Therapy Approval Process*
Experimental therapies following standard pathways can be approved by one of two different FDA-led review bodies - CDER and CBER. ALS treatments across the board face unique challenges to approval - which require unique solutions like Expanded Access and legislative support like Right to Try.
CBER vs. CDER
The FDA approves treatments through two primary centers: CDER, which reviews drugs, and CBER, which oversees biologics like vaccines, blood products, and gene or cell therapies. Approval begins with preclinical testing, followed by clinical trials (Phases 1–3) to evaluate safety and effectiveness. If results are positive, the sponsor submits an NDA to CDER or a BLA to CBER, and after FDA review and facility inspection, the product may be approved with ongoing post-market safety monitoring.
CBER
CBER is the Center for Biologics Evaluation and Research. Per the FDA website, "CBER protects and advances the public health by ensuring that biological products are safe and effective and available to those who need them. CBER also provides the public with information to promote the safe and appropriate use of biological products." Examples of ALS treatments that have been or may be reviewed by CBER include NurOwn and RAPA-501.
CDER
CDER is the Center for Drug Evaluation and Research. Per the FDA website, "CDER regulates over-the-counter and prescription drugs, including biological therapeutics and generic drugs. This work covers more than just medicines. For example, fluoride toothpaste, antiperspirants, dandruff shampoos and sunscreens are all considered drugs." Examples of ALS treatments that have been or may be reviewed by CDER include Radicavca, Qalsody, NP001, Pridopidine, and CNMAu8.
Approval Challenges
ALS treatments face major hurdles in the FDA approval process because the disease progresses quickly, patient numbers are small, and symptoms vary widely. Clinical trials often struggle to show clear benefits in a short time, and promising therapies can be delayed by the need for long-term safety data and complex regulatory requirements. These challenges slow the arrival of new treatments for a rapidly fatal disease.
​
Below is a diagram that conveys the trail process lined up against the average ALS patient lifespan following diagnosis.
So what can be done about these challenges?
To address the challenges of developing ALS treatments, the FDA offers alternative pathways and solutions to speed access and support approval. Programs such as Fast Track, Breakthrough Therapy, and Priority Review can shorten review timelines for promising therapies that address unmet medical needs. The Accelerated Approval pathway allows treatments to reach patients sooner based on surrogate endpoints, with confirmatory trials required after approval. Additionally, Expanded Access (Compassionate Use) programs provide a way for patients with serious or life-threatening conditions to access investigational treatments outside of clinical trials. Collaborative efforts, such as adaptive trial designs, patient registries, and biomarker research, also help generate meaningful data faster, improving the chances that effective ALS therapies can reach patients more quickly. Patients might also see benefit from legislative action such as Right to Try, which opened pathways to patient access to treatments from companies that are willing and able to provide them.
What does this mean for the NurOwn Citizens' Petition?
Petitioners are asking the FDA to consider the complexity of the approval process and trial design when it comes to ALS, as well as the unique solutions required to meet the unmet needs of ALS patients, when evaluating NurOwn. Most critically, petitioners are asking the FDA to consider the EAP and Right to Try data. These programs were created to support patient access beyond a standard trial, and they offer meaningful data regarding the broader patient population and long-term treatment effects.
*
This is a community-led site. We do our best to keep information current and accurate, but may not be able to update immediately when a process with the FDA or US Federal Government changes. If you have questions on processes or would like immediate clarification, please contact the FDA or US Federal Government directly.