Vas Narasimhan. AP Images

No­var­tis makes new da­ta in­tegri­ty com­mit­ment

Look­ing to slow the fall­out from No­var­tis’ da­ta ma­nip­u­la­tion fi­as­co with its bil­lion-dol­lar gene ther­a­py Zol­gens­ma (onasemno­gene abepar­vovec-xioi), CEO Vas­ant Narasimhan told an in­vestor con­fer­ence on Mon­day that the com­pa­ny will be more proac­tive in re­port­ing da­ta in­tegri­ty is­sues to the FDA.

“We are vol­un­tar­i­ly and proac­tive­ly tak­ing a pledge with the FDA to en­sure that we will in­form them with­in five busi­ness days of any cred­i­ble al­le­ga­tion re­lat­ed to da­ta in­tegri­ty,” Narasimhan said.

He al­so said No­var­tis has re­spond­ed to the FDA’s Form 483 and is mak­ing doc­u­ments avail­able as re­quest­ed, while re­it­er­at­ing that the da­ta ma­nip­u­la­tion un­cov­ered at the San Diego site “does not im­pact the safe­ty, ef­fi­ca­cy or qual­i­ty of Zol­gens­ma.”

The pledge fol­lows sharp com­ments made last week by act­ing FDA Com­mis­sion­er Ned Sharp­less, who did not men­tion No­var­tis by name, but clear­ly sin­gled the com­pa­ny out in his re­marks be­fore the Re­search!Amer­i­ca 2019 Na­tion­al Health Fo­rum.

While ad­dress­ing da­ta fraud, Sharp­less said: “So it should not be sur­pris­ing to me now at FDA that if peo­ple will lie to get their pa­per pub­lished or get their grant fund­ed, then peo­ple will al­so lie to get a bil­lion dol­lar med­ical prod­uct ap­proved.”

Five De­mo­c­ra­t­ic sen­a­tors (in­clud­ing pres­i­den­tial hope­fuls Bernie Sanders and Eliz­a­beth War­ren) al­so pounced on the da­ta ma­nip­u­la­tion last month, say­ing this “scan­dal smacks of the phar­ma­ceu­ti­cal in­dus­try’s priv­i­lege and greed.” They al­so called on the FDA to hold No­var­tis ac­count­able and they ques­tioned whether FDA has plans to re-is­sue a fi­nal rule on fal­si­fied da­ta that the agency with­drew last Sep­tem­ber.

No­var­tis’ com­mit­ment Mon­day to re­port al­le­ga­tions of da­ta in­tegri­ty trans­gres­sions with­in five busi­ness days brings up an in­ter­est­ing gray area in FDA’s reg­u­la­tions for when com­pa­nies should re­port such is­sues that do not re­sult in se­ri­ous ad­verse events.

As Green­leaf Health ex­perts and for­mer FDA of­fi­cials wrote on the Food and Drug Law In­sti­tute’s web­site, “While it is clear sus­pen­sion or ter­mi­na­tion of the tri­al re­quires the spon­sor to no­ti­fy FDA, spon­sors are of­ten per­plexed about when to no­ti­fy FDA when the pro­to­col non­com­pli­ance is­sue is sig­nif­i­cant, but yet not so sig­nif­i­cant that it re­quires sus­pen­sion or ter­mi­na­tion of the tri­al.”

In ad­di­tion, they note that in the case of emer­gency de­vi­a­tions made to pro­tect the life or well-be­ing of a sub­ject, the FDA must re­ceive no­tice with­in five work­ing days of the de­vi­a­tion, al­though the FDA’s reg­u­la­tions “are not so clear­ly de­fined con­cern­ing when the spon­sor’s du­ty aris­es to re­port a se­ri­ous breach of pro­to­col that does not re­sult in a se­ri­ous ad­verse event, but may cause FDA to ques­tion the re­li­a­bil­i­ty of the da­ta.”

But the ex­perts note that proac­tive re­port­ing to the agency and work­ing with the FDA can be help­ful. Spon­sors “of­ten will find that it is ad­van­ta­geous to re­port da­ta in­tegri­ty is­sues to FDA soon af­ter dis­cov­ery rather than wait­ing for FDA to dis­cov­er the da­ta in­tegri­ty is­sue dur­ing an in­spec­tion,” they added.

RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.


Zachary Brennan

managing editor, RAPS

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

Mer­ck helps bankroll new part­ner Themis' game plan to fin­ish the chikun­gun­ya race and be­gin on­colyt­ic virus quest

As Themis gears up for a Phase III trial of its chikungunya vaccine, the Vienna-based biotech has closed out €40 million ($44 million) to foot the clinical and manufacturing bills.

Its heavyweight partners at Merck — which signed a pact around a mysterious “blockbuster indication” last month — jumped into the Series D, led by new investors Farallon Capital and Hadean Ventures. Adjuvant Capital also joined, as did current investors Global Health Investment Fund, aws Gruenderfonds, Omnes Capital, Ventech and Wellington Partners Life Sciences.