Marathon's cheap, old steroid breezes through the FDA for Duchenne MD, and gets priced at $89K

Jef­frey Aronin, Marathon

For a sim­ple sys­temic steroid that is cheap and eas­i­ly avail­able out­side the US, de­flaza­cort has re­ceived un­usu­al­ly care­ful sup­port from the FDA, breez­ing through a reg­u­la­to­ry process that has be­dev­iled sev­er­al de­vel­op­ers try­ing to ad­dress the root cause of the dis­ease.

De­flaza­cort won a pri­or­i­ty re­view from the agency as a new ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy — cut­ting four months off the reg­u­la­to­ry process — along with an or­phan drug des­ig­na­tion and rare pe­di­atric dis­ease sta­tus. And to­day, the FDA hand­ed Marathon Phar­ma­ceu­ti­cals a green light to start mar­ket­ing the drug to all boys 5 and old­er whose lives are be­ing slow­ly de­stroyed by the dis­ease.

The cor­ti­cos­teroid will be sold as Em­flaza af­ter one study in­volv­ing 196 DMD pa­tients in­di­cat­ed that it could im­prove the strength of pa­tients with this dis­ease. In an­oth­er tri­al with 29 male pa­tients that last­ed 104 weeks, the FDA re­port­ed, “de­flaza­cort demon­strat­ed a nu­mer­i­cal ad­van­tage over place­bo on an as­sess­ment of av­er­age mus­cle strength.”

The larg­er of those two stud­ies was com­plet­ed 21 years ago, ac­cord­ing to an ab­stract post­ed in Neu­rol­o­gy last year.

For that, the com­pa­ny plans to price the drug at $89,000 a year, ac­cord­ing to the Chica­go Tri­bune, more than $88,oo0 a year more than what Cana­di­ans would pay for the same drug. But like oth­er cas­es in­volv­ing price goug­ing, the com­pa­ny went on to de­fend the price, say­ing that in­sur­ers will cov­er most of the price.

In ad­di­tion to the ap­proval, Marathon wins a rare pe­di­atric dis­ease pri­or­i­ty re­view vouch­er. These vouch­ers have fetched hun­dreds of mil­lions of dol­lars from drug com­pa­nies look­ing for a quick and sure way to slash four months off of one of their own drug re­views.

De­flaza­cort has been avail­able in coun­tries around the world for decades.

Cur­rent­ly, you can buy de­flaza­cort from on­line phar­ma­cies in Cana­da for less than a buck a pill. Un­til fair­ly re­cent­ly it was sold by Shire in the UK, for ex­am­ple, as Cal­cort in 6 mg tablets and used to treat a va­ri­ety of chron­ic au­toim­mune con­di­tions like rheuma­toid arthri­tis. (A spokesper­son for Shire says that the drug changed hands.) But be­cause it’s nev­er been ap­proved in Amer­i­ca, where oth­er steroids have been avail­able for those same con­di­tions, it gets the full reg­u­la­to­ry treat­ment in the US.

One Duchenne mom emailed me to say that she felt her fam­i­ly was be­ing held “hostage” by Marathon, af­ter be­ing able to buy the drug for a thou­sand dol­lars a year. And more crit­i­cism be­gan to sur­face on Fri­day, as the Wall Street Jour­nal re­port­ed that Chris­tine Mc­Sh­er­ry, who runs the non­prof­it Jett Foun­da­tion, said she is “dis­ap­point­ed that Marathon in­creased my cost for the drug by more than $87,000 a year.” A num­ber of pa­tient ad­vo­cates said they were wor­ried es­pe­cial­ly for fam­i­lies with high de­ductible in­sur­ance plans.

De­flaza­cort now joins a list of con­tro­ver­sial meds that have been ac­quired and then sold at an as­tro­nom­i­cal markup. Mar­tin Shkre­li be­came the poster child of the pric­ing de­bate with his 5,000% price hike on Dara­prim, an­oth­er old gener­ic drug that tar­gets a rare con­di­tion, which came with­out any ad­di­tion­al re­search. And ex­ec­u­tives at Valeant and My­lan were hauled in front of law­mak­ers last year and grilled on their own pric­ing tac­tics for ag­ing prod­ucts.

None of it is against the law in the US, though, which is one rea­son why it’s so con­tro­ver­sial. The FDA is re­quired by law to be ag­nos­tic on pric­ing is­sues.

“This is the first treat­ment ap­proved for a wide range of pa­tients with Duchenne mus­cu­lar dy­s­tro­phy,” said Bil­ly Dunn, MD, di­rec­tor of the Di­vi­sion of Neu­rol­o­gy Prod­ucts in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search. “We hope that this treat­ment op­tion will ben­e­fit many pa­tients with DMD.”

Up un­til Marathon, though, Duchenne MD has been a com­bat zone at the FDA.

Sarep­ta won a con­tro­ver­sial ap­proval for its DMD drug last year af­ter fight­ing for years to get an OK based on da­ta from a tiny, bad­ly flawed study. Their ap­proval came with a la­bel that tells pa­tients that the com­pa­ny has yet to gain clear ev­i­dence of ef­fi­ca­cy. PTC was shut out, much to their cha­grin. And Bio­Marin‘s dris­apersen was shown the door as well.

Marathon did not re­spond to my queries. The biotech is run by CEO Jef­frey Aronin, who start­ed Ova­tion and sold it to Lund­beck for $900 mil­lion in 2009.

I asked the FDA why the drug war­rant­ed VIP ap­proval. Their re­sponse:

De­flaza­cort has nev­er been ap­proved for any use in the Unit­ed States. Un­der U.S. law, it was re­viewed as a “new drug” and as­sessed for safe­ty and ef­fi­ca­cy for the spe­cif­ic con­di­tions of use in the la­bel­ing (pre­scrib­ing in­for­ma­tion). Ver­sions of de­flaza­cort are avail­able in some coun­tries for oth­er in­di­ca­tions, but not for DMD.  The U.S. ap­proval is the first any­where for DMD. The FDA-ap­proved prod­uct la­bel­ing in­cludes safe­ty and clin­i­cal in­for­ma­tion spe­cif­ic to the drug’s use in DMD.  If a drug meets the statu­to­ry re­quire­ments for or­phan drug des­ig­na­tionex­pe­dit­ed pro­grams, and rare pe­di­atric dis­ease des­ig­na­tion, then a spon­sor is el­i­gi­ble to re­ceive those ben­e­fits.

“We are in a new era in the treat­ment of Duchenne mus­cu­lar dy­s­tro­phy. For the first time, pa­tients in the U.S. with Duchenne will have wide­spread ac­cess to an FDA ap­proved med­i­cine that is in­di­cat­ed for all ge­net­ic forms of the con­di­tion. We are pleased that this de­vel­op­ment will help pa­tients with this dis­ease stay stronger longer,” said Tim­o­thy M. Cun­niff, Pharm.D., Ex­ec­u­tive Vice Pres­i­dent, Re­search & De­vel­op­ment, Marathon Phar­ma­ceu­ti­cals, in a state­ment.

 

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Video Re­play: End­points at #JPM20 — news­mak­ers on deal­mak­ing, pric­ing and man­u­fac­tur­ing

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For a media company on a mission to connect the biopharma world in bigger and better ways, we’re proud of how we were able to extend the reach of our franchise event. Paid subscribers were given access to the stream in real time, and now, two days later, we’re opening it up to everyone in this post.

Endpoints@JPM: (left to right) Steve Pearson, Nick Leschly, Bari Talente, Stephen Ubl, John Carroll

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He cuts a Jeff Bezos figure on stage at the Colonial Room, the JP Morgan presentation hall for A-list biotechs: lean and bald, fast-talking and vest-wearing. He explains in simple language, apologizing when he has to brush on the data. It helps that he has a good story to tell.

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Neon Ther­a­peu­tics makes one last re­treat, sell­ing it­self cheap in a bar­gain base­ment M&A deal

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Mark Pruzanski

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SAN FRANCISCO – It’s not quite Dewey defeats Truman, but Goldman Sachs calling 2019 “The Year of NASH” may well go down in the annals of worst biotech predictions.

Goldman Sachs slapped the label on weeks before 2019’s JP Morgan conference, projecting that long-discussed treatments for the obesity-driven condition suspected to lurk in millions of Americans would begin to bear fruit and investors would move accordingly. That did not quite happen.

“If you look at 2019, it was just a string of disappointing news,” Pascal Prigent, CEO of NASH-focused biotech Genfit, told Endpoints News in an interview.

The Year of NASH, or nonalcoholic steatohepatitis, became a year of NASH failures. Gilead failed two large Phase III trials. CymaBay went from a $1 billion company to a $100 million company after they found their drug was killing patients’ liver cells. Cirius withdrew an $86 million IPO bid after a disastrous readout. Industry-wide, there were few acqusitions in a market often projected to be worth $35 billion.

Gilead, after dominating the NASH discussion at the 2019 JPM, gave one quick mention to the program in their 2020 presentation before pivoting to other drugs.

“As promising as some of the mechanisms looked in earlier stages, when push comes to shove in large study settings, they just haven’t proven out,” Mark Pruzanski, CEO of the NASH-focused biotech Intercept, told Endpoints in an interview.

As biotech turns from 2019, the failures have refocused eyes away from Gilead and back toward two startups, both facing key events in the coming months: Intercept, which first alerted investors to NASH at JPM 2014, and the France-based Genfit.

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