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.

 

George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.

Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.

An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.

New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

On a roll, Mer­ck blazes through a new seg­ment of the bio­mark­er trail

Merck has notched an approval for using Keytruda to treat a biomarker-based subset of first-line colorectal cancer patients with unresectable or metastatic tumors, as the pharma giant continues to find new niches for its blockbuster PD-1 star.

The OK is significant in a number of ways. Not only does it build on an accelerated approval for all tumors characterized as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR); it also marks the first single treatment for colorectal cancer that doesn’t contain chemotherapy.

Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.

Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi preps an­oth­er round of lay­offs as new ex­ecs look to slim down the glob­al phar­ma in an on­go­ing re­or­ga­ni­za­tion — re­port

Sanofi is reportedly once again sharpening up the budget axe as the pharma giant prepares to chop more jobs.

Reuters reports this morning that Sanofi has its sights set on cutting 1,680 jobs in Europe, where the unions have been combating rounds of cutbacks at the Paris-based player. Sanofi itself hasn’t said anything about these cuts, as the company looks to discuss a 3-year plan with staff representatives.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.

Covid-19 roundup: Vac­cines will need to beat place­bo by 50% to qual­i­fy for FDA OK; UK tri­al drops Kale­tra

The FDA will set the bar for approving a Covid-19 vaccine at 50% efficacy, the Wall Street Journal reported, meaning any successful candidate will have to reduce the risk of coronavirus disease by at least half compared to placebo.

That requirement is part of guidance that the agency is set to release later today, laying out detailed criteria for vaccine developers — some of whom are eyeing an OK by the end of the year, in line with expectations at Operation Warp Speed.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 84,700+ biopharma pros reading Endpoints daily — and it's free.