The FDA grant­ed ac­cel­er­at­ed ap­proval to De­nali Ther­a­peu­tics’ rare dis­ease drug Avlayah, buck­ing a re­cent trend of re­jec­tions in the space that has put the agency un­der po­lit­i­cal pres­sure.

Avlayah, whose gener­ic name is tiv­i­de­no­fusp al­fa, will be able to treat pa­tients with Hunter syn­drome, a rare ge­net­ic dis­or­der that man­i­fests in child­hood, who weigh at least 5 kg “pri­or to ad­vanced neu­ro­log­ic im­pair­ment,” ac­cord­ing to a De­nali press re­lease. It comes with a boxed warn­ing for hy­per­sen­si­tiv­i­ty (in­clud­ing ana­phy­lax­is) that’s con­sis­tent with Avlayah’s drug class, De­nali said. The com­pa­ny al­so re­ceived a rare pe­di­atric dis­ease pri­or­i­ty re­view vouch­er.

Sea­port Ther­a­peu­tics is build­ing a fail-safe in­to its Phase 2b de­pres­sion tri­al.

The biotech is test­ing its drug, called SPT-300, in pa­tients with ma­jor de­pres­sive dis­or­der. But ex­ec­u­tives be­lieve SPT-300 could prove more promis­ing in a sub­pop­u­la­tion of de­pres­sion pa­tients who al­so have what’s called “anx­ious dis­tress,” a spe­cif­ic di­ag­nos­tic cri­te­ri­on in the DSM-5 di­ag­nos­tic man­u­al.

The study’s pri­ma­ry end­point will mea­sure SPT-300’s ef­fect in all pa­tients. But Sea­port will al­so try to quan­ti­fy im­prove­ments in that sub­pop­u­la­tion as a way to pro­vide “op­tion­al­i­ty,” chief sci­en­tif­ic of­fi­cer Michael Chen told End­points News. It’s a plan that, if the tri­al ul­ti­mate­ly fails, still leaves Sea­port a path for­ward to fu­ture stud­ies and a po­ten­tial ap­proval in a small group of pa­tients.

Take­da’s mul­ti-year re­struc­tur­ing will con­tin­ue un­der in­com­ing CEO Julie Kim.

The com­pa­ny an­nounced Wednes­day that the board has ap­proved "next steps" in its trans­for­ma­tion that will put the fo­cus on up­com­ing launch­es and its late-stage pipeline. De­tails were sparse, but Take­da said the changes are ex­pect­ed to save JPY 200 bil­lion ($1.3 bil­lion) by fis­cal 2028.

A Take­da spokesper­son said the changes are fo­cused on “stan­dard­iz­ing and sim­pli­fy­ing ways of work­ing.” The spokesper­son de­clined to pro­vide fur­ther de­tail, not­ing that “the specifics will vary by or­ga­ni­za­tion and by coun­try.”

The com­pa­ny said in a news re­lease that its next steps are “aligned with the pre­vi­ous­ly an­nounced or­ga­ni­za­tion­al struc­ture.”

Out­go­ing CEO Christophe We­ber laid out plans in 2024 to sim­pli­fy Take­da’s struc­ture, re­move lay­ers and broad­en roles. The com­pa­ny has since closed a re­search cen­ter in San Diego, trimmed its pipeline and made hun­dreds of staff cuts.

The FDA ap­proved Cor­cept Ther­a­peu­tic­s' Li­fy­or­li in com­bi­na­tion with nab-pa­cli­tax­el for the treat­ment of three dif­fer­ent kinds of can­cer in pa­tients who have re­ceived up to three pri­or lines of treat­ments.

The Wednes­day ap­proval came more than three months ahead of the com­pa­ny's Ju­ly 11 user fee goal date. In Jan­u­ary, the agency re­ject­ed the same drug to treat a rare hor­mon­al dis­or­der called Cush­ing’s syn­drome.

A mul­ti­cen­ter, open-la­bel tri­al of 381 pa­tients test­ed Li­fy­or­li, a se­lec­tive glu­co­cor­ti­coid re­cep­tor an­tag­o­nist, with nab-pa­cli­tax­el for adults with plat­inum-re­sis­tant ep­ithe­lial ovar­i­an, fal­lop­i­an tube, or pri­ma­ry peri­toneal can­cer. It was test­ed in pa­tients who have re­ceived one to three pri­or lines of treat­ment, at least one of which in­clud­ed be­va­cizum­ab.