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Also known as Lonapegsomatropin [SKYTROFA®]
Lonapegsomatropin · 4 trials · 8 indications
To evaluate the efficacy of lonapegsomatropin as compared to somatropin in children and adolescents with TS, SHOX-D, SGA, or ISS
An Adverse Event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and which did not necessarily have a causal relationship with the treatment. An AE was considered a TEAE if it occurred on or after the first dose of investigational product and was not present prior to the first dose, or it was present at the first dose but increased in severity during the trial. A serious AE was any untoward medical occurrence at any dose that met any of the following criteria: resulted in death; was life threatening; required or prolonged inpatient hospitalization; resulted in persistent or significant disability/incapacity; resulted in a congenital anomaly/birth defect in a neonate/infant born to a mother exposed to the trial drug or was considered a significant medical event by the investigator.
Trunk percent fat was assessed by dual-energy X-ray absorptiometry.
| Arm | Type | Description |
|---|---|---|
| Lonapegsomatropin, once daily | EXPERIMENTAL | Participants will receive Lonapegsomatropin by subcutaneous injection for 2 years (104 weeks) |
| somatropin, once daily | ACTIVE_COMPARATOR | Participants will receive somatropin by subcutaneous injection for 1 year (52 weeks) followed by lonapegsomatropin for 1 year (52 weeks) |
| Lonapegsomatropin/Lonapegsomatropin | EXPERIMENTAL | Participants who had completed treatment with lonapegsomatropin in TCH-306 study were enrolled in the extension study and received lonapegsomatropin administered once weekly by subcutaneous injection for a treatment period of up to 52 weeks. |
| Placebo/Lonapegsomatropin | EXPERIMENTAL | Participants who had completed treatment with placebo in TCH-306 study were enrolled in the extension study and received lonapegsomatropin administered once weekly by subcutaneous injection for a treatment period of up to 52 weeks. |
| Somatropin/Lonapegsomatropin | EXPERIMENTAL | Participants who had completed treatment with somatropin in TCH-306 study were enrolled in the extension study and received lonapegsomatropin administered once weekly by subcutaneous injection for a treatment period of up to 52 weeks. |
| Lonapegsomatropin | EXPERIMENTAL | Lonapegsomatropin administered once-weekly by subcutaneous injection. |
| Placebo | PLACEBO_COMPARATOR | Placebo for Lonapegsomatropin administered once-weekly by subcutaneous injection. |
| Somatropin | ACTIVE_COMPARATOR | Somatropin administered once-daily by subcutaneous injection. |
| Lonapegsomatropin at 0.24 mg hGH/kg/week | EXPERIMENTAL | Lonapegsomatropin at 0.24 mg hGH/kg/week administered once-weekly by subcutaneous injection |
| Lonapegsomatropin at 0.30 mg hGH/kg/week | EXPERIMENTAL | Lonapegsomatropin at 0.30 mg hGH/kg/week administered once-weekly by subcutaneous injection |
| Lonapegsomatropin at 0.36 mg hGH/kg/week | EXPERIMENTAL | Lonapegsomatropin at 0.36 mg hGH/kg/week administered once-weekly by subcutaneous injection |
| Somatropin at 0.05 mg/kg/day | ACTIVE_COMPARATOR | Somatropin at 0.05 mg/kg/day administered once-daily by subcutaneous injection |
| Name | Type | Description |
|---|---|---|
| Lonapegsomatropin [SKYTROFA®] | COMBINATION_PRODUCT | Subcutaneous injection once weekly |
| Somatropin Pen Injector | COMBINATION_PRODUCT | Subcutaneous injection once daily |
| Lonapegsomatropin | DRUG | Study participants were individually dosed with subcutaneous injection of Lonapegsomatropin once-weekly for 52 weeks. |
| Placebo | OTHER | The placebo for lonapegsomatropin drug product contained the same excipients as lonapegsomatropin drug product but does not contain lonapegsomatropin itself. The placebo solution was administered by subcutaneous (SC) injection via syringe and needle. Due to the different hGH dose requirements, depending on participant's age and concomitant use of oral estrogen, this trial has 3 dosing groups and the placebo received the same dose volume as if they were randomized to once-weekly lonapegsomatropin. |
| Somatropin | DRUG | Somatropin solution is provided in a pre-filled pen intended for daily subcutaneous injection. Due to the different hGH dose requirements, depending on participant's age and concomitant use of oral estrogen, this trial has 3 dosing groups per arm, followed by gradual increasing dose titration to a target maintenance dose. |
Inclusion Criteria: 1. Chronological age between ≥2 and \<18 years, at start of screening. 2. Naïve to growth hormone and growth hormone promoting therapies. 3. Prepubertal. 4. Able to stand without assistance. 5. Diagnosis of TS, SHOX-D, SGA, or ISS with impaired growth or short stature, according...
Lonapegsomatropin is an investigational once-weekly growth hormone therapy being studied for Turner Syndrome, Growth Hormone Deficiency, and Adult Growth Hormone Deficiency. It is a long-acting form of somatropin designed to treat conditions related to growth hormone deficiency in both children and adults.
Lonapegsomatropin targets the growth hormone receptor (GHR) as an agonist. By activating this receptor, it mimics the action of natural growth hormone, which is intended to promote growth and development in patients with growth hormone deficiency or related conditions.
Lonapegsomatropin is developed by Ascendis Pharma A/S, a biopharmaceutical company listed on the NASDAQ under the ticker symbol ASND. The company is conducting clinical trials to evaluate the drug's safety and efficacy across multiple indications.
Lonapegsomatropin is in Phase 3 clinical development for growth hormone deficiency and related conditions. It is an investigational drug and has not been approved by regulatory authorities. Clinical trials are ongoing to assess its efficacy and safety in adult and pediatric populations.
Lonapegsomatropin is being studied in several trials, including NCT04615273, a Phase 3 trial comparing it to placebo and daily somatropin in adults with growth hormone deficiency, and NCT05171855, a long-term safety study. A Phase 2 trial (NCT05690386) is evaluating it in Turner Syndrome, and NCT07221851 is a Phase 3 trial in children with short stature.
Lonapegsomatropin is also known as SKYTROFA. The drug is marketed under the brand name SKYTROFA, which is the same compound being investigated in clinical trials for growth hormone deficiency and Turner Syndrome.