The FDA ap­proved the now-gener­ic drug leu­cov­orin to treat pa­tients with a form of cere­bral fo­late de­fi­cien­cy, sat­is­fy­ing a month­s­long ef­fort to widen the drug’s use for a con­di­tion with symp­toms that can re­sem­ble a form of autism.

But the agency did not re­ly on ad­di­tion­al clin­i­cal da­ta, and com­ments made by two se­nior FDA of­fi­cials on a press call Mon­day sug­gest­ed the ap­pli­ca­tion was giv­en the kind of per­mis­sive, flex­i­ble re­view that oth­er drug­mak­ers have been hope­ful for — but not grant­ed — un­der the cur­rent agency lead­er­ship.

Tues­day's ap­proval was tech­ni­cal­ly giv­en to GSK, the orig­i­nal mak­er of leu­cov­orin be­fore it went gener­ic. Of­fi­cials at the FDA said the la­bel change will trick­le down to gener­ic com­pa­nies that are pro­duc­ing it now that GSK no longer does.

With its lat­est read­out, Ver­tex ap­pears well on its way to es­tab­lish­ing it­self in an­oth­er dis­ease area be­yond cys­tic fi­bro­sis.

The Boston biotech de­scribed a Phase 3 win on Mon­day for its drug povetaci­cept in treat­ing a se­vere kid­ney dis­ease, paving the way to com­plete an ac­cel­er­at­ed ap­proval fil­ing by month’s end. The read­out was Ver­tex’s most-an­tic­i­pat­ed da­ta re­veal for this year's first half and al­so a test of the wis­dom be­hind get­ting its hands on the drug through its $4.9 bil­lion buy­out of Alpine Im­mune Sci­ences in 2024.

The in­ter­im read­out comes from the on­go­ing RAINIER tri­al, study­ing the dis­ease called im­munoglob­u­lin A nephropa­thy, or IgAN. The dis­ease de­te­ri­o­rates kid­ney func­tion over time, with most adult pa­tients pro­gress­ing to end-stage kid­ney dis­ease or dy­ing with­in 20 years of di­ag­no­sis, ac­cord­ing to Ver­tex. Povetaci­cept blocks two cy­tokines, BAFF and APRIL, that are im­pli­cat­ed in a va­ri­ety of au­toim­mune con­di­tions, in­clud­ing IgAN.

Gene ther­a­pies were sup­posed to be com­pe­ti­tion-proof. They are sci­en­tif­i­cal­ly com­plex, re­quire be­spoke man­u­fac­tur­ing, of­ten go af­ter small pa­tient pop­u­la­tions and can cost mil­lions per dose.

But that as­sump­tion is be­ing test­ed by a wave of com­peti­tors be­ing de­vel­oped in Chi­na — with prices start­ing at a frac­tion of what's cur­rent­ly avail­able in the US.

An End­points News analy­sis looked at the world's 10 most ex­pen­sive gene ther­a­pies and found 77 com­peti­tors in de­vel­op­ment around the world. Of those, 48 are from Chi­na — more than dou­ble the num­ber un­der de­vel­op­ment in the US, and 7 times what's be­ing worked on in Eu­rope.

The de­vel­op­ment of these gene ther­a­pies is the lat­est piece of ev­i­dence that Chi­na is com­pet­ing high­er and high­er on the spec­trum of in­no­v­a­tive sci­ence.