Clinical Ascendis gets FDA nod on once a week pediatric...

Ascendis gets FDA nod on once a week pediatric growth hormone deficiency injection

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Ascendis Pharma Lonapegsomatropin-tcgd injection, Skytrofa, which is a weekly somatropin formulation, has recently been approved by FDA for children having growth hormone deficiency.

The injection has been approved for once a week dosage for children weighing no less than 11.5 kg, having insufficient growth largely because of inadequate secretion of endogenous growth hormone.   

The said deficiency, caused by anterior pituitary gland, is a rare disorder among children in United States with a ratio of occurrence to be 1 to 3,500.

The approval includes an auto-injector along with cartridges which can store the medicine for 6 months at room temperature after taking out of the refrigerator.

Paul Thornton, MB BCh, MRCPI, a clinical investigator and pediatric endocrinologist in Fort Worth, Texas, stated, “Today’s approval represents an important new choice for children with GH deficiency and their families, who will now have a weekly treatment option.” He further added, “In the pivotal head-to-head clinical trial, weekly Skytrofa demonstrated higher annualizedized height velocity at week 52 compared to somatropin. This weekly treatment could reduce treatment burden and potentially replace the daily somatropin therapies, which have been the standard of care for over 30 years.”  

A 52-weeks global phase III height trial was conducted for Skytrofa, the results of which led to the FDA’s approval for the drug. It was a randomized, open-label, parallel-group trial in which 161 treatment-naïve children with growth hormone deficiency were tested for weekly skytrofa against the daily somatropin formulation.

The principal endpoint of the study was the annualized height velocity of the weekly skytrofa group against the other group at completion of 52 weeks. Whereas, adverse events, injection-site reactions, change in height SDS and incidence of anti-human GH antibodies were among other included endpoints. 

At the 52nd week, the annualized height velocity for Skytrofa was 11.2 cm/year and that for the daily somatropin was 10.3 cm/year, having a difference of 0.9 cm/year. Moreover, there seemed to have no serious adverse effects or no discontinuations for skytrofa were reported throughout. The commonly noted adverse reactions were infections like nausea and vomiting, diarrhea, arthralgia and arthritis, abdominal pain, cough and haemorrhage.  

The FDA, in a press release, said that the drug may interact with oral estrogen, glucocorticoid treatment, insulin and other antihyperglycemic agents.

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