Ascendis wins approval for once-weekly growth hormone drug as Pfizer, Opko Health keep up the pressure
Denmark’s Ascendis Pharma won its first FDA approval Wednesday afternoon, beating out a rival program at Pfizer for pediatric growth hormone deficiency.
Ascendis notched the OK for lonapegsomatropin, to be marketed as Skytrofa, as the first once-weekly treatment for children aged one and older who don’t produce enough growth hormone and weigh at least 11.5 kilograms, the biotech announced. There’s no info on the pricing yet and Endpoints News has reached out for comment, but CEO Jan Møller Mikkelsen said on a conference call Wednesday the cost will be “responsible.”
Previous GHD treatments require daily injections of somatropin, a synthetic form of human growth hormone, but Ascendis hopes the sustained release over weekly dosing will provide patients with a more convenient option. The company said the approval covers not only the drug, but cartridges that can be stored at room temperature for up to six months.
“This once-weekly treatment could reduce treatment burden and potentially replace the daily somatropin therapies, which have been the standard of care for over 30 years,” Ascendis Phase III investigator Paul Thornton said in a statement.
Wednesday’s approval was based on a Phase III trial that enrolled 161 children who hadn’t taken any prior treatment. The study compared lonapegsomatropin to Genotropin daily injections, and demonstrated Ascendis’ program was not just non-inferior to after one year, but also superior. Children taking the Ascendis drug grew an average of 0.9 cm per year faster than those on Genotropin.
The FDA’s decision also came earlier than expected after Ascendis’ PDUFA date was pushed back in June. Originally slated for a June decision, regulators delayed the PDUFA to September 25 after Ascendis submitted additional information as part of its application.
At least one analyst appeared confident in Skytrofa’s ability to shake things up. SVB Leerink’s Joseph Schwartz wrote to investors Wednesday evening that, despite the limited information revealed on the conference call regarding pricing and other matters, Ascendis’ focus on long-term gains rather than strictly sales numbers is a positive sign.
“Considering the growth hormone market is fragmented with multiple undifferentiated products that must be treated daily, [Ascendis] believes that market is primed for disruption,” Schwartz wrote. He added that he expects the drug to be approved in Europe by the end of the year and that Ascendis is setting up access programs to help patients switch from daily treatment.
Ascendis’ approval beat out a candidate touted by Pfizer and Opko Health, which revealed its latest Phase III data in October 2019 and October 2020. In the first study, the pair’s somatrogon program showed non-inferiority to Genotropin and that the drug arm saw patients grow at an average of 0.33 cm per year faster than standard of care.
The second trial, meanwhile, observed improvement on the average score on a common GHD test, known as the Life Interference Questionnaire, after 12 weeks on each treatment.
Pfizer and Opko are expected to hear back from the FDA in October and are also hoping their long-acting hormone treatment can shake up the market, particularly after a high-profile flop way back in 2016. But Ascendis beat them to the punch by a little over a month, giving the biotech a potential leg up on getting its drug to market.