FDA asks why Novartis took two months to launch formal internal probe, after AveXis flagged data manipulation

August 23, 2019 09:49 AM EDT

FDA asks why Novartis took two months to launch formal internal probe, after AveXis flagged data manipulation

Natalie Grover

Reporter

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Ryan Watts, Denali CEO

August 6, 2020 08:42 AM EDTUpdated 02:14 PM

Deals

Biogen hands Denali $1B-plus in cash, $1B-plus in milestones to partner on late-stage Parkinson’s drug

John Carroll

Editor & Founder

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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Michel Vounatsos, Biogen CEO (via YouTube)

August 7, 2020 07:47 AM EDTUpdated 10:38 AM

R&D

UPDATED: Biogen scores a priority review for its Alzheimer's drug aducanumab, moving one giant leap forward in its controversial quest

John Carroll

Editor & Founder

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Levi Garraway, Roche CMO (Source: Genentech)

August 7, 2020 03:37 PM EDTUpdated 05:07 PM

Regulatory

UPDATED: FDA hands out a quick OK for potential SMA blockbuster risdiplam, giving Genentech and Roche a chance to challenge rivals on the price

Max Gelman

Associate Editor

US regulators handed Roche and Genentech a big win Friday afternoon, one that has market-shaping potential for its high-priced rivals from Novartis and Biogen.

The FDA has green-lit the companies’ spinal muscular atrophy drug risdiplam, which will be marketed as Evrysdi in the US, for use in patients two months and older. It’s the first SMA drug that can be taken orally, as Biogen’s Spinraza is injected into the spine while Novartis’ Zolgensma is a gene therapy.

ENDPOINTS CAREERS

Director of Regulatory Affairs

Recursion Pharmaceuticals

Salt Lake City, UT

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Moncef Slaoui, Getty Images

August 7, 2020 12:54 PM EDT

R&D

Coronavirus

When will it end? Big Pharma's top vaccine expert at OWS offers a speedy timeline for a Covid-19 vaccine — either before or right after the election

John Carroll

Editor & Founder

Moncef Slaoui hasn’t started making plans for his summer vacation next year. But he offers high odds that all Americans will be able to do that in the not too distant future.

In an interview with a pair of sympathetic podcasters at the conservative American Enterprise Institute, Slaoui provides an education to listeners on how any drug or vaccine can be sped through trials. And he leaves the door wide open to the notion that the leading vaccine developers can demonstrate efficacy and safety in a compelling fashion as early as October — or as late as the end of this year.

August 3, 2020 10:53 AM EDT

R&D

Regulatory

J&J gets a fresh OK for esketamine, but is it really the game-changer for depression Trump keeps tweeting about?

John Carroll

Editor & Founder

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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August 7, 2020 10:28 AM EDT

R&D

Perceptive fields SPAC #3 as another group of biotechs scoops up $364M in latest Nasdaq romp

Nicole DeFeudis

Associate Editor

There’s no sign that the windfall of cash dropping biotech’s way on Wall Street is abating. Three more biopharmas priced IPOs on Thursday and Friday morning, riding a historic boom with a $364 million payoff.

London-based biotech Freeline Therapeutics took home the lion’s share of the cash with $159 million after pricing 8,823,529 shares at $18 a pop. Checkmate Pharmaceuticals, of Cambridge, MA, raised $75 million with an offer of 5 million shares at $15 — right at the midpoint of its range. And Arya Sciences Acquisition Corp III, the third in a series from Perceptive, priced 13,000,000 shares at $10 per share.

President Trump (AP Images)

August 7, 2020 07:19 AM EDTUpdated 09:23 AM

Outsourcing

Pharma

Regulatory

UPDATED: FDA takes the lead on defining essential under Trump's 'Buy American' executive order — as pharma warns of supply chain disruption

Amber Tong

Editor

President Donald Trump has signed an executive order detailing how the federal government should help on-shore drug manufacturing — and the FDA will play a central role.

The agency now has three months to draw up the list of “essential medicines, medical countermeasures, and their critical inputs” that the US must have available at all times. Various departments and agencies are then directed to buy these drugs and their ingredients from American manufacturers.

ENDPOINTS CAREERS

Director of Clinical Operations

Recursion Pharmaceuticals

Salt Lake City, UT

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Douglas Fambrough, Dicerna CEO (Boehringer Ingelheim via YouTube)

August 6, 2020 07:01 AM EDTUpdated 10:32 AM

R&D

Roche-backed Dicerna pushes into the pack racing toward the blockbuster hep B goal line, armed with PhI data

John Carroll

Editor & Founder

Dicerna has lined up a set of proof-of-concept data from a small cohort of hepatitis B patients in a match-up against some heavyweight rivals which got out in front of this race. And right in the front row you’ll find a team from Roche, which paid $200 million in cash and offered another $1.5 billion in milestones to partner with Dicerna $DRNA on their RNAi program for hep B.

Right now it’s looking competitive, with lots of big challenges ahead.

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Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

August 3, 2020 01:40 PM EDTUpdated August 4, 03:38 AM

R&D

Coronavirus

When will it end? Goldman economist gives late-stage vaccines a good shot at targeting 'large shares' of the US by mid-2021 — but the downside is daunting

John Carroll

Editor & Founder

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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