FDA clears next-gen tu­mor se­quenc­ing test from Memo­r­i­al Sloan Ket­ter­ing

The FDA on Wednes­day an­nounced it has cleared a next-gen­er­a­tion se­quenc­ing (NGS) test de­vel­oped by Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter that can de­tect 468 unique gene mu­ta­tions and oth­er mol­e­c­u­lar bio­mark­ers in a pa­tient’s tu­mor.

The test, re­ferred to as MSK-IM­PACT (In­te­grat­ed Mu­ta­tion Pro­fil­ing of Ac­tion­able Can­cer Tar­gets), was al­ready ap­proved for use with sam­ples from pa­tients in New York by the New York State De­part­ment of Health un­der its clin­i­cal lab­o­ra­to­ry eval­u­a­tion pro­gram.

In con­junc­tion with the an­nounce­ment, FDA al­so laid out a new frame­work for clear­ing NGS-based tu­mor pro­fil­ing tests based on rec­om­men­da­tions from ac­cred­it­ed third-par­ty re­view­ers, the first of which is NYS­DOH.

“The goal of al­low­ing NGS-based tu­mor pro­fil­ing tests to un­der­go re­view by ac­cred­it­ed third-par­ties is to re­duce the bur­den on test de­vel­op­ers and stream­line the reg­u­la­to­ry as­sess­ment of these types of in­no­v­a­tive prod­ucts,” FDA Com­mis­sion­er Scott Got­tlieb said.

FDA gained the au­thor­i­ty to ac­cred­it third-par­ty re­view­ers un­der the FDA Mod­ern­iza­tion Act to in­crease the ef­fi­cien­cy of re­views for cer­tain de­vices that re­quire a 510(k). The agency cur­rent­ly lists sev­en third-par­ty re­view­ers, in­clud­ing NYS­DOH, that are ac­cred­it­ed to re­view a wide range of Class I and Class II de­vices.

FDA al­so says it has clas­si­fied the IM­PACT test and oth­er NGS-based tu­mor pro­fil­ing tests as Class II de­vices and that lab­o­ra­to­ries that de­vel­op such tests will be able to sub­mit ap­pli­ca­tions to ei­ther FDA or NYS­DOH.

“Mov­ing for­ward, lab­o­ra­to­ries whose NGS-based tu­mor pro­fil­ing tests have been ap­proved by NYS­DOH do not need to sub­mit a sep­a­rate 510(k) ap­pli­ca­tion,” FDA said. Lab­o­ra­to­ries can in­stead for­ward their NYS­DOH ap­pli­ca­tion and the state’s re­view mem­o­ran­dum and rec­om­men­da­tion to FDA to re­ceive 510(k) clear­ance.

FDA re­viewed the IM­PACT test un­der its de no­vo path­way, through which spon­sors can re­quest that the agency clas­si­fy a low- to mod­er­ate-risk de­vice that has no legal­ly mar­ket­ed pred­i­cate de­vice as Class I or Class II.

For its re­view, FDA said it looked at in­for­ma­tion from MSK, in­clud­ing what was sub­mit­ted pre­vi­ous­ly to NYS­DOH “to in­form and ex­pe­dite” its de­ci­sion.

MSK-IM­PACT

The IM­PACT test works by com­par­ing a pa­tient’s tu­mor to a nor­mal tis­sue sam­ple to look for mu­ta­tions in 468 genes and oth­er bio­mark­ers linked to var­i­ous forms of can­cer.

FDA says the IM­PACT test is “high­ly ac­cu­rate” and can iden­ti­fy mu­ta­tions in more genes than any oth­er test it has re­viewed in the past.

How­ev­er, FDA says that while the re­sults of the test can pro­vide valu­able in­sights to pa­tients and physi­cians, its re­sults alone “are not con­clu­sive for choos­ing a cor­re­spond­ing treat­ment.”

As such, the test’s in­di­ca­tions for use state that “the test is in­tend­ed to pro­vide in­for­ma­tion on so­mat­ic mu­ta­tions (point mu­ta­tions and small in­ser­tions and dele­tions) and mi­crosatel­lite in­sta­bil­i­ty for use by qual­i­fied health care pro­fes­sion­als in ac­cor­dance with pro­fes­sion­al guide­lines, and is not con­clu­sive or pre­scrip­tive for la­beled use of any spe­cif­ic ther­a­peu­tic prod­uct.”


First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.

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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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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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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Sanofi takes a $260M hit to ex­tri­cate it­self from a dis­as­trous al­liance with Lex­i­con

Sanofi spent $300 million in cash to get into a $1.7 billion alliance with Lexicon on their SGLT1/2 diabetes drug sotagliflozin. And now that the drug has been spurned by the FDA after burning through a program that provided mixed late-stage data and a late shot at a last-place finish, the French pharma giant is forking over another $260 million to get out of the deal.

Sanofi’s unhappiness was already apparent when the company — now under new CEO Paul Hudson — posted a statement back in July that they were dropping the deal. But it wasn’t that simple. 

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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.