Novartis scores its latest FDA OK — this time for a new sickle cell disease drug picked up in a $665M deal

(Image: Associated Press)

November 15, 2019 03:50 PM ESTUpdated 04:01 PM

Novartis scores its latest FDA OK — this time for a new sickle cell disease drug picked up in a $665M deal

John Carroll

Editor & Founder

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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Optimizing Cell and Gene Therapy Development and Production: How Technology Providers Like Corning Life Sciences are Spurring Innovation

Corning Life Sciences

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

May 18, 2022 10:57 AM EDTUpdated 03:04 PM

R&D

Cell/Gene Tx

Pfizer, Sarepta and two others suggest Duchenne drug safety issues tied to "class effect"

Max Gelman

Senior Editor

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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May 17, 2022 10:13 AM EDT

Bioregnum

Presenting a live Endpoints News event: Managing a biotech in turbulent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

ENDPOINTS CAREERS

Senior Finance Associate

Atlas Venture

Cambridge, MA, USA

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Pearl Huang, former Cygnal Therapeutics president and CEO

May 18, 2022 10:28 AM EDTUpdated 01:01 PM

R&D

UPDATED: Flagship builds a new startup out of pieces from 2 of its biotechs. And a Roche vet leaves to do something new

John Carroll

Editor & Founder

Flagship has crafted a new startup out of pieces from a pair of fledglings in the VC’s nest. And a prominent Roche veteran who ran one of the biotechs won’t be making the next leg of the journey.

The new company is called Sonata Therapeutics, which is picking up the work that Inzen was doing related to the cellular microenvironment and combining with Flagship’s Cygnal Therapeutics, which came out of stealth more than 3 years ago and put Pearl Huang — the BeiGene founder and former Roche SVP — at the helm.

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May 17, 2022 10:02 AM EDT

R&D

Manufacturing

Amidst R&D reshuffle, Vertex expands its presence in Boston, aiming to become number one

Tyler Patchen

News Reporter

Vertex Pharmaceuticals has been one of the buzzier names in the bustling Boston biotech scene, but now the company is looking to vault to number one status — at least in terms of physical footprint.

At a ribbon cutting on Tuesday for its new Jeffrey Leiden Center for Cell and Genetic Therapies at the Boston Seaport, Vertex announced it would embark on a new project: The company will build a 344,000 square foot facility in the seaport to accommodate the company’s growing R&D needs, especially in its cell and gene therapies program.

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Martin Shkreli (Dennis Van Tine/MediaPunch/IPX)

May 18, 2022 02:22 PM EDT

People

Infamous biotech exec Martin Shkreli gets out of prison, hits the street

John Carroll

Editor & Founder

Martin Shkreli, the infamous biotech CEO who made headlines for his jeering assault on a legion of critics in and out of Congress, is back on the streets after 4 years inside a federal penitentiary.

Shkreli’s attorney put out a statement Wednesday afternoon saying that the “pharma bro” had been transferred to a halfway house in New York with a few more months to go under federal custody, slated to end September 14. Attorney Benjamin Brafman acknowledged the release and vowed that he and Shkreli are keeping quiet.

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May 18, 2022 01:59 PM EDTUpdated 03:20 PM

Pharma

Despite federal efforts to diversify clinical trials, progress remains 'stagnant' — report

Nicole DeFeudis

Editor

While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report from the National Academies of Sciences, Engineering and Medicine.

Swaths of patients in racial and ethnic minority groups, as well as LGBTQIA+, pregnant and older adult populations continue to be left out of clinical trials. While some advances have been made in the last 30 years — women now account for roughly half of clinical trial participants — growth in other areas remains stagnant, according to the report, which was mandated by Congress and sponsored by the NIH.

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Senior Research Scientist, Platform Biology

Siduma Therapeutics

New Haven, CT, USA

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Paul Chaplin, Bavarian Nordic president and CEO

May 18, 2022 01:37 PM EDTUpdated 01:48 PM

Deals

Pharma

Manufacturing

Bavarian Nordic secures BARDA contract for smallpox vaccine

Tyler Patchen

News Reporter

It seems that smallpox vaccination production is weighing on the mind of the US government. And manufacturer Bavarian Nordic is the latest company to benefit.

Just a few days after Emergent, a company that has made government contracts its lifeblood, acquired the exclusive rights to Tembexa from Chimerix, with a $225 million cash payment and an expected BARDA contract, the agency has offered a contract for smallpox vaccine production.

Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

May 18, 2022 12:34 PM EDT

FDA+

House committee unanimously advances FDA user fee legislation with accelerated approval tweaks

Zachary Brennan

Senior Editor

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.