Sarep­ta’s eteplirsen spurned as Eu­ro­pean reg­u­la­tors turn thumbs down on con­tro­ver­sial Duchenne MD drug

Sarep­ta’s ap­proval for eteplirsen — or Ex­ondys51 — in the US re­mains one of the most con­tro­ver­sial OKs in FDA his­to­ry, earn­ing a rare la­bel that says the biotech has yet to pro­duce ev­i­dence the drug works.

But light­ning won’t strike twice on the reg­u­la­to­ry path­way for this biotech.

The biotech re­port­ed Thurs­day af­ter­noon that the CHMP is prep­ping a neg­a­tive vote, bar­ring the drug from the con­ti­nent. And Sarep­ta shares im­me­di­ate­ly tum­bled 5% in af­ter-mar­ket trad­ing.

Doug In­gram

The drug, which has a sol­id set of sup­port­ers in the DMD com­mu­ni­ty, is get­ting snubbed de­spite the Eu­ro­peans’ ac­cep­tance of PTC Ther­a­peu­tics’ ri­val drug, which has failed re­peat­ed piv­otal stud­ies. But in DMD, reg­u­la­tors have a ten­den­cy to make things up as they go along, fre­quent­ly bend­ing the rules to ac­com­mo­date a pas­sion­ate group of pa­tients and par­ents — or not. PTC has been re­peat­ed­ly slapped down by the FDA, even af­ter Janet Wood­cock over­ruled a host of agency in­sid­ers with her in­sis­tence on ap­prov­ing eteplirsen.

Sarep­ta CEO Doug In­gram had this to say:

Un­for­tu­nate­ly, the CHMP’s trend vote was neg­a­tive. Based on dis­cus­sions with CHMP rep­re­sen­ta­tives, it is our un­der­stand­ing that the CHMP did not con­clude that eteplirsen is in­ef­fec­tive for ex­on 51 amenable pa­tients, but rather that Sarep­ta has not yet met the reg­u­la­to­ry thresh­old for con­di­tion­al ap­proval, in part due to the use of ex­ter­nal con­trols as com­para­tors in the stud­ies. Sarep­ta plans to file for re-ex­am­i­na­tion and will re­quest that a Sci­en­tif­ic Ad­vi­so­ry Group (SAG), which is made up of DMD and neu­ro­mus­cu­lar spe­cial­ists, be con­vened to pro­vide ex­pert guid­ance and in­sight in­to, among oth­er things, the va­lid­i­ty of the ex­ter­nal con­trols used and the im­por­tance of slow­ing pul­monary de­cline in pa­tients with DMD.

Hop­ing to take some of the sting out of the news, while dis­ap­point­ing some an­a­lysts with its Q1 per­for­mance, Sarep­ta al­so an­nounced a deal to part­ner with My­onexus Ther­a­peu­tics on its work de­vel­op­ing gene ther­a­pies for Limb-gir­dle mus­cu­lar dy­s­tro­phies. Sarep­ta paid $60 mil­lion up­front and of­fered $45 mil­lion more in mile­stones for the deal, which al­so pro­vides the biotech with a buy­out op­tion at proof-of-con­cept.

“My­onexus and its fo­cus on gene ther­a­py us­ing the AAVrh.74 vec­tor to treat forms of LGMD aligns bril­liant­ly with our vi­sion to emerge as one of the most mean­ing­ful glob­al pre­ci­sion ge­net­ic med­i­cine com­pa­nies by fo­cus­ing on the use of ge­net­ic med­i­cine to im­prove the lives of those with rare fa­tal dis­eases,” said In­gram in a pre­pared state­ment.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Tim Van Hauwermeiren, argenx CEO

Ar­genx pur­chas­es $100M+ FDA pri­or­i­ty re­view vouch­er from blue­bird bio

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”