One day af­ter step­ping down as CDER Di­rec­tor, George Tid­marsh has said he will fight the in­ves­ti­ga­tions in­to his con­duct and is re­con­sid­er­ing his de­ci­sion.

Tid­marsh's in­ten­tions were shared with a hand­ful of se­nior FDA staff in the Of­fice of New Drugs (OND) dur­ing a meet­ing of su­per­vi­sors on Mon­day, which was held vir­tu­al­ly. At the meet­ing, OND Di­rec­tor Mary Thanh Hai re­layed Tid­marsh's com­ments to staff that he was on ad­min­is­tra­tive leave and that he's not re­sign­ing, ac­cord­ing to a per­son fa­mil­iar with the ses­sion. The per­son spoke to End­points News on con­di­tion of anonymi­ty.

End­points spoke to Tid­marsh af­ter­ward, who said that he was sec­ond-guess­ing his de­ci­sion to re­sign. On Sun­day, Tid­marsh had said “I’m go­ing to fight it. It’s my name and cred­i­bil­i­ty."

FDA Com­mis­sion­er Mar­ty Makary twice this week hint­ed that the agency will soon take more “dereg­u­la­to­ry” ac­tion, in ad­di­tion to the FDA’s re­cent move to re­lax biosim­i­lar clin­i­cal tri­al re­quire­ments.

In a press con­fer­ence on Wednes­day un­veil­ing the long-await­ed biosim­i­lar guid­ance, Makary told re­porters that po­ten­tial­ly next week, the agency will "make an­oth­er an­nounce­ment on the elim­i­na­tion" of cer­tain uniden­ti­fied an­i­mal test­ing re­quire­ments for new drug de­vel­op­ment.

And on Thurs­day in New York City, Makary told the Galien Fo­rum, "You're go­ing to see some very spe­cif­ic an­nounce­ments com­ing out very soon on guid­ance, and they're go­ing to be mov­ing in one di­rec­tion. I'll tell you right now, dereg­u­la­to­ry."

Makary's dereg­u­la­to­ry moves will like­ly help an in­dus­try that's been the tar­get of many of HHS Sec­re­tary Robert F. Kennedy Jr.'s con­fronta­tion­al cri­tiques.

Aca­d­e­mics and biotechs look­ing at be­spoke gene ther­a­pies like the one used to treat ba­by KJ Mul­doon now have a trove of new in­for­ma­tion for nav­i­gat­ing a unique FDA ap­proval process.

Uni­ver­si­ty of Penn­syl­va­nia sci­en­tist Ki­ran Musunuru and Re­bec­ca Ahrens-Nick­las of the Chil­dren's Hos­pi­tal of Philadel­phia, the re­searchers be­hind ba­by KJ's treat­ment, on Fri­day took the rare step of re­leas­ing pre-clin­i­cal tri­al com­mu­ni­ca­tions with the FDA and the agen­cy's re­spons­es as part of their pa­per pub­lished in the Amer­i­can Jour­nal of Hu­man Ge­net­ics.

Those types of com­mu­ni­ca­tions are al­most al­ways kept con­fi­den­tial by com­pa­nies, and could of­fer a road map to oth­er teams hop­ing to ex­pand on Ahrens-Nick­las and Musunuru's work.

HHS has ap­proved ap­pli­ca­tions from eight drug­mak­ers to par­tic­i­pate in the gov­ern­ment’s pi­lot pro­gram test­ing a re­bate mod­el for cer­tain 340B dis­counts.

The green light from HHS’ Health Re­sources and Ser­vices Ad­min­is­tra­tion means com­pa­nies can be­gin im­ple­ment­ing their re­bate mod­el plans in Jan­u­ary, mark­ing a sig­nif­i­cant change to the way the 340B dis­count pro­gram has op­er­at­ed for decades. Un­der a re­bate mod­el, health cen­ters would pur­chase cer­tain 340B-el­i­gi­ble drugs at mar­ket price, then re­coup the dis­counts via re­bates, as op­posed to up­front.

A hand­ful of phar­ma com­pa­nies have been bat­tling HHS in court for months over their own pro­posed 340B re­bate mod­els. For now, the pi­lot pro­gram is lim­it­ed to drugs sub­ject to the first round of Medicare ne­go­ti­a­tions, a group that in­cludes Bris­tol My­ers Squibb’s blood thin­ner Eliquis, J&J’s plaque pso­ri­a­sis drug Ste­lara, and oth­ers.

The FDA has turned down Bio­haven’s drug for spin­ocere­bel­lar atax­ia, a neu­rode­gen­er­a­tive dis­ease that leads to loss of co­or­di­na­tion and bal­ance, cit­ing is­sues with us­ing re­al-world ev­i­dence and nat­ur­al his­to­ry stud­ies. As a re­sult, Bio­haven is cut­ting costs, paus­ing pro­grams and may lay off staff.

Known as tro­r­ilu­zole, it’s the lat­est rare dis­ease drug to be re­ject­ed or face road bumps un­der new FDA lead­er­ship. The chal­lenges have come seem­ing­ly af­ter com­pa­nies and the agency had pre­vi­ous­ly agreed on how a study would be con­duct­ed — elic­it­ing frus­tra­tion from drug de­vel­op­ers. Oth­ers, like UniQure and Replimune, have al­so been turned away by the agency for us­ing tri­als with­out a typ­i­cal con­trol arm that’s em­ployed in ran­dom­ized con­trolled tri­als.