While oth­er drug­mak­ers have crit­i­cized the FDA’s re­cent judg­ment on rare dis­ease treat­ments, Prime Med­i­cine says the agency’s rhetoric pushed it to move for­ward with a rare im­mune dis­ease ther­a­py it had pre­vi­ous­ly side­lined.

The gene edit­ing com­pa­ny said Tues­day it plans to ask the FDA for ac­cel­er­at­ed ap­proval of a treat­ment for chron­ic gran­u­lo­ma­tous dis­ease that so far gen­er­at­ed promis­ing da­ta in just two pa­tients. It's a change of heart from Prime’s de­ci­sion in May to shelve the ther­a­py be­cause the cost of get­ting it to ap­proval was too high.

The FDA has agreed to the rare step of hold­ing a for­mal ev­i­den­tiary pub­lic hear­ing for Van­da Phar­ma­ceu­ti­cals, in which the com­pa­ny will ar­gue that the agency should not have re­ject­ed its sleep dis­or­der drug Het­lioz for use in treat­ing jet lag.

The hear­ing is the lat­est de­vel­op­ment in a long saga be­tween Van­da and the FDA. The agency first re­ject­ed Het­lioz for jet lag in 2019, and then turned it down again last month af­ter rais­ing con­cerns about Van­da’s clin­i­cal tri­al pro­to­cols. In be­tween those de­nials, the com­pa­ny has filed for­mal dis­pute re­quests, and chal­lenged the FDA's ini­tial de­nial of a hear­ing in court.

No date or agen­da for the hear­ing have been set yet, Van­da's CFO Kevin Moran told End­points News on Tues­day.

The drug pric­ing watch­dog ICER is call­ing on the Cen­ters for Medicare and Med­ic­aid Ser­vices to low­er the price of Takeda's block­buster ul­cer­a­tive col­i­tis and Crohn’s dis­ease drug En­tyvio so that it's on par with J&J's Ste­lara and its biosim­i­lars.

ICER on Tues­day re­leased its mod­el­ing as part of a re­port on the rel­a­tive ad­van­tage of En­tyvio com­pared to Ste­lara and Mer­ck­'s Rem­i­cade and as­so­ci­at­ed biosim­i­lars, find­ing that it should not be priced above Ste­lara but should be priced slight­ly more than Rem­i­cade.

"Cur­rent ev­i­dence sup­ports a 30-day pre­mi­um of $210 to $230 above what CMS is cur­rent­ly pay­ing for in­flix­imab ," ICER wrote. But when com­pared to Ste­lara, En­tyvio "was not as­so­ci­at­ed with im­por­tant health gains in ul­cer­a­tive col­i­tis or Crohn’s dis­ease. Mod­el­ing con­firmed that the ev­i­dence does not sup­port a price pre­mi­um" over Ste­lara.

The FDA has just out­lined which ap­pli­ca­tions might be new­ly el­i­gi­ble for three years of ex­clu­siv­i­ty, in­clud­ing ones where a drug may have been ap­proved in a pre­vi­ous form.

Build­ing off de­f­i­n­i­tions from a 1994 rule, the 14-page draft guid­ance spells out what types of fol­low-on phar­ma ap­pli­ca­tions and clin­i­cal ac­tiv­i­ty would al­low a com­pa­ny to win the three years of clin­i­cal in­ves­ti­ga­tion ex­clu­siv­i­ty — not to be con­fused with the five years of pro­tec­tion for new chem­i­cal en­ti­ties or the sev­en-year ex­clu­siv­i­ty for or­phan drugs treat­ing dis­eases that af­fect few­er than 200,000 peo­ple.

The new dis­t