Exploring FDA-approved cell, gene therapeutic products

Cell Therapies 

Abecma (idecabtagene vicleucel) 

Abecma is a CAR T-cell therapy used to treat multiple myeloma. It modifies a patient's T cells to target BCMA protein on cancer cells, offering a personalized approach to treating this type of cancer. 

• Manufacturer: Bristol Myers Squibb and bluebird bio 

• FDA Approval Date: March 26, 2021 

• Cost: Approximately $419,500 per treatment 

Breyanzi (lisocabtagene maraleucel) 

Breyanzi is a CAR T-cell therapy for treating certain types of aggressive B-cell lymphomas like DLBCL. It modifies T cells to target CD19 proteins on the surface of lymphoma cells, offering a potent treatment option. 

• Manufacturer: Bristol Myers Squibb 

• FDA Approval Date: February 5, 2021 

• Cost: Approximately $410,300 per treatment 

Carvykti (ciltacabtagene autoleucel) 

Carvykti is a CAR T-cell therapy for multiple myeloma that modifies T cells to target BCMA on cancer cells, providing an advanced treatment option for relapsed or refractory cases. 

• Manufacturer: Johnson & Johnson and Legend Biotech 

• FDA Approval Date: February 28, 2022 

• Cost: Approximately $465,000 per treatment 

Kymriah (tisagenlecleucel) 

Kymriah is the first FDA-approved CAR T-cell therapy and is used to treat certain types of large B-cell lymphoma and B-cell precursor acute lymphoblastic leukemia in children and young adults. This therapy involves reprogramming a patient's own T cells to target and kill cancer cells. 

• Manufacturer: Novartis 

• FDA Approval Date: August 30, 2017 

• Cost: Approximately $475,000 per treatment 

Lantidra (donislecel) 

Lantidra is an innovative T-cell therapy for treating type 1 diabetes. It involves transplanting islet cells to help restore insulin production in patients with severe diabetes. 

• Manufacturer: CellTrans Inc. 

• FDA Approval Date: June 28, 2023 

• Cost: Approximately $450,000 per treatment 

MACI (Matrix-Induced Autologous Chondrocyte Implantation) 

MACI is a cell therapy used to repair cartilage damage in the knee. It involves harvesting a patient's cartilage cells, expanding them in a lab, and implanting them into the damaged area with a collagen membrane to promote the growth of healthy cartilage and improve joint function. 

• Manufacturer: Vericel Corp 

• FDA Approval Date: December 13, 2016 

• Cost: Approximately $40,000 per treatment 

Provenge (sipuleucel-T) 

Provenge is a cell-based therapeutic vaccine used in the treatment of advanced prostate cancer. It is designed specifically for patients with asymptomatic or minimally symptomatic metastatic castrate-resistant prostate cancer. Provenge works by stimulating the patient's own immune system to target and attack prostate cancer cells. 

• Manufacturer: Dendreon Corp 

• FDA Approval Date: April 29, 2010 

• Cost: Approximately $93,000 per treatment  

StrataGraft  

StrataGraft utilizes cultured living donor skin cells, specifically, keratinocytes and fibroblasts, which are applied to wounds to replace damaged or lost skin in individuals suffering from severe burns. 

• Manufacturer: Stratatech Corporation 

• FDA Approval Date: June 15, 2021 

• Cost: Approximately $4,222 per topical film 

Tecartus (brexucabtagene autoleucel) 

Tecartus is a CAR T cell therapy for treating mantle cell lymphoma (MCL) and adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). The therapy modifies the patient's T cells to target CD19-expressing cancer cells.  

• Manufacturer: Kite Pharma, a Gilead Company 

• FDA Approval Date: July 24, 2020 (MCL); October 1, 2021 (ALL)  

• Cost: Approximately $373,000 per treatment 

Yescarta (axicabtagene ciloleucel) 

Yescarta is another CAR T cell therapy approved for treating certain types of large B-cell lymphoma and follicular lymphoma. It modifies a patient's T cells to target CD19 on the surface of cancer cells. 

• Manufacturer: Kite Pharma, a Gilead Company 

• FDA Approval Date: October 18, 2017 

• Cost: Approximately $373,000 per treatment 

Cord Blood Transplants 

Clevecord, Ducord, Hemacord (HPC, Cord Blood), and similar products are key cell and gene therapy components. They are specifically used for cord blood transplants, which are critical for treating various hematological and genetic disorders. These products are important because they provide a source of hematopoietic stem cells that can regenerate healthy blood and immune systems in patients.  

Clevecord (HPC, Cord Blood) 

• Manufacturer: Cleveland Cord Blood Center 

• FDA Approval Date: May 11, 2016 

Ducord (HPC, Cord Blood) 

• Manufacturer: Duke University School of Medicine 

• FDA Approval Date: May 31, 2012 

Hemacord (HPC, Cord Blood) 

• Manufacturer: New York Blood Center 

• FDA Approval Date: November 10, 2011 

HPC, Cord Blood - MD Anderson Cord Blood Bank 

• Manufacturer: MD Anderson 

• FDA Approval Date: March 11, 2013 

HPC, Cord Blood – Bloodworks 

• Manufacturer: Bloodworks 

• FDA Approval Date: June 9, 2011 

HPC, Cord Blood – LifeSouth 

• Manufacturer: LifeSouth Community Blood Centers 

• FDA Approval Date: April 5, 2013 

The cost of cord blood transplants can vary, but it generally ranges from $25,000 to $40,000, not including additional expenses such as hospital stays and post-transplant care.  

Gene Therapies  

Adstiladrin (nadofaragene firadenovec)  

Adstiladrin is a gene therapy used for the treatment of high-grade, non-muscle invasive bladder cancer that is unresponsive to Bacillus Calmette–Guérin therapy. 

• Manufacturer: Ferring Pharmaceuticals 

• FDA Approval Date: December 18, 2019 

• Cost: Approximately $60,000 per unit dose 

Beqvez (elivaldogene tavalentivec) 

Beqvez is a targeted gene therapy for Hodgkin lymphoma and systemic anaplastic large-cell lymphoma. It combines an antibody with a chemotherapy drug to bind to cancer cells and deliver treatment directly. 

• Manufacturer: Orchard Therapeutics 

• FDA Approval Date: May 20, 2023 

• Cost: Approximately $2.9 million per treatment 

Casgevy (valoctocogene roxaparvovec) 

Casgevy is a one-time gene therapy treatment for individuals aged 12 and older with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). This therapy utilizes the gene-editing technology CRISPR/Cas9 to modify the patient's own blood-forming stem cells, enabling the production of new, healthy red blood cells. 

• Manufacturer: BioMarin Pharmaceutical 

• FDA Approval Date: December 8, 2023 (SCD); January 16, 2024 (TDT) 

• Cost: Approximately $2.5 million per treatment 

Elevidys (delandistrogene moxeparvovec-rokl) 

Elevidys is a prescribed gene therapy intended for the treatment of ambulatory children aged 4–5 years old diagnosed with Duchenne muscular dystrophy and confirmed dystrophin gene mutations. 

• Manufacturer: Sarepta Therapeutics 

• FDA Approval Date: June 22, 2023 

• Cost: Approximately $2.9 million per treatment 

Hemgenix (etranacogene dezaparvovec) 

Hemgenix is a gene therapy for hemophilia B, providing a functional copy of the gene responsible for producing factor IX. 

• Manufacturer: CSL Behring 

• FDA Approval Date: November 22, 2022 

• Cost: Approximately $3.5 million 

Luxturna (voretigene neparvovec) 

Luxturna is a gene therapy for inherited retinal dystrophy caused by RPE65 gene mutations. It delivers a correct copy of the gene to retinal cells, helping to restore vision and reduce vision loss. 

• Manufacturer: Spark Therapeutics 

• FDA Approval Date: December 19, 2017 

• Cost: Approximately $850,000 per treatment (both eyes) 

Roctavian (valoctocogene roxaparvovec) 

Roctavian is a gene therapy developed to treat hemophilia A, a genetic bleeding disorder caused by a deficiency or dysfunction of clotting factor VIII. 

• Manufacturer: BioMarin Pharmaceutical 

• FDA Approval Date: June 24, 2023 

• Cost: Approximately $3 million per treatment 

Skysona (elivaldogene autotemcel) 

Skysona is a gene therapy used to treat cerebral adrenoleukodystrophy (CALD), a rare and progressive genetic disorder that affects the nervous system. It is designed for pediatric patients with CALD who have specific genetic mutations and are at risk of developing severe neurological symptoms. 

• Manufacturer: bluebird bio 

• FDA Approval Date: September 16, 2022 

• Cost: Approximately $3 million per treatment 

Vyjuvek (beremagene geperpavec) 

Vyjuvek is a topical gene therapy for treating dystrophic epidermolysis bullosa, a genetic skin disorder. 

• Manufacturer: Krystal Biotech 

• FDA Approval Date: May 19, 2023 

• Cost: Approximately $2.8 million per treatment 

Zolgensma (onasemnogene abeparvovec) 

Zolgensma is a gene therapy for spinal muscular atrophy caused by mutations in the SMN1 gene. It delivers a functional copy of the SMN1 gene to motor neuron cells, improving muscle function and survival.  

• Manufacturer: Novartis Gene Therapies 

• FDA Approval Date: May 24, 2019 

• Cost: Approximately $2.1 million per treatment 

Zynteglo (betibeglogene autotemcel) 

Zynteglo is a gene therapy for beta-thalassemia, a genetic blood disorder that reduces hemoglobin production. The therapy involves inserting a functional beta-globin gene into the patient's hematopoietic stem cells. 

• Manufacturer: bluebird bio 

• FDA Approval Date: August 17, 2022 

• Cost: Approximately $2.8 million per treatment 

Additional Therapies 

Imlygic (talimogene laherparepvec)  

Imlygic is an oncolytic viral therapy for treating unresectable melanoma lesions in the skin and lymph nodes. Derived from the herpes simplex virus, it replicates selectively within tumors to induce their destruction and stimulate an immune response against cancer. 

• Manufacturer: Amgen 

• FDA Approval Date: October 27, 2015 

• Cost: Approximately $65,000 per treatment  

Rethymic (allogeneic processed thymus tissue-agdc)

Rethymic is a tissue-based therapy used for treating congenital athymia, a condition where children are born without a thymus, leading to severe immunodeficiency. This therapy is uniquely crafted to support the development of children's immune systems, enhancing their ability to combat infections. 

• Manufacturer: Enzyvant Therapeutics 

• FDA Approval Date: October 8, 2021 

• Cost: $2.7 million per treatment