challenging endocrine disorder.
Read Announcement- Drug:
- YORVIPATH
- Announced Date:
- August 12, 2024
- Indication:
- For Hypoparathyroidism in Adults
Announcement
Ascendis Pharma announced that the U.S. Food & Drug Administration (FDA) has approved YORVIPATH® (palopegteriparatide; developed as TransCon PTH) for the treatment of hypoparathyroidism in adults.
AI Summary
Ascendis Pharma announced that the FDA has approved YORVIPATH® (palopegteriparatide), a treatment for hypoparathyroidism in adults. This new therapy, developed using the innovative TransCon technology platform, is designed to provide continuous daily exposure to parathyroid hormone over a 24-hour period. Hypoparathyroidism is a rare endocrine disorder that impairs calcium and phosphate balance, affecting multiple organs and the quality of life for thousands of patients in the U.S. The FDA based its approval on robust clinical data from Phase 2 and Phase 3 trials, which demonstrated the potential of YORVIPATH to address the underlying cause of the disease. In addition to this regulatory milestone, Ascendis Pharma is committed to supporting patients through dedicated access and assistance programs, promising an important new option for managing this chronic condition.
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TransCon IL-2 β/γ FDA Regulatory Events
TransCon IL-2 β/γ is a drug developed by Ascendis Pharma A/S for the following indication: Advanced cancer.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- TransCon IL-2 β/γ
- Announced Date:
- September 13, 2024
- Indication:
- Advanced cancer
Announcement
Ascendis Pharma announced initial data showing signs of clinical activity in heavily pre-treated patients with platinum-resistant ovarian cancer (PROC) treated with TransCon IL-2 β/γ in combination with chemotherapy in its ongoing Phase 1/2 IL-Believe Trial of TransCon IL-2 β/γ.
AI Summary
Ascendis Pharma reported promising early data from its Phase 1/2 IL-Believe Trial for TransCon IL-2 β/γ. In the study, heavily pre-treated patients with platinum-resistant ovarian cancer received TransCon IL-2 β/γ alongside chemotherapy. Initial results showed that 29% (4 out of 14) of efficacy-evaluable patients experienced anti-tumor responses, with both confirmed and unconfirmed partial responses. These patients had already undergone multiple treatment lines, including therapies like mirvetuximab soravtansine-gynx, before enrolling in the trial.
The safety profile of TransCon IL-2 β/γ combined with chemotherapy was generally acceptable. The most common side effects reported were fatigue, thrombocytopenia, neutropenia, and anemia, with most adverse events being mild to moderate in severity. The early findings suggest that this novel immunotherapy may offer a new treatment option for patients with limited alternatives.
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