Illustration of a black metal pipeline on a yellow background

What Is the Drug Development Pipeline?

5 Second Summary

Right now, thousands of potentially beneficial therapies for neuromuscular diseases are being researched and tested. Here’s what that means for you and why MDA calls it “the pipeline of promise.”

You’ve probably heard of the drug development pipeline when people talk about research for neuromuscular disease therapies. But what is it, and why is it called a pipeline?

The world of pharmaceutical research and development is complex. Any company that wants to sell a drug in the United States must go through a series of steps to develop a drug that is safe and effective.

The pipeline represents this process and all the drug candidates that are being researched or tested in one of these stages:

Drug development stages

Stage 1 Discovery and development: Early laboratory testing to turn an idea into a potential drug

Stage 2 Preclinical research: Laboratory and animal testing to establish basic safety of the drug

Stage 3 Clinical trials: Studying how the drug works in people

Stage 4 FDA Review: The US Food and Drug Administration (FDA) examines data from each stage and decides whether to approve the drug.

An active pipeline

It’s called a pipeline, not a straw, because the biopharmaceutical industry is busy. There are thousands of potentially beneficial therapies in development at any given time.

This is an especially promising time for neuromuscular diseases, with about 165 potentially disease-altering drugs currently in the clinical trial stage.

Clinical trials go through three phases before a drug may be approved. Each phase serves a different purpose.

Phases of a clinical trial

Phase 1: Safety and dosing

Approximately 70% of drugs move to the next phase.

Phase 2: Efficacy and side effects

Approximately 33% of drugs move to the next phase.

Phase 3: Efficacy and monitoring adverse reactions

Approximately 25% of drugs move to FDA review.

Look at this PIPE Chart to see details on some of the promising drugs in clinical trials. If you don’t see your disease in the chart, don’t worry — it shows just a fraction of the clinical trials in progress. Scroll down to “How to Find Clinical Trials” to learn how to find a clinical trial that might be right for you or your child.

Important drug approvals

In the past decade, the drug development pipeline has led to some exciting treatment advances for MDA’s community. As of January 2024, the FDA has approved more than 20 new drugs to treat neuromuscular diseases. This is largely due to the remarkable progress made in the field of genetic medicine.

MDA is proud to play a significant role in this progress. To date, MDA-sponsored research has directly contributed to the development of eight FDA-approved therapies, including Relyvrio© and Rilutek© for amyotrophic lateral sclerosis (ALS); Myozyme© for Pompe disease; Keveyis© for periodic paralysis; Spinraza© for spinal muscular atrophy (SMA); and Exondys 51©, Emflaza©, Vyondys 53©, and Agamree© for Duchenne muscular dystrophy (DMD).

MDA also funded the first gene therapy trial for any form of muscular dystrophy in 1999 and the first vector-based gene therapy trial for DMD in 2006. Many of the gene therapies in clinical trials for neuromuscular diseases in the United States are based on strategies developed with MDA funding.

Progress across diseases

Families coping with ultra-rare diseases can feel frustrated as they see therapies developed for more prevalent neuromuscular diseases, like ALS and DMD. But there is a lot of positive news on that front, too.

In addition to advances in genetic medicine, the last decade has seen advances in clinical trial design and drug manufacturing, making it more feasible to develop drugs for specific rare diseases or classes of rare diseases with similar characteristics. Some biotech companies have formed to pursue rare disease research, and MDA created the Kickstart program to develop gene therapies for ultra-rare disorders.

In addition, scientific advances often translate across diseases. “The methods and platforms used to develop drugs for diseases like DMD and SMA have high promise to be applied to the super rare diseases,” Alan H. Beggs, PhD, director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital, told Quest Media.

Right now, an unprecedented flow of potential neuromuscular disease therapies is moving from discovery and development to preclinical research to clinical trials, giving the MDA community hope that we’ll see more drugs approved to treat neuromuscular diseases in the coming years. That is what MDA calls the “pipeline of promise.”

Amy Bernstein is a writer and editor for Quest Media.

Types of Therapies in the Pipeline

Researchers are exploring many ways to treat neuromuscular diseases by addressing specific symptoms or altering body mechanisms. Some of the most promising candidates fall into one of the categories below or combine them to target multiple pathways of the disease.

Antibody treatments: A natural component of our immune system, antibodies are proteins that can identify and selectively bind and neutralize a targeted protein. For therapies, antibodies have been designed in the lab to target potential cellular contributors to diseases.

Cell therapy: These therapies integrate new, healthy cells into the body to replace diseased or damaged cells, use interactive factors to modulate the function of existing cells, or use immune cells to remove dysfunctional cells.

Gene therapy: These therapies use techniques to modify a person’s genes to change the course of a disease. This might involve introducing a healthy copy of a gene to replace a non-working or missing gene, inactivating a malfunctioning gene, or introducing a new or modified gene into the body.

RNA-targeted therapies: RNA, similar to DNA, is present in all living cells and serves many functions, including acting as a messenger carrying instructions from DNA for controlling the synthesis of proteins. By targeting RNA, these therapies seek to reduce, restore, or modify protein expression. Exon skipping is an example of a therapy that modifies messenger RNA (mRNA) to deliver new instructions to cells. Other types of RNA being used in therapies include small interfering RNAs (siRNAs) and microRNAs (miRNAs). Antisense oligonucleotides (ASOs) are a synthetic form of RNA or DNA.

Small molecule drugs: Small molecules are organic compounds with low molecular weight, which allows them to pass through cell membranes easily. Small molecule drugs are designed to engage biological targets inside cells and modify disease pathways by activating or inhibiting certain functions.

How to Find Clinical Trials

Many people in the MDA community are interested in participating in clinical trials. Here is how you can find and learn about clinical trials for neuromuscular diseases.

Find a trial

MDA’s resources for finding clinical trials include the Clinical Trial Finder tool, Clinical Trial Updates list, Quest Magazine, and Quest Blog.

Your MDA Care Center team is an excellent resource for finding clinical trials that might be right for you or your child. They keep up with the latest research, and many Care Centers serve as clinical trial sites.

Dig deeper

Your next stop is the National Library of Medicine’s, an online database of clinical research studies taking place in the United States and more than 200 countries.

This website is a wealth of information if you know how to find it. Review the How to Search for Clinical Studies page to get started.

Once you know how to search, you can zero in on the studies that are relevant to you. Listings include important information, such as:

  • Whether a study is actively enrolling
  • Eligibility requirements
  • What the study is measuring
  • How the study is designed
  • How long it is expected to last
  • Who to contact about the study

Before you join a study, know what questions to ask. Many trials have a study coordinator who can answer questions and help address barriers to participation for eligible individuals.

Consider compassionate use

In cases where an individual with a life-threatening condition may benefit from an investigational drug but cannot participate in a clinical trial, a physician may ask the drug developer and health authorities for permission to administer the drug to that individual outside of the clinical trial. This is called “expanded access” or “compassionate use.”

Learn more about these options through the Reagen-Udall Foundation Expanded Access Navigator.

Next Steps and Useful Resources

Disclaimer: No content on this site should ever be used as a substitute for direct medical advice from your doctor or other qualified clinician.