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.


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.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

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How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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Gilead claims Tru­va­da patents in HHS’ com­plaint are in­valid

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But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

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Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Tri­fec­ta of sick­le cell dis­ease ther­a­pies ex­tend life ex­pectan­cy, but are not cost-ef­fec­tive — ICER

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.