expanded access program

People with amyotrophic lateral sclerosis (ALS) who continued treatment with NurOwn (debamestrocel) after completing a Phase 3 trial experienced additional benefits with extended treatment, data show. The findings come from 10 adults who received up to six additional doses of NurOwn over nearly one year in an expanded…

Amyotrophic lateral sclerosis (ALS) patients not eligible to participate in clinical trials of pridopidine — a small molecule developed by Prilenia Therapeutics to slow disease progression in ALS — will soon be able to receive the experimental therapy via an expanded access program (EAP). EAPs, also known…

The U.S. Food and Drug Administration (FDA) has published on its website a five-year action plan meant to help advance the development of — and access to — treatments that may make life better and longer for people with amyotrophic lateral sclerosis (ALS) or other rare neurodegenerative diseases. The…

Amylyx Pharmaceuticals has launched an expanded access program (EAP) in the U.S. that enables eligible adults with amyotrophic lateral sclerosis (ALS) to access the company’s experimental treatment, AMX0035. To be eligible for the program (NCT05286372), patients must have experienced their first signs of weakness three years or…

Amyotrophic lateral sclerosis (ALS) patients who completed the expanded access protocol (EAP) for NurOwn may soon be eligible for three additional doses of the cell-based therapy, BrainStorm Cell Therapeutics reported. EAPs, also known as compassionate use programs, are intended to make investigational therapies available outside of a clinical trial…

Clene, and its wholly owned subsidiary Clene Nanomedicine, have launched an expanded access program (EAP) in the U.S. to allow certain people with amyotrophic lateral sclerosis (ALS) to gain access to its experimental oral therapy CNM-Au8. Called CNMAu8.EAP02, the program will focus on patients who are not eligible…

BrainStorm Cell Therapeutics has opened an expanded access program (EAP) in the U.S. to allow certain amyotrophic lateral sclerosis (ALS) patients to gain access to its investigational cell-based therapy NurOwn. EAPs, also known as compassionate use programs, are intended to make investigational therapies available outside of a clinical…

After gathering input from patients, researchers, and advocates, the U.S Food and Drug Administration (FDA) has released new guidance on the development of therapies for amyotrophic lateral sclerosis (ALS). Among its recommendations, it is advising more communication with companies early on in the product development process, access to…

Biohaven Pharmaceuticals has established an expanded access program (EAP) for patients with amyotrophic lateral sclerosis (ALS) for its investigational treatment candidate, BHV-0223, the company recently announced. The United States Food and Drug Administration regulates the expanded access program to help provide access to therapies that are currently in development and have…