Astel­las has picked up boast­ing rights to gain­ing the first FDA ap­proval of a FLT3 in­hibitor for a spe­cif­ic group of FLT3 pos­i­tive acute myeloid leukemia pa­tients — which ac­counts for close to a third of the pop­u­la­tion.

Back in May the Japan­ese phar­ma re­port­ed that the agency had of­fered a snap 6-month re­view on its fast-track pro­gram for gilter­i­tinib, some­thing that’s be­come quite com­mon in the on­col­o­gy group.

That’s good news for the 30% or so of the 19,000 new­ly di­ag­nosed AML cas­es each year that will be linked to the ge­net­ic mu­ta­tion. The drug has been in a piv­otal study where in­ves­ti­ga­tors have been study­ing it’s abil­i­ty to cut re­laps­es in the pa­tient pop­u­la­tion.

The drug will be sold as Xospa­ta, and Astel­las set the whole­sale price at $22,500 for 30 days, which would trans­late to $270,000 for a year of ther­a­py — though pay­ers ne­go­ti­ate down from that fig­ure. In a state­ment, the com­pa­ny said that their in­ter­im analy­sis of the piv­otal pro­gram tracked a me­di­an du­ra­tion of re­sponse of 4.6 months.

Alexan­der Ed­ward Perl

This OK de­liv­ers a “new, high­ly-ef­fec­tive, and well-tol­er­at­ed treat­ment op­tion to the clin­ic for a group of tru­ly high-risk pa­tients who, un­til to­day, had no spe­cif­ic ther­a­pies avail­able be­yond chemother­a­py to treat their dis­ease,” says Alexan­der Perl, an as­so­ciate pro­fes­sor of Hema­tol­ogy-On­col­o­gy at the Uni­ver­si­ty of Penn­syl­va­nia. Perl has been lead­ing the clin­i­cal tri­al pro­gram for this drug.

“Al­though we’re wait­ing for the fi­nal analy­sis of (the Phase III study) AD­MI­RAL, the avail­able da­ta with gilter­i­tinib show few­er and milder side ef­fects than typ­i­cal­ly is seen with tra­di­tion­al chemother­a­py,” Perl added, not­ing that they al­so tracked a sig­nif­i­cant­ly high­er rate of com­plete re­spons­es.

The ap­proval marks the FDA’s 54th OK of the year as it goes deep­er in­to record ter­ri­to­ry af­ter pass­ing the old record set in 1996.

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.

The National Institutes of Health has again declined to use so-called “march-in” rights to lower the price of Astellas and Pfizer’s prostate cancer drug Xtandi despite being invented at UCLA with grants from the US Army and NIH.

“Given the remaining patent life and the lengthy administrative process involved for a march-in proceeding, NIH does not believe that use of the march-in authority would be an effective means of lowering the price of the drug,” NIH told prostate cancer patients Robert Sachs and Clare Love, in a letter shared with Endpoints News. The institutes’ analyses found Xtandi “to be widely available to the public,” an indication that there was not a pressing need for the US to act.