The next Em­flaza: Ac­er Ther­a­peu­tics looks to take a page out of Marathon's book

Last year, Marathon Phar­ma­ceu­ti­cals took an in­ex­pen­sive steroid avail­able for sale in the EU and Cana­da and brought it through the US FDA ap­proval process be­fore trum­pet­ing an $89,000 an­nu­al price.

The move, crit­i­cized by law­mak­ers and oth­ers, of­fered a test case for how a com­pa­ny can use FDA’s ap­proval sys­tem and the US pric­ing sys­tem to reap out­sized re­wards with­out do­ing any of the de­vel­op­ment work.

Frank David

Now, ac­cord­ing to a new preprint from phar­ma­ceu­ti­cal con­sul­tants at the firm Phar­mag­el­lan, an­oth­er com­pa­ny called Ac­er Ther­a­peu­tics, chaired by the cur­rent CEO of Mar­tin Shkre­li’s old com­pa­ny Retrophin, ac­quired celipro­lol, a cheap be­ta block­er long avail­able out­side the US but nev­er sub­mit­ted for FDA ap­proval. Ac­er has said it will seek FDA ap­proval for the drug this year.

“If Ac­er launch­es Ed­si­vo [celipro­lol] at a high US price (a log­i­cal bet), this will be a reprise of last year’s Em­flaza to-do,” Phar­mag­el­lan man­ag­ing di­rec­tor Frank David said. “Ac­er bought the da­ta rights and ob­tained or­phan des­ig­na­tion, but has not con­duct­ed fur­ther tri­als as far as we can tell.”

An Ac­er ex­ec­u­tive told Fo­cus via email: “No com­ment.”

What Can be Done?

Look­ing to ad­vance the dis­cus­sion on how to pre-emp­tive­ly iden­ti­fy and block such be­hav­ior, David and Richa Dix­it said “al­ter­ations to FDA’s ex­ist­ing rules on per­son­al im­por­ta­tion” may be one so­lu­tion.

“Re­fin­ing these cri­te­ria to al­low Amer­i­cans to im­port gener­ics in cer­tain sit­u­a­tions where a drug with the same ac­tive in­gre­di­ent is al­ready avail­able in the US – for ex­am­ple, if the gener­ic was launched abroad be­fore the first FDA ap­proval – would per­mit con­tin­ued im­por­ta­tion of drugs like de­flaza­cort and celipro­lol, and thus elim­i­nate the in­cen­tive for fu­ture com­pa­nies to take a sim­i­lar ap­proach,” they write.

And though FDA has been re­sis­tant in the past to al­ter­ing im­por­ta­tion or re-im­por­ta­tion poli­cies out of sup­ply chain safe­ty con­cerns, David told Fo­cus he thinks these are unique sit­u­a­tions.

“We al­ready al­low pa­tients to im­port drugs un­avail­able in the US for per­son­al use (al­though I reck­on this is cum­ber­some, and it’s ob­vi­ous­ly not cov­ered by pay­ers),” David said via email. “So in prin­ci­ple, FDA al­ready ac­knowl­edges it’s ap­pro­pri­ate to have a path­way for pa­tients to ac­cess drugs from abroad that aren’t ap­proved here, and ac­cepts the trade­offs. The is­sue with celipro­lol and de­flaza­cort is that un­der our cur­rent poli­cies, the abil­i­ty to per­son­al­ly im­port halts when/if the drug is ap­proved by FDA. That’s cer­tain­ly ap­pro­pri­ate in the case of on-patent meds, but here we’re talk­ing about drugs that have al­ready gone gener­ic.”

In ad­di­tion, drug pric­ing scan­dals tend to get lumped to­geth­er. But the Em­flaza ex­am­ple should not be mis­con­strued as the same as say, Shkre­li’s old com­pa­ny Tur­ing Phar­ma­ceu­ti­cals ac­quir­ing Dara­prim (pyrimethamine) and jack­ing up the price over night.

“Here, we fo­cused on a nar­row sub­set, epit­o­mized by Em­flaza: old, cheap drugs on­ly avail­able in the EU that are then brought to the US by third par­ties and get the pric­ing and patent ben­e­fits of ‘in­no­va­tor’ drugs. These aren’t go­ing to be com­mon – we on­ly found one loom­ing ex­am­ple in our work! – but they’re unique com­pared with dara­prim, etc., and so­lu­tions to those lat­ter sit­u­a­tions aren’t go­ing to pre­vent fu­ture Em­flaza-like sce­nar­ios,” David said.

First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email for more in­for­ma­tion.


Zachary Brennan

managing editor, RAPS

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