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.

Outside of Covid-19, 2021 has been the year of the accelerated approval.

Beginning last spring, FDA openly challenged six “dangling” accelerated approvals (hadn’t confirmed their clinical benefit yet), three of which were later pulled by the companies.

Then in June, FDA pulled out the accelerated approval pathway, seemingly out of nowhere, to sign off on Biogen’s controversial Alzheimer’s drug Aduhelm. It hadn’t even been mentioned at the drug’s adcomm.

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.