Gene Therapy Program | FDA-Approved Gene Therapies (2024)

FAQs

What are 3 FDA-approved gene therapies? ›

Casgevy™ and Lyfgenia™ gene therapy for sickle cell disease. Elevidys gene therapy for duch*enne muscular dystrophy. Leukemia and lymphoma CAR T-cell therapy (KYMRIAH™)

Today, the U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of duch*enne muscular dystrophy (DMD) for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in the DMD gene.

Tisagenlecleucel was the first gene therapy to receive approval from the FDA for any indication.

On December 8, 2023, the U.S. Food and Drug Administration (FDA) approved Casgevy, a groundbreaking CRISPR-based gene editing therapy from Vertex Pharmaceuticals and CRISPR Therapeutics, for sickle cell disease (SCD).

There are basically three types of gene therapy: ex vivo, in vivo, and in situ. In ex vivo gene therapy, the target cells are removed from the patient's body, engineered either by the addition of the therapeutic gene or by other genetic manipulations that allow correction of the phenotype of the disease.

The most common gene therapy vectors are viruses. That's because they can recognize certain cells and carry genetic material into the genes of those cells. Researchers change the viruses, replacing genes that cause disease with genes needed to stop disease.

In the future, genetic therapies may be used to prevent, treat, or cure certain inherited disorders, such as cystic fibrosis, alpha-1 antitrypsin deficiency, hemophilia, beta thalassemia, and sickle cell disease. They also may be used to treat cancers or infections, including HIV.

Because gene therapy techniques are relatively new, some risks may be unpredictable; however, medical researchers, institutions, and regulatory agencies are working to ensure that gene therapy research, clinical trials, and approved treatments are as safe as possible.

Cell and gene therapy costs pose complex payer calculus

A recent report in Nature using gene therapies approved as of December 2020 and those in late-stage clinical trials concluded the annual cost of cell and gene therapies averaged $20.4 billion per year.

Why are there so few approved gene therapies? ›

Many genetic disorders that can potentially be treated with gene therapy are extremely rare, some affecting just one person out of a million. Gene therapy could be life-saving for these patients, but the high cost of developing a treatment makes it an unappealing prospect for pharmaceutical companies.

This one-time therapy involves the insertion of a functional copy of a missing or faulty gene into a person's cells by way of a viral or non-viral genetically-engineered vector (delivery vehicle). The goal of this therapy is for the new gene to produce a protein the body couldn't (previously) produce adequately.

Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research (CBER), emphasized that there are now 17 FDA-approved gene therapies on the market, to treat everything from relapsed pediatric and young adult acute lymphoid leukemia to spinal muscular atrophy (SMA).

There are currently five FDA-approved gene therapy treatments available in the United States. Three are approved for adults while two are approved for pediatrics only.

Initially the treatment will be for those aged twelve-months through adulthood. There is no upper age limit for treatment.

Laboratory assessments — from preclinical research through post-marketing surveillance — determine whether a cell-based therapy for cancer succeeds or fails. Between 2010 and 2023, the US Food and Drug Administration (FDA) approved 34 cell and gene therapies¹.

CAR T-cell therapy is FDA-approved to treat aggressive B-cell lymphomas in adults (Yescarta and Kymriah), B-cell leukemia in children and young adults (Kymriah), and most recently, relapsed or refractory mantle cell lymphoma (MCL) in adults (Tecartus). What is CAR T cell therapy?

It is important to note that the FDA approved three therapeutic vaccines: Bacillus Calmette-Guerin (BCG) in the 1970s [2,20], Sipuleucel-T in 2010 [2,13], and talimogene laherparepvec in 2015 [12]. BCG is considered the “gold standard treatment” for the treatment of high-risk non-muscle-invasive bladder cancer.