FDA accepts TransCon hGH biopharmace product licensing applications in China in Phase III clinical

Danish biopharmaceutical company Ascendis Pharma recently announced that the U.S. Food and Drug Administration (FDA) has accepted a weekly biopharmaceutical license application (BLA) for long-acting growth hormone TransCon hGH, a human growth hormone (hGH) long-acting pre-drug given once a week to treat childhood growth hormone deficiency (GHD).
fda has designated the target date for action under the Prescription Drug User Charge Act (PDUFA) as June 25, 2021.
the FDA also said it did not plan to convene an advisory committee meeting to discuss the application.
, there is no long-acting growth hormone for GHD in children.
if approved, TransCon hGH would be the first long-acting growth hormone therapy to treat GHD in children.
the new GHD drug, it's worth noting that Noor and Nord's weekly long-acting growth hormone derivative Sogroya (somapacitan-beco) was recently approved by the U.S. FDA for adult treatment of GHD.
Sogroya is the first human growth hormone (hGH) therapy to treat adult GHD only once a week, while other FDA-approved hGH preparations must be injected daily.
Sogroya is modified by natural hGH to enhance its binding to the plasma protein albumin, making it suitable for weekly dosing.
, Sogroya is also developing treatments for GHD in children.
TransCon hGH is the only human growth hormone pre-drug in the world designed using the patented transient connection technology.
the drug's mechanism of effect is different from that of other technology's long-acting growth hormone similaries.
it guarantees the release of unretouched, active human growth hormone for 7 days in the body, ensuring that the tissue distribution of active human growth hormone in the body is consistent with the daily recombinant growth hormone (rhGH).
in the United States and Europe, TransCon hGH has been granted the orphan drug of treatment for GHD (ODD).
Ascendis plans to submit transCon hGH marketing authorization applications (MAAs) to the European Medicines Agency (EMA) in the third quarter of this year.
addition, the company is conducting Phase III clinical trials of TransCon hGH in Greater China through a strategic investment in VISEN Pharmaceuticals.
child growth hormone deficiency (GHD) is a serious and rare disease caused by a lack of growth hormone secreted by the pituitary gland.
children with GHD are not only short in stature, but also have metabolic abnormalities, psychosocial challenges, cognitive impairment and poor quality of life.
, the standard of care for GHD has been one injection of hGH per day to improve growth and metabolic effects.
for caregivers and patients, the burden of daily injections is high, which can lead to poor compliance and reduce overall treatment effectiveness.
BLA for the treatment of GHD in children based on TransCon hGH is based on a clinical development project that includes eight clinical trials evaluating efficacy and safety in more than 400 GHD patients.
March last year, Ascendis published top-line data from TransCon hGH's heiGHt study for the treatment of GHD children in Stage III.
a randomized, open-label, positive drug-controlled study that compared TransCon hGH once a week with Genotropin once a day.
results show that the study reached its main end point: in week 52, TransCon hGH was no inferior to daily hGH in terms of annualized height rate (AHV, in centimeters/year) and better than daily hGH.
specific data were 11.2 cm/year for transCon hGH treatment group AHV and 10.3 cm/year for the transCon hGH treatment group using covance analysis (ANCOVA) (difference between the two groups: 0.86 cm/year, 95% CI:0.22-1.50, p=0.0088).
it is worth noting that at each follow-up, the TransCon hGH treatment group had AHV higher than the daily hGH treatment group, and the treatment differences were statistically significant from the 26th week, including the 26th week.
4% and 11% of patients (AHV<8.0 cm/year) responded poorly in the TransCon hGH treatment group and the daily hGH treatment group.
sensitivity analysis completed by the study supports the main results and indicates the robustness of these results.
TransCon means Transient Connect.
Ascendis' proprietary TransCon platform is an innovative technology designed to create new therapies that optimize therapeutic outcomes, including efficacy, safety, and frequency of treatment.
TransCon molecule has three components: an unmodated mother drug, an inert carrier that protects it, and a link that temporarily connects two people.
when connected, the carrier insethers and protects the mother drug from removal.
when injected into the body, physiological acidity and temperature conditions begin to release unretouched active parent drugs in a predictable manner.
the mother drug is unmoded, its original mode of action should remain unchanged.
TransCon technology can be widely used in proteins, peptides or small molecules in a variety of therapeutic fields, and can be used systematically or locally.
, Ascendis is using TransCon technology to build a leading, fully integrated rare disease company.
the company uses TransCon technology to create new therapies with clinically proven maternal drugs that have best-in-class efficacy, safety and/or convenience in the best of their kind.
addition to children's GHD, the company is also developing TransCon hGH treatments for adult GHD in Phase III clinical development.
In addition, the company has two rare endocrinology assets in Phase II clinical: (1) TransCon PTH is a long-acting thyroid hormone (PTH) pre-drug (prodrug) for the treatment of hypothyroidism;
In 2018, Ascendis established a joint venture with Vivo Capital to focus on Greater China and to develop and promote Ascendis's endocrine rare disease treatment programs in Greater China, covering Chinese mainland, Hong Kong, Macau and Taiwan.
October 2019, TransCon hGH's Phase III clinical trial application was approved by the National Drug Administration (NMPA) to conduct a Phase III clinical study in China for the treatment of childhood growth hormone deficiency (GHD).
TransCon hGH will be China's first unretouched long-acting human growth hormone, used once a week, to continuously release the same human growth hormone as the daily preparation.
original source: Ascendis Pharma A/S Announces U.S. Food and Administration (FDA) Accepts Biologics License Application (BLA) for TransCon™ hGH for Pediatrics Grow Hormone Defoy (GHD) This article was originally published from Bio Valley, please download BioGuard APP (GHD)