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.
© 2022 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Ad Disclosure: The rate information is obtained by Bankrate from the listed institutions. Bankrate cannot guaranty the accuracy or availability of any rates shown above. Institutions may have different rates on their own websites than those posted on Bankrate.com. The listings that appear on this page are from companies from which this website receives compensation, which may impact how, where, and in what order products appear. This table does not include all companies or all available products.
All rates are subject to change without notice and may vary depending on location. These quotes are from banks, thrifts, and credit unions, some of whom have paid for a link to their own Web site where you can find additional information. Those with a paid link are our Advertisers. Those without a paid link are listings we obtain to improve the consumer shopping experience and are not Advertisers. To receive the Bankrate.com rate from an Advertiser, please identify yourself as a Bankrate customer. Bank and thrift deposits are insured by the Federal Deposit Insurance Corp. Credit union deposits are insured by the National Credit Union Administration.
Consumer Satisfaction: Bankrate attempts to verify the accuracy and availability of its Advertisers' terms through its quality assurance process and requires Advertisers to agree to our Terms and Conditions and to adhere to our Quality Control Program. If you believe that you have received an inaccurate quote or are otherwise not satisfied with the services provided to you by the institution you choose, please click here.
Rate collection and criteria: Click here for more information on rate collection and criteria.