See Why Ascendis Pharma Stock Is Shining On Thursday

The FDA on Wednesday gave a thumbs-up to Ascendis Pharma A/S's ASND long-acting growth hormone lonapegsomatropin-tcgd. Marketed as Skytrofa, the somatropin prodrug has been approved in kids ages one and older with growth hormone deficiency (GHD) who weigh at least 25.4 pounds. 

The Company says that it is the first childhood GHD therapy that can be taken just once a week. Patients who make the switch from daily somatropin, the standard of care for more than three decades, could see up to 86% fewer injection days per year, Ascendis said. The Company is also testing its drug in adult GHD.

Ascendis expects to launch the drug in the U.S. "shortly" alongside a "suite" of patient support programs, including education for families on how to inject the med. 

Skytrofa approval includes a bespoke autoinjector and cartridges, which can be kept at room temperature for up to six months after they're first removed from the refrigerator. 

Skytrofa is the first FDA-approved drug to leverage Ascendis' TransCon technology, apart from its once-weekly distinction. The FDA based its approval on Skytrofa's phase 3 heiGHt study results, which pitted the drug against Pfizer Inc's PFE daily somatropin drug Genotropin in 161 children.

For the study's primary endpoint, Skytrofa patients grew an average of 11.2 cm/year at the 52-week mark, compared to 10.3 cm/year in the daily somatropin cohort. 

Earnings: Successful Skytrofa launch will be vital to Ascendis, which reported a net loss of €134.4 million, or €2.50 per share in Q2. Executives on Wednesday's call said a green light in Europe could come as early as the fourth quarter. The cash balance at Q2 was €641.3 million. 

Related content: Benzinga's Full FDA Calendar

Analyst Take: Credit Suisse and SVB Leerink beefed up the price target to $185 and $182, respectively, with Outperform ratings unchanged.

Price Action: ASND shares are up 25.2% at $154.83 during the market session on the last check Thursday.

Photo by Tasy Hong from Pixabay