Tag: ALS

Cell and Gene Therapy, FDA, Industry, Regulations

FDA sets advisory meeting date for Brainstorm’s ALS cell therapy

June 6, 2023

Via: Biopharma Dive

The Food and Drug Administration has set a date for an upcoming meeting in which an outside group of experts will evaluate an experimental ALS medicine the agency has appeared wary of approving. The medicine’s developer, Brainstorm Cell Therapeutics, disclosed […]

Cell and Gene Therapy, Industry

FDA Approves Drug for Rare Form of ALS

April 26, 2023


The U.S. Food and Drug Administration on Tuesday approved a new drug for people who have a rare, inherited type of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The medication, toferson (Qalsody), targets a mutation in the […]

FDA, Regulations

BrainStorm files ALS drug application over FDA protest

March 27, 2023

Via: Biopharma Dive

A yearslong disagreement between the Food and Drug Administration and a developer of an experimental medicine for ALS will get a public hearing sometime in the future, setting up another high-profile debate of what amount of evidence merits an approval […]

Cell and Gene Therapy, Industry

UniQure nabs another gene therapy for ALS

January 31, 2023

Via: Biopharma Dive

Apic Bio, a privately held developer of gene therapies for rare disorders, has agreed to sell the rights to one of its experimental ALS treatments to the biotechnology company UniQure in a deal announced Tuesday. Per deal terms, UniQure will […]


NRG announces £16m Series A for IND for Parkinson’s and ALS

November 9, 2022

Via: Pharmaphorum

A pre-clinical pipeline of potential first-in-class brain-penetrant small molecule inhibitors of the mitochondrial permeability pore (mPTP) are to be developed. These will act through a novel mechanism of action and be used in treatment of Parkinson’s and Amyotrophic Lateral Sclerosis […]

Clinical Trials, Research and Development

Longer-Term Use of Investigational Drug Stabilizes ALS Symptoms

September 22, 2022

Via: GEN

In an open-label extension of a Phase III clinical trial sponsored by the pharmaceutical company Biogen, investigators have shown that the use of the drug tofersen for one year may help stabilize muscle strength and motor control in patients with […]

FDA, Regulations

FDA Panel Skeptical of Controversial ALS Drug Ahead of Vote

September 6, 2022


U.S. Food and Drug Administration panel will once again consider approval for an experimental drug for ALS, a rare second review for a disease that has no cure. The same panel that will meet Wednesday voted last March not to […]

Cell and Gene Therapy, Industry

Stem cell-gene therapy shows promise in ALS safety trial

September 5, 2022

Via: World Pharma News

Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially protect diseased motor neurons in the spinal cord of […]

FDA, Regulations

FDA Approves Radicava ORS (edaravone) for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

May 13, 2022


Mitsubishi Tanabe Pharma Corporation (MTPA) today announced the U.S. Food and Drug Administration (FDA) has approved Radicava ORS (edaravone), the oral form of edaravone, for the treatment of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that currently has no cure […]

Clinical Trials, Research and Development

Biogen’s ALS drug has failed phase 3. Will FDA approve anyway?

October 18, 2021

Via: FierceBiotech

Stop me if you think you’ve heard this one before. A Biogen neurodegenerative disease prospect has failed a pivotal study, but the Big Biotech has latched onto biomarker data and “trends favoring” the treatment as it heads into talks with […]

Clinical Trials, Research and Development

Bad news for Biohaven as verdiperstat flunks first phase 3 test

September 28, 2021

Via: Pharmaphorum

Myeloperoxidase (MPO) inhibitor verdiperstat was no better than placebo in the M-STAR trial in patients with MSA, and looks likely to be abandoned for this indication. It’s not the end of the line for the drug, which Biohaven picked up […]