Night­star's drug for rare vi­sion loss ad­mit­ted to FDA's gene ther­a­py speed­way

In the lat­est show of reg­u­la­to­ry sup­port for gene ther­a­pies, Ox­ford spin­out Night­star Ther­a­peu­tics just got ac­cess to a fair­ly new “fast-lane” to speed its reti­nal gene ther­a­py to mar­ket.

The Lon­don-based com­pa­ny scored an FDA des­ig­na­tion called Re­gen­er­a­tive Med­i­cine Ad­vanced Ther­a­py, or RMAT, for its Phase III drug NSR-REP1. The ther­a­py is de­signed to treat choroi­deremia, a rare ge­net­ic con­di­tion that caus­es pro­gres­sive vi­sion loss, by cor­rect­ing mu­ta­tions in the CHM gene.

David Fel­lows

“Re­ceiv­ing RMAT des­ig­na­tion for NSR-REP1 high­lights the po­ten­tial of this gene ther­a­py to main­tain and im­prove vi­su­al acu­ity in choroi­deremia,” said Dave Fel­lows, Night­star’s CEO, in a state­ment. “This des­ig­na­tion fur­ther un­der­scores a recog­ni­tion of the se­ri­ous na­ture of choroi­deremia and the ur­gent need to de­vel­op new treat­ments for those af­fect­ed by in­her­it­ed reti­nal dis­eases that would oth­er­wise lead to blind­ness. We look for­ward to work­ing close­ly with the FDA to dis­cuss the NSR-REP1 de­vel­op­ment pro­gram and to de­ter­mine how we can ac­cel­er­ate the path­way for mak­ing NSR-REP1 avail­able to choroi­deremia pa­tients.”

RMAT in­cludes all the ben­e­fits of the fast track and break­through des­ig­na­tions, in­clud­ing ear­ly in­ter­ac­tions be­tween the FDA and spon­sors. Back when RMAT was first cre­at­ed (as part of the 21st Cen­tu­ry Cures Act), drug­mak­ers wor­ried gene ther­a­pies would be ex­clud­ed. That’s be­cause the FDA didn’t ex­plic­it­ly in­clude gene ther­a­pies in the first de­scrip­tion of the des­ig­na­tion. The agency reme­died that, how­ev­er, last No­vem­ber.

Un­like the break­through des­ig­na­tion, RMAT doesn’t re­quire ev­i­dence that the drug may of­fer a sub­stan­tial im­prove­ment over avail­able ther­a­pies. And like break­through des­ig­na­tions, get­ting an RMAT des­ig­na­tion doesn’t mean the prod­uct will be ap­proved and does not change the stan­dards for safe­ty and ef­fec­tive­ness need­ed for ap­proval.

RMAT des­ig­na­tion for NSR-REP1 was based on clin­i­cal da­ta sup­port­ing the main­te­nance and im­prove­ment of vi­su­al acu­ity from com­plet­ed Phase I/II tri­als in choroi­deremia pa­tients, the com­pa­ny said. The drug is now in Phase III tri­als.

Night­star’s stock $NITE, which first list­ed on Nas­daq last Sep­tem­ber, is up near­ly 6% on the news.

 

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.

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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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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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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.”