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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Biotech and Big Pharma: A blueprint for a successful partnership

Noël Brown

Managing Director and Head of US Biotechnology Investment Banking

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

Credit: Shutterstock

October 26, 2021 08:03 AM EDTUpdated 53 minutes ago

China

In Focus

How China turned the tables on biopharma's global dealmaking

Amber Tong

Senior Editor

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

October 26, 2021 10:23 AM EDT

R&D

Pharma

Novartis dumps AveXis program for Rett syndrome after failing repeat round of preclinical testing

Paul Schloesser

Editorial Intern

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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ENDPOINTS CAREERS

Vice President, Investor Relations

Sage Therapeutics

Boston, MA, USA

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October 26, 2021 07:16 AM EDTUpdated 07:51 AM

R&D

FDA+

FDA is much worse than its regulatory peers at proactively disclosing data, researchers find

Zachary Brennan

Senior Editor

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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October 26, 2021 04:52 PM EDT

R&D

Angion's organ damage drug strikes out again, this time in high-risk kidney transplant patients

Kyle Blankenship

Managing Editor

After flopping a test in Covid-19 earlier this year, Angion’s lead organ damage drug has now hit the skids again in kidney transplant patients.

Angion and partner Vifor Pharma’s ANG-3777 failed to beat out placebo in terms of improving eGFR, a measure of kidney function, in patients who had received a deceased donor kidney transplant and were at high risk of developing what is known as delayed graft function, according to Phase III results released Tuesday.

(Photo courtesy Pfizer)

October 26, 2021 04:29 PM EDTUpdated 04:45 PM

FDA+

Coronavirus

FDA's vaccine adcomm votes almost unanimously in favor of Pfizer's Covid-19 vaccine for younger children

Zachary Brennan

Senior Editor

The FDA’s Vaccines and Related Biological Products Advisory Committee on Tuesday voted 17-0, with one panelist abstaining, that the benefits of the Pfizer-BioNTech Covid-19 vaccine outweigh the risks for children between the ages of five and 12.

The vote will likely trigger a process that could allow the shots to begin rolling out as early as next week.

The vaccine, which is one-third of the adult Pfizer dose, proved to be about 90% effective in a placebo-controlled trial in which about 1,500 kids in this age range received the vaccine, and only about 12% of those receiving the vaccine had any adverse event. All serious adverse events in the trial were unrelated to the vaccine.

An image of Alzheimer's brain tissue. The red show gingipains, a protein from P. gingivalis, intermixing with neurons (yellow) and glial cells (green)

October 26, 2021 04:00 PM EDTUpdated 04:18 PM

R&D

An Alzheimer's darkhorse fails its first big trial, but offers hope for a long-overlooked hypothesis

Jason Mast

Editor

Three years ago, Cortexyme emerged out of obscurity with some big-name backers and an unorthodox approach to treating Alzheimer’s.

They moved their drug into a pivotal study the next year, offering one of the first major tests for a hypothesis that has fluttered on the outskirts of Alzheimer’s research for decades: that, in many cases, the disease is driven by infectious agents — the havoc they wreak in the brain and the inflammation the body uses to try to fend them off. And that quashing the infection could slow patients’ cognitive decline.

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ENDPOINTS CAREERS

Account Supervisor, Healthcare

New York, NY, USA

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Peter Nell, Mammoth Biosciences CBO

October 26, 2021 09:00 AM EDTUpdated 10:34 AM

Deals

Cell/Gene Tx

UPDATED: Jennifer Doudna spinout inks a Mammoth CRISPR deal with Vertex worth nearly $700M

Nicole DeFeudis

Editor

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

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October 25, 2021 06:32 AM EDTUpdated 07:01 AM

R&D

Novartis' second attempt to replicate a stunning cancer result falls flat

Jason Mast

Editor

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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