FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typ­i­cal­ly re­leas­es guid­ance to help gener­ic drug man­u­fac­tur­ers de­vel­op new copy­cats of small mol­e­cule drugs, of­ten­times in prepa­ra­tion for a brand name prod­uct’s patents or ex­clu­siv­i­ty to ex­pire.

This week, FDA re­leased such bioe­quiv­a­lence guid­ance for any gener­ic drug­mak­ers look­ing to take on Sarep­ta’s Duchenne mus­cu­lar dy­s­tro­phy (DMD) drug Ex­ondys 51 (eteplirsen), even though the drug’s spon­sor has yet to con­vert the ac­cel­er­at­ed ap­proval to a full ap­proval, show­ing clin­i­cal ben­e­fit.

A com­pa­ny spokesper­son told End­points News that they’re not aware of any eteplirsen gener­ics in de­vel­op­ment, adding to the guid­ance:

This has been list­ed on FDA’s planned prod­uct-spe­cif­ic guid­ance for 2022 for quite some time so there is very lit­tle to say.  With re­gard to the post­mar­ket­ing com­mit­ments – MIS­SION is on­go­ing and we con­tin­ue to re­lease new analy­ses and da­ta. We re­cent­ly re­leased re­al world ev­i­dence for eteplirsen at the World Mus­cle So­ci­ety con­fer­ence show­ing a greater than 5 year sur­vival ben­e­fit in pa­tients tak­ing eteplirsen.

DMD is a rare mus­cle dis­or­der af­fect­ing ~1 in 3,500 male births world­wide, with few treat­ment op­tions, but loss of mus­cle func­tion starts ear­ly, and loss of mo­bil­i­ty can oc­cur at about 12 years old, with pre­ma­ture deaths of­ten oc­cur­ring in the third or fourth decade of life, ac­cord­ing to a new re­port from SVB Se­cu­ri­ties on the ex­on skip­ping land­scape for DMD.

Ex­ondys 51 is one of three Sarep­ta ac­cel­er­at­ed ap­provals in this space that are still await­ing com­plet­ed con­fir­ma­to­ry tri­als and full ap­provals, and it re­mains un­known if all will cross that fin­ish line.

“More broad­ly, ques­tions re­main on the mag­ni­tude of clin­i­cal ben­e­fit from very low lev­els of dy­s­trophin pro­vid­ed by al­ready ap­proved as­sets,” SVB an­a­lysts wrote. That was one of the key ques­tions at the FDA ad­comm that vot­ed 6-7 against the idea that eteplirsen in­duces pro­duc­tion of dy­s­trophin to a lev­el that is rea­son­ably like­ly to pre­dict clin­i­cal ben­e­fit.

Sarep­ta’s ap­proved ex­on skip­ping oligonu­cleotides that have won ac­cel­er­at­ed ap­provals (Ex­ondys 51, Vyondys 53 and Amondys 45) have shown <1% mean dy­s­trophin in­creas­es by west­ern blot, ac­cord­ing to SVB Se­cu­ri­ties, but Nip­pon Shinyaku’s ap­proved Vil­tep­so (vil­to­larsen) showed a 0.76% in­crease at week 12 and a 5% in­crease at week 24, at 80 mg/kg. Sarep­ta’s next-gen­er­a­tion SRP-5051 (vesleteplirsen) yield­ed 3.06% at week 12 at 20mg/kg (n=2) and 6.55% at 30 mg/kg (n=4), SVB not­ed.

“These as­sets pushed up the bar for a dif­fer­en­ti­at­ing/com­mer­cial­ly vi­able read­out in the in­di­ca­tion, a sharp dif­fer­ence from the time when SRPT’s first gen­er­a­tion as­sets were grant­ed ac­cel­er­at­ed ap­proval when no oth­er op­tions were avail­able,” SVB added.

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.

Paul Hudson, Sanofi CEO (ROMUALD MEIGNEUX/Sipa via AP Images)

Sanofi and Am­gen are bring­ing cash to cov­er the ta­ble stakes for the Hori­zon M&A game

With the market cap on Horizon Therapeutics $HZNP pushed up to the $23 billion mark today, one of the Big Pharmas in the hunt for a major league buyout deal signaled it’s playing the M&A game with cash.

Paris-based Sanofi, where CEO Paul Hudson has been largely focused on some risky biotech acquisitions to win some respect for its future pipeline prospects, issued a statement early Friday — complying with rule 2.12 of the Irish takeover rules — making clear that while the certainty or size of an offer can’t be determined, any offer “will be solely in cash.” And Amgen CEO Robert Bradway came right in behind him, filing a statement on the London Stock Exchange overnight that any offer they may make will “likely” be in cash as well.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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