FDA posed a sprawling set of questions for two-day gene therapy hearing. Here’s what companies and experts will be watching for

September 1, 2021 11:29 AM EDTUpdated 01:44 PM

FDA posed a sprawling set of questions for two-day gene therapy hearing. Here's what companies and experts will be watching for

Jason Mast

Editor

Amber Tong

Senior Editor

Gene therapy is having a moment. Once confined to academic labs and a couple of companies, the field now encompasses hundreds of trials, dozens of companies and billions of investment dollars.

With greater power, though, comes greater responsibility. The FDA is asking a panel of outside experts to try and define those responsibilities as they debate a lengthy list of 15 questions Thursday and Friday, in a two-day marathon meeting on the future of a field that has promised one-time treatments for some of the most famous and fatal diseases known to medicine.

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Why it Works: Manufacturing a Vaccine in a Multi-Product Facility.

Grand River Aseptic Manufacturing

COVID-19 launched the pharmaceutical industry to the frontline in the battle against the fast-spreading global pandemic. The goal: distribute a safe, effective vaccine as quickly as possible. Major players in the vaccine market needed to partner with contract development and manufacturing organizations (CDMOs) to achieve the goal of mass vaccine quantities under expedited timelines. With CDMOs stepping up to play a critical role in the vaccine manufacturing process, multi-product CDMO facilities took the spotlight. Partnerships quickly formed as the race to save lives and fight a pandemic was on.

Alexander Lefterov/Endpoints News

September 27, 2021 07:12 AM EDTUpdated 09:08 AM

Coronavirus

In Focus

The coronavirus vaccine that the world forgot could still help save it

Jason Mast

Editor

Back at the beginning of the pandemic — back when we still called the virus “novel” and a single case in Washington state could make headlines — there emerged the story of the coronavirus vaccine that the world forgot.

It was an allegory for our pandemic ill-preparedness. At a time when the world had been caught so flat-footed, there were a pair of scientists who had seen the crisis coming, lab-coated Cassandras with an antidote if only the world had listened sooner.

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Habib Dable, Acceleron CEO

September 24, 2021 10:09 PM EDTUpdated September 27, 07:15 AM

Deals

R&D

Days of heated rumors culminate in a report that Acceleron is in advanced buyout talks

John Carroll

Editor & Founder

Days of frothy rumors about possible M&A discussions at Acceleron were capped late Friday with a Bloomberg report asserting that the biotech company is in advanced talks for an $11 billion buyout deal.

Bloomberg was unable to identify any bidders in the deal, but speculation has been running rampant that the surging value of Acceleron stock had to be the result of leaks around the auction of the company. As of early Monday morning, we’re still awaiting the final word.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 118,600+ biopharma pros reading Endpoints daily — and it's free.

ENDPOINTS CAREERS

Senior Analyst, Oncology

Brass Tacks Health

Remote

view job offer post your job now

September 28, 2021 10:49 AM EDT

R&D

Cell/Gene Tx

Safety fears force Pfizer to change pivotal DMD gene therapy trial protocol

Kyle Blankenship

Managing Editor

As one of the biggest players in an increasingly packed gene therapy space, Pfizer has taken an early lead over specialists like Sarepta in taking a Duchenne muscular dystrophy (DMD) candidate into late-stage testing. But new safety fears have led Pfizer to scale back that trial, cutting out patients with certain genetic mutations.

Pfizer has amended its enrollment protocol for a Phase III test for gene therapy fordadistrogene movaparvovec in DMD after investigators flagged severe side effects tied to specific mutations, according to a letter the drugmaker sent to Parent Project Muscular Dystrophy, a patient advocacy group.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 118,600+ biopharma pros reading Endpoints daily — and it's free.

Merck CEO Rob Davis

September 27, 2021 06:04 PM EDTUpdated 14 hours ago

Deals

Pharma

Merck emerges as lead bidder in potential Acceleron buyout with deal possible this week — report

Kyle Blankenship

Managing Editor

With rumors swirling about a potential buyout of biotech Acceleron and its lead PAH drug sotatercept, market watchers have been keeping close tabs on industry movers and shakers due up for an expensive bolt-on. According to a new report, it appears Merck may be the one.

Merck is in “advanced talks” on a deal to acquire Cambridge, MA-based Acceleron in what previous reports pegged as a potential $11 billion buyout, the Wall Street Journal reported Monday. A deal could come as early as this week, according to the Journal.

Peter Marks (Jim Lo Scalzo/Pool via AP Images)

September 28, 2021 06:51 AM EDTUpdated 08:12 AM

FDA+

Coronavirus

Turmoil at CBER: Peter Marks grabs control of FDA's Office of Vaccines ahead of 2 key career leader departures

Zachary Brennan

Senior Editor

FDA’s top vaccine official Peter Marks is pulling the plug on a months-long transition for two top career vaccine officials who abruptly called it quits in late August.

Marion Gruber, director of the FDA’s Office of Vaccines Research & Review and 32-year veteran of the agency, her deputy director Phil Krause, announced their departures and then raised concerns with Covid-19 booster shots ahead of and during a recent Covid-19 booster vaccine advisory committee.

September 28, 2021 10:57 AM EDTUpdated 03:06 PM

R&D

In Focus

Contract research is having a moment right now. Will M&A splashes drive the industry to even greater heights?

Nicole DeFeudis

Editor

Contract research organizations are a fairly mysterious bunch. They’re typically considered the skilled laborers behind big drug development — the stage crews who run the trials behind some of the most (and least) successful data reveals in biopharma history.

But all that is changing.

This year, a couple of huge, out-of-the-blue M&A deals sounded the alarm on just how much money is flying around in this corner of the industry.

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

ENDPOINTS CAREERS

Senior Scientific Research Analyst

American Association for Cancer Research

Philadelphia, PA, USA

view job offer post your job now

Albert Bourla, Pfizer CEO (John Thys, Pool via AP Images)

September 28, 2021 10:17 AM EDTUpdated 10:39 AM

Coronavirus

Covid-19 roundup: Pfizer/BioNTech submit vaccine data to FDA for younger children; Doctors kept prescribing hydroxychloroquine

Zachary Brennan

Senior Editor

Pfizer and BioNTech said Tuesday they submitted to FDA positive data from a Phase II/III trial of their Covid-19 vaccine in children aged 5 to less than 12 years old.

A formal EUA submission for the vaccine in these children is expected to follow “in the coming weeks,” the companies said in a statement.

The trial of 2,268 healthy participants aged 5 to less than 12 years old showed the vaccine was safe and elicited robust neutralizing antibody responses using a two-dose regimen of 10 μg doses, which is one-third the dose that’s administered to adults.

Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Ciaran McCrickard)

September 27, 2021 07:31 PM EDT

FDA+

Seizing Aduhelm precedent, Biogen, Eisai get the ball rolling on another accelerated Alzheimer's approval

Amber Tong

Senior Editor

Biogen — together with partners at Eisai — will be the first to breeze down that Alzheimer’s trail it blazed with the FDA’s historic and controversial accelerated approval of Aduhelm.

Eisai disclosed it’s initiated a rolling BLA submission for lecanemab, or BAN2401, the anti-amyloid beta antibody that Biogen has long touted as a follow-on to Aduhelm (then aducanumab). Following the precedent that the agency has now set, the two companies are gunning for an OK based on data suggesting that the drug could lower amyloid beta plaques.