Sarep­ta rock­ets up af­ter FDA ap­proves its con­tro­ver­sial Duchenne drug

Af­ter years of fierce de­bate in a see­saw bat­tle over the fate of Sarep­ta’s Duchenne mus­cu­lar dy­s­tro­phy drug eteplirsen, the FDA has grant­ed the drug con­di­tion­al ap­proval in the US, mak­ing this the first drug OK’d to treat the lethal dis­ease.

Janet Wood­cock

The ap­proval comes with the threat of swift ret­ri­bu­tion, though, if in­ves­ti­ga­tors fail to fol­low through with pos­i­tive da­ta from a big­ger study. The agency’s state­ment in­cludes a re­quire­ment that Sarep­ta will now have to con­firm that the drug ac­tu­al­ly works as de­signed, spurring pro­duc­tion of dy­s­trophin in a way that can im­prove mo­tor func­tion in the 13% of pa­tients amenable to ex­on 51 skip­ping. And the agency says that it may with­draw its OK with­out that da­ta.

For now, though, Sarep­ta will move fast to start mar­ket­ing the drug.

The agency’s de­ci­sion came months af­ter a pan­el of out­side ex­perts vot­ed against an ap­proval and FDA in­sid­ers, no­tably Ron Farkas, who re­cent­ly left the agency, re­ject­ed the biotech’s ar­gu­ment at every turn this year. Par­ents and pa­tients, though, turned out in the hun­dreds to voice their full sup­port for eteplirsen, mount­ing a lob­by­ing cam­paign that would re­cruit a long list of elect­ed of­fi­cials as well as physi­cians who treat this dis­ease.

Sarep­ta’s $SRPT share price, which has been on a roller coast­er ride at every twist and turn in the eteplirsen saga, ripped up 81% on the news. The drug will now be sold as Ex­ondys 51.

The drug, though, will re­main con­tro­ver­sial. The agency’s Janet Wood­cock over­ruled oth­ers in the FDA in giv­ing this drug a green light, in­clud­ing El­lis Unger, the di­rec­tor of the Of­fice of Drug Eval­u­a­tion, who protest­ed against Wood­cock’s de­ci­sion. And while the drug has its avowed sup­port­ers, a le­gion of crit­ics has not­ed that Sarep­ta nev­er pro­vid­ed the da­ta need­ed to win an ap­proval, with Wood­cock seen as cav­ing to the lob­by­ing cam­paign in a way that would like­ly dog the agency for years to come.

The big de­bate over this drug cen­tered on its tiny pi­lot study, which gath­ered da­ta on on­ly a dozen boys. Sarep­ta says that their in­ves­ti­ga­tors gath­ered clear ev­i­dence that their drug helped boys with this crip­pling, dev­as­tat­ing dis­ease con­tin­ue to walk fur­ther dis­tances than his­tor­i­cal da­ta would con­sid­er pos­si­ble with­out a ther­a­peu­tic ef­fect. FDA in­sid­ers like Farkas, though, re­ject­ed the his­tor­i­cal com­par­isons and crit­i­cized the biotech for fail­ing to pur­sue a larg­er study ear­li­er.

The ap­proval comes af­ter sev­er­al po­ten­tial ri­vals have been slapped down, with Bio­Marin’s can­di­date re­ject­ed — and now shelved — and PTC’s drug re­fused a hear­ing af­ter in­sid­ers at the agency con­clud­ed that the com­pa­ny sim­ply had not made its case.

Those ear­li­er re­fusals leave Sarep­ta with a clear shot at its mar­ket.

Janet Wood­cock, the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search di­rec­tor, said:

“Pa­tients with a par­tic­u­lar type of Duchenne mus­cu­lar dy­s­tro­phy will now have ac­cess to an ap­proved treat­ment for this rare and dev­as­tat­ing dis­ease. In rare dis­eases, new drug de­vel­op­ment is es­pe­cial­ly chal­leng­ing due to the small num­bers of peo­ple af­fect­ed by each dis­ease and the lack of med­ical un­der­stand­ing of many dis­or­ders. Ac­cel­er­at­ed ap­proval makes this drug avail­able to pa­tients based on ini­tial da­ta, but we ea­ger­ly await learn­ing more about the ef­fi­ca­cy of this drug through a con­fir­ma­to­ry clin­i­cal tri­al that the com­pa­ny must con­duct af­ter ap­proval.”

 

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Deborah Dunsire. Lundbeck

Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation for a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.