The FDA con­firms that Sarep­ta neme­sis Ronald Farkas has left the agency

The FDA’s Ronald Farkas was one of Sarep­ta’s tough­est crit­ics. When the biotech made a bid to win an ac­cel­er­at­ed ap­proval for their Duchenne mus­cu­lar dy­s­tro­phy drug, the MD from the di­vi­sion of neu­rol­o­gy prod­ucts shot them down at every turn, spurn­ing the biotech’s shaky case that a tiny tri­al with just 12 pa­tients had pro­vid­ed any ev­i­dence to make it wor­thy of be­com­ing the first ap­proved drug to treat the lethal, rare dis­ease.

Now, the neme­sis of thou­sands of Sarep­ta’s ad­vo­cates in the DMD com­mu­ni­ty has left the agency. A spokesper­son con­firmed the news, first re­port­ed by Ben­zin­ga, to End­points News on Wednes­day morn­ing.

“Dr. Farkas no longer works at the agency,” the spokesper­son said in an email state­ment, de­clin­ing to am­pli­fy on the news of his de­par­ture. Soon af­ter, a spokesper­son for Parex­el al­so con­firmed that Farkas had joined the CRO ear­li­er in the month. He’s now vice pres­i­dent, in­te­grat­ed prod­uct de­vel­op­ment, Parex­el Con­sult­ing.

And there’s con­sid­er­able re­joic­ing among eteplirsen ad­vo­cates over spec­u­la­tion that his de­par­ture may clear a com­plex hur­dle on the way to an ap­proval.

Sarep­ta’s in­vestors are tap­ping in­to that sen­ti­ment. The biotech’s shares shot up 26% this morn­ing af­ter ru­mors of his ex­it spread overnight. But the ar­gu­ment over eteplirsen’s fu­ture is still rid­ing on the out­come of a re­cent de­ci­sion to see if the FDA could gath­er one round of con­vinc­ing ev­i­dence that the drug might work as planned.

Sarep­ta was able to make its case twice to the FDA. First for a pan­el meet­ing that was post­poned for weath­er and a sec­ond time in a point-by-point re­but­tal of the FDA’s po­si­tion. None of it worked, though. Farkas and the FDA team didn’t budge, and the pan­el of ex­perts vot­ed against an ap­proval.

The FDA group has proved equal­ly scathing about oth­er at­tempts on DMD. PTC was turned away at the door when the agency re­fused to file its ap­pli­ca­tion, cit­ing some ma­jor weak­ness­es. And Bio­Marin stum­bled bad­ly when they re­ject­ed their drug, set­ting the biotech on course to even­tu­al­ly jet­ti­son the whole pro­gram af­ter the Eu­ro­peans fol­lowed suit.

That left the FDA fend­ing off one of the most ag­gres­sive lob­by­ing cam­paigns it’s ever seen. Pa­tients, fam­i­lies and ad­vo­cates have pushed hard for an ear­ly OK on thin da­ta, in­sist­ing that the drug does work. And that push led to the com­pro­mise so­lu­tion to look for some signs that the drug is hav­ing the in­tend­ed ef­fect in spurring the pro­duc­tion of dy­s­trophin.

As of now, that’s still where the mat­ter sits, leav­ing many an­a­lysts to won­der just how long the FDA plans to take on this. With Farkas out of the pic­ture, ad­vo­cates are hope­ful that they’ll have an ap­proval soon. But there are no guar­an­tees when it comes to the FDA,

UP­DATE: A lit­tle af­ter the mar­kets closed Wednes­day, Jef­feries’ Gena Wang not­ed that FDA staffers in the neu­rol­o­gy di­vi­sion say that Farkas left the FDA a cou­ple of weeks ago. His de­ci­sion to leave, she adds, was prob­a­bly made back in Au­gust, in­di­cat­ing that the de­par­ture had noth­ing to do with the loom­ing de­ci­sion on eteplirsen. And he’s been scout­ing a new po­si­tion for more than a year.

In a chat with an uniden­ti­fied key opin­ion leader in the field, Wang adds that it’s un­like­ly that Farkas’ de­par­ture will make much of a dif­fer­ence in what­ev­er the FDA ul­ti­mate­ly de­cides. The big de­ci­sion like­ly rests large­ly with the di­rec­tor for Of­fice of Drug Eval­u­a­tion, Dr. El­lis Unger. Al­so, don’t as­sume that Farkas’ de­par­ture means an ap­proval is com­ing.

“Ac­cord­ing to our KOL, a longer de­lay from the FDA sug­gests a less like­ly pos­si­bil­i­ty that the agency has been con­vinced with new­ly sub­mit­ted bio­mark­er da­ta, and ad­di­tion­al da­ta could po­ten­tial­ly be re­quest­ed,” Wang adds. “Our KOL al­so not­ed, once the PDU­FA date has been passed, there is no mech­a­nism to force a de­ci­sion with­in a stip­u­lat­ed amount of time.”

It’s worth not­ing that both sides of this ar­gu­ment have of­ten been wrong about next steps for eteplirsen. This is one sto­ry filled with dra­ma and un­cer­tain­ty.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.

The key dates for KRAS watch­ers through the end of the year — the trail is nar­row and risks are ex­treme

There’s nothing quite like a big patent win when it comes to burnishing your prospects in the pipeline. And for Amgen, which seems to have rescued Enbrel for a run to 2029, the cheering section on Wall Street is now fixed on AMG 510 and a key rival.

And it didn’t take much data to do it. 

There was the first snapshot of a handful of patients, with a 50% response rate. Then came word that Amgen researchers are also tracking responses in different cancers, at least one in colorectal cancer and appendiceal too. 

Bain's Or­ly Mis­han joins Pfiz­er's neu­ro spin­out Cerev­el; On­colyt­ic virus biotech taps Sil­la­Jen ex­ec He­le­na Chaye as CEO

→ Bain Capital is deploying one of its top investors to Cerevel Therapeutics, steering a $350 million-plus neuro play carved out of Pfizer. Orly Mishan — a co-founder and principal of Bain’s life sciences unit — was involved in the partnership that birthed the biotech spinout in the first place. As Cerevel’s first chief business officer, she is tasked with corporate development, program management as well as technical operations. 

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.