Joe Wiley, Amryt CEO

Am­ryt earns a pri­or­i­ty re­view in a rare dis­ease that makes pa­tients' skin frag­ile as but­ter­fly wings

A year af­ter flash­ing mixed Phase III da­ta, an Irish drug­mak­er no­to­ri­ous for own­ing some of the coun­try’s most ex­pen­sive drugs is get­ting an ex­pe­dit­ed look at its treat­ment for epi­der­mol­y­sis bul­losa (EB) — a rare dis­ease where a pa­tient’s skin be­comes as frag­ile as but­ter­fly wings.

The FDA grant­ed pri­or­i­ty re­view to Am­ryt’s Oleogel-S10, ac­cel­er­at­ing the drug’s as­sess­ment time from 10 months to six months, the com­pa­ny said on Thurs­day. The agency set its PDU­FA date for Nov. 30, and CEO Joe Wi­ley said launch plans are “well ad­vanced.”

Am­ryt orig­i­nal­ly picked up the drug, al­so known as episal­van, from the small Ger­man drug­mak­er Birken. The drug was al­ready EMA-ap­proved for par­tial thick­ness wounds, but Am­ryt saw po­ten­tial in EB and rushed it in­to a Phase III tri­al al­most im­me­di­ate­ly.

The FDA’s de­ci­sion will be based on da­ta from Am­ryt’s EASE tri­al, in which 41.3% of pa­tients who re­ceived Oleogel-S10 saw first com­plete clo­sure of EB tar­get wounds with­in 45 days. That com­pares to 28.9% of pa­tients in the con­trol group, trans­lat­ing to a 44% in­crease in the prob­a­bil­i­ty of tar­get wound clo­sure (and a p-val­ue of 0.013), ac­cord­ing to Am­ryt.

The drug missed on key sec­ondary end­points, though, in­clud­ing the pro­por­tion of com­plete­ly closed tar­get wounds with­in the 90-day treat­ment pe­ri­od. In­ves­ti­ga­tors say they saw a greater re­duc­tion of to­tal body wound bur­den in those on Oleogel-S10 — mea­sured by a com­mon­ly used scale and to­tal sur­face area of EB par­tial thick­ness wounds — but the dif­fer­ence wasn’t sta­tis­ti­cal­ly sig­nif­i­cant.

At the time, Wi­ley cel­e­brat­ed the re­sults as a “sig­nif­i­cant mile­stone” for the com­pa­ny, and he’s al­ready brand­ed the EB ver­sion of the drug as Fil­su­vez.

There are cur­rent­ly no ap­proved treat­ments for EB, which af­fects rough­ly 1 in 20,000 peo­ple. Chron­ic and re­laps­ing wounds leave pa­tients vul­ner­a­ble to re­peat­ed in­fec­tions, ac­cord­ing to the FDA, and some end up with fi­bro­sis, de­for­mi­ties and cu­ta­neous car­cino­gen­e­sis.

“Skin le­sions are as­so­ci­at­ed with itch­ing and pain, the lat­ter ag­gra­vat­ed dur­ing dress­ing changes,” the agency said in a 2019 state­ment, up­on re­leas­ing new guid­ance to help com­pa­nies de­vel­op treat­ments.

Sev­er­al oth­ers are work­ing on treat­ments for the rare con­di­tion, in­clud­ing Abeona, which last sum­mer reini­ti­at­ed a Phase III tri­al for its cell ther­a­py af­ter a brief pause due to Covid-19. Krys­tal Biotech said it com­plet­ed en­roll­ment in a Phase III study for its own dy­s­troph­ic EB can­di­date back in March, and ex­pects top-line re­sults in Q4. Cas­tle Creek Phar­ma­ceu­ti­cals bought out Fi­bro­cell Sci­ence back in De­cem­ber 2019, and is now in Phase III with its re­ces­sive dy­s­troph­ic EB can­di­date FCX-007.

Fil­su­vez would make Am­ryt’s fourth ap­proved prod­uct, af­ter Episal­van and two drugs the com­pa­ny picked up from Aege­ri­on: Myalept and Jux­tapid. The lat­ter two have con­sis­tent­ly ranked among the most ex­pen­sive drugs in the US. Myalept now is fourth on GoodRx’s list of the most cost­ly drugs in the coun­try.

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.

Thomas Gad, Y-mAbs Therapeutics founder and interim CEO

FDA re­jects Y-mAbs’ neu­rob­las­toma drug af­ter tak­ing is­sue with clin­i­cal tri­al de­sign

Uncertainty about clinical trial evidence has led the FDA to hand down a complete response letter for Y-mAbs’ neuroblastoma drug, casting a cloud on the future of a candidate that had gone through a long development journey in a rare pediatric cancer.

Y-mAbs said it’s disappointed “but not surprised” given that the agency’s oncology drug advisory committee had voted 16-0 against its drug’s approval a few weeks ago.

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Raul Rodriguez, Rigel Pharma CEO

Rigel Phar­ma scores FDA ap­proval for leukemia, kick­ing off show­down with Servi­er in IDH1

When Rigel Pharma bought olutasidenib from Forma Therapeutics, it acquired a drug that already secured a PDUFA date at the FDA — for February 2023. But regulators are ready to give their OK sooner than that.

The FDA has approved the IDH1 inhibitor as a treatment for adult patients with relapsed or refractory acute myeloid leukemia who have a susceptible IDH-1 (isocitrate dehydrogenase-1) mutation as detected by an FDA-greenlit test. Rigel will market it as Rezlidhia.

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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Philip Tagari switch­es Am­gen's dis­cov­ery lab for in­sitro's ma­chine learn­ing tools; CEO Joaquin Du­a­to to chair J&J's board

In February, Philip Tagari will take a few days of retirement and then immediately return to industry. He won’t be leading the therapeutics discovery unit for a large biopharma, though.

He’ll trade in his Amgen hat for chief scientist at a machine learning startup that has reeled in hundreds of millions in capital to lay the groundwork for a much-hyped new model of drug discovery that aims to speed up the time to new clinical assets.

Tim Pearson, Carrick Therapeutics CEO

Pfiz­er backs $60M in­fu­sion in­to Car­rick, teams up on breast can­cer treat­ment

In a big week for Carrick Therapeutics, the company announced $60 million in funding for its lead breast cancer drug and development of a second program, as well as a collaboration with Pfizer for combo development.

The $35 million from Pfizer comes with an agreement under which Pfizer will support Carrick’s Phase II study of samuraciclib in combination with Pfizer’s Faslodex for advanced breast cancer. Along with the investment, Adam Schayowitz, vice president and development head of breast cancer, colorectal cancer and melanoma at Pfizer global product development, will join Carrick’s scientific advisory board.

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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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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.”