Leigh Ann Anderson, PharmD. Nov 30, 2025.
1. Lenmeldy
- Estimated Cost: $4.25 million per one time treatment
- Manufacturer: Orchard Therapeutics
- Use: Metachromatic leukodystrophy (MLD)
- Initial FDA Approval Date: March 18, 2024
Lenmeldy (atidarsagene autotemcel) is an innovative autologous hematopoietic stem cell (HSC) gene therapy specifically designed for the treatment of children diagnosed with metachromatic leukodystrophy (MLD), a rare genetic disorder characterized by the progressive degeneration of myelin in the nervous system. By utilizing the patient’s own stem cells, Lenmeldy aims to rectify the underlying enzyme deficiency caused by genetic mutations, offering hope where conventional treatments have failed. As the first approved therapy for MLD, Lenmeldy holds the remarkable potential to restore enzymatic function, thereby halting or significantly slowing the progression of this devastating disease, which if left untreated, leads to severe neurological decline and ultimately death. Administered exclusively at Qualified Treatment Centers, Lenmeldy requires a multidisciplinary approach to ensure comprehensive care and support for both the child and their family throughout the treatment process, emphasizing the importance of specialized medical supervision in managing this complex therapeutic approach.
The wholesale acquisition cost (WAC) of Lenmeldy was reported to be US$4.25 million according to Orchard Therapeutics, making it the most expensive drug in the U.S. at the time. Officials at Orchard cite the value of therapy to patients and their families, possible caregiver wage loss, and the potential to lower healthcare utilization.
MLD is a rare and potentially fatal genetic disease of the metabolic system caused by a mutation in the arylsulfatase-A (ARSA) gene. This mutation causes accumulation of sulfatides (a type of fat) that destroy the protective layer of the nerves called the myelin sheath. Patients eventually lose the ability to move, talk, swallow, eat and see. Mortality is estimated at 50% at 5 years for late infantile disease and 44% at 10 years for juvenile patients.
Lenmeldy works by inserting one or more functional copies of the human ARSA gene into the genome of a patient’s own hematopoietic stem cells (HSCs), outside the body, using a lentiviral vector. Cells are then infused back into the patient where they can migrate across the blood-brain barrier into the central nervous system and express the functional enzyme.
Open-label studies looked at data from 37 pediatric patients with early-onset MLD with 12 years of follow-up (median 6.8 years). All children with pre-symptomatic late infantile MLD who were treated with Lenmeldy were alive at 6 years of age, compared to 58% in the natural history group. At 5 years of age, 71% of treated children were able to walk without assistance.
Lenmeldy is approved for use in the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile metachromatic leukodystrophy (MLD).
Common adverse reactions (≥10%) include fever and low white blood cells, mouth sores, respiratory tract infections, rash, medical line (device related) infections, other viral infections, fever, gastrointestinal (stomach area) infections and enlarged liver. Common laboratory abnormalities were elevated D-dimer, neutropenia, and elevated liver enzymes.
2. Hemgenix
- Estimated Cost: $3.5 million per one-time dose
- Manufacturer: CSL Behring
- Use: Hemophilia B
- Initial FDA Approval Date: November 22, 2022
Hemgenix (etranacogene dezaparvovec-drlb) is approved to treat hemophilia B, a rare, lifelong bleeding disorder that significantly impacts the quality of life for those affected. This groundbreaking treatment is administered as a single, one-time intravenous (IV) infusion, representing a notable advancement in therapeutic options for patients. With a list price of $3.5 million, Hemgenix holds the title of being one of the most expensive drugs available in the U.S. as of now. The medication works by enabling individuals living with hemophilia B to produce their own factor IX, a crucial blood clotting factor, which can dramatically lower the risk of bleeding episodes that patients frequently face.
The condition of Hemophilia B is characterized by a genetic defect affecting a single gene, which leads to insufficient production of factor IX. This protein, primarily produced by the liver, plays a vital role in the body’s ability to form blood clots. As a result, individuals with hemophilia B are particularly susceptible to excessive bleeding, especially in vulnerable areas such as joints, muscles, and internal organs. This increased risk of bleeding can lead to severe complications, including pain, swelling, and long-term joint damage, which may significantly impair mobility and overall health.
Historically, patients with hemophilia B have been required to adhere to strict and often costly lifelong infusion schedules to receive factor IX replacement therapy. These frequent infusions not only create a financial burden but also pose an inconvenience to patients and their families, as they must manage recurring visits to healthcare facilities. Hemgenix represents a transformative change in this landscape, offering hope for a more manageable and potentially life-changing solution for those diagnosed with this rare disorder. The introduction of such innovative therapies often signifies a new era in treatment paradigms, shifting the focus towards more holistic and sustainable healthcare practices for individuals burdened by chronic conditions like hemophilia B.
Hemgenix is an adeno-associated virus vector-based gene therapy. It works by supplying a noninfectious viral vector (AAV5) to carry genetic DNA instructions to the liver, where factor IX proteins are then generated. These genetic instructions remain in the target cells, but generally do not become a part of a person’s own DNA.
In the HOPE-B clinical trial, Hemgenix reduced the rate of annual bleeds and 94% of patients discontinued factor IX infusions. Seven to 18 months after Hemgenix, the mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by 54%.
The most commonly reported side effects (incidence ≥5%) with this gene therapy include liver enzyme elevations, headache, elevated enzymes in the blood (creatine kinase), flu-like symptoms, infusion-related reactions, fatigue, nausea, elevated liver enzymes and feeling unwell.
3. Elevidys
- Estimated Cost: $3.2 million per one-time dose
- Manufacturer: Sarepta Therapeutics
- Use: Duchenne Muscular Dystrophy (DMD)
- Initial FDA Approval Date: June 22, 2023
Elevidys (delandistrogene moxeparvovec-rokl) is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD) who are ambulatory and have a confirmed mutation in the DMD gene.
- Limitations of Use: Elevidys is not recommended in patients with:
- Preexisting liver impairment (defined as gamma-glutamyl transferase [GGT] > 2 x upper limit of normal or total bilirubin > the upper limit of normal not due to Gilbert’s syndrome) or active hepatic viral infection due to the high risk of acute serious liver injury and acute liver failure.
- Recent vaccination (within 4 weeks of treatment) due to immunogenicity and potential safety concerns.
- Active or recent (within 4 weeks) infections due to safety concerns.
Elevidys is given as a one-time, single-dose intravenous (IV) infusion and was marketed at approval with a $3.2 million price tag.
Patients selected for treatment have anti-AAVrh74 total binding antibody titers <1:400. Elevidys should not be used in patients with any deletion in exon 8 and/or exon 9 in the DMD gene, including a deletion of any portion or the entirety of these exons.
Duchenne Muscular Dystrophy (DMD) is the most common childhood form of muscular dystrophy and typically occurs in boys. It is an inherited degenerative disease that results in muscle weakness and loss of muscle tissue, which worsens over time. The average life expectancy is about 27 years.
DMD is caused by a dystrophin gene mutation that yields low levels of the dystrophin protein, needed to strengthen muscle fibers. Elevidys was designed to treat the underlying cause of DMD by delivering a gene that codes for a functional shortened dystrophin (called Elevidys micro-dystrophin) into the muscle tissue.
Elevidys labeling contains a Boxed Warning, the agency’s most prominent safety warning, detailing that acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred with Elevidys. Patients with preexisting liver impairment may be at higher risk. See the full prescribing information for the complete boxed warning information.
Warnings and precautions include infusion-related reactions, acute serious liver injury, immune-mediated myositis (muscle inflammation), myocarditis (heart muscle inflammation) and pre-existing immunity against AAVrh74.
Common side effects include vomiting and nausea, liver injury, pyrexia (fever), and thrombocytopenia (low platelet counts) and troponin-I increased.
Related: What are the new drugs for Duchenne muscular dystrophy?
4. Skysona
- Estimated Cost: $3 million per one-time dose
- Manufacturer: bluebird bio, Inc.
- Use: Cerebral Adrenoleukodystrophy (CALD)
- Initial FDA Approval Date: September 16, 2022
Skysona (elivaldogene autotemcel or eli-cel) gene therapy was approved as the first treatment to slow the progression of neurologic brain dysfunction in boys 4 to 17 years of age with early, active cerebral adrenoleukodystrophy (CALD).
- This indication is approved under accelerated approval based on 24-month Major Functional Disability (MFD)-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s)
Skysona is made from the patient’s own stem cells and modified to contain a copy of the gene to make a functional enzyme called ALDP (adrenoleukodystrophy protein). Skysona is given as a single, one-time intravenous (IV) infusion treatment at a cost of $3 million.
Cerebral adrenoleukodystrophy (CALD) is a rare, inherited and fatal neurodegenerative disease that occurs primarily in young boys (median age of onset 7 years). CALD occurs due to a mutation in the ABCD1 gene which prevents the production of the ALDP enzyme. This prevents the breakdown of very long-chain fatty acids, which results in destruction of the sheath (myelin) that surrounds the nerve. CALD results in loss of ability to communicate, cortical blindness, need for tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement. Nearly half of patients who do not receive treatment die within 5 years of symptom onset.
In studies, Skysona-treated patients had an estimated 72% likelihood of Major Functional Disability (MFD)- free survival at 24 months from time of first neurologic function score (NFS) ≥ 1, compared to untreated patients who had an estimated 43% likelihood of MFD-free survival.
Skysona carries a Boxed Warning for hematologic malignancies, including life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia. Warnings and precautions include serious infections, prolonged cytopenias, delayed platelet engraftment, and the risk of neutrophil engraftment failure
The most common adverse reactions (≥ 20%) include: mucositis (mouth sores, inflammation), nausea, vomiting, febrile neutropenia (fever with low white blood cell count), alopecia (hair loss), decreased appetite, abdominal pain, constipation, pyrexia (fever), diarrhea, headache, rash, as well as laboratory abnormalities (≥ 40%): leukopenia, lymphopenia, thrombocytopenia, neutropenia, anemia, and hypokalemia.
5. Zynteglo
- Estimated Cost: $2.8 million per one-time dose
- Manufacturer: bluebird bio, Inc.
- Use: Beta-thalassemia
- Initial FDA Approval Date: September 16, 2022
Zynteglo (betibeglogene autotemcel or beti-cel) is an innovative lentiviral vector (LVV) gene therapy that has received approval for use in treating both adult and pediatric patients suffering from beta-thalassemia, particularly those who require regular red blood cell (RBC) transfusions. This therapy represents a notable advancement in the management of this condition, specifically tailored for each patient using their own stem cells. Zynteglo is administered through a one-time, single-dose intravenous (IV) infusion, with an associated cost of approximately $2.8 million.
Beta-thalassemia is classified as a rare genetic blood disease that profoundly affects the production of hemoglobin in the body. Individuals afflicted by this condition can experience severe anemia, marked by a significant shortage of healthy red blood cells, which often necessitates a lifelong routine of RBC transfusions. Patients typically endure this transfusion process every 2 to 5 weeks, which can be both physically taxing and logistically challenging, often impacting their overall quality of life.
The mechanism behind Zynteglo involves the introduction of functional copies of a modified version of the beta-globin gene, specifically the βA-T87Q-globin gene, into the patient’s own hematopoietic (blood) stem cells. This process occurs outside of the body (ex-vivo), whereby scientists extract the patient’s stem cells, modify them with the engineered gene, and then reinfuse these corrected cells back into the patient. The successful integration of this modified gene can lead to the production of normal or near-normal levels of total hemoglobin, which may significantly eliminate, or at least substantially reduce, the need for ongoing RBC transfusions.
Given its personalized approach, Zynteglo addresses not only the symptoms associated with beta-thalassemia but also focuses on altering the underlying genetic defect at a cellular level. This targeted therapy offers hope for patients who have long faced the burdens of frequent transfusions and the complications that accompany them, marking a potential turning point in the treatment landscape for this challenging condition.
In studies, 89% (32/36) of evaluable patients achieved transfusion independence, defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average total hemoglobin of at least 9 g/dL.
Warnings and precautions associated with Zynteglo include delayed platelet engraftment, risk of neutrophil engraftment failure, risk of insertional oncogenesis, and hypersensitivity reactions.
In studies, the most common adverse reactions (≥20%) were: mucositis (inflamed mouth / digestive tract), febrile neutropenia (low white blood cell count with fever), vomiting, pyrexia (fever), alopecia (hair loss), epistaxis (nose bleed), abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and pruritus (itch).
The most common Grade 3 or 4 laboratory abnormalities (> 50%) include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia.
6. Zolgensma
- Estimated Cost: $2.125 million per one-time dose
- Manufacturer: Novartis
- Use: Spinal Muscular Atrophy
- Initial FDA Approval Date: May 24, 2019
Zolgensma (generic name: onasemnogene abeparvovec-xioi) is a gene therapy approved to treat Spinal Muscular Atrophy (SMA) in pediatric patients less than 2 years of age with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
- Limitations of Use
- The safety and effectiveness of repeat administration of Zolgensma have not been evaluated.
- The use of Zolgensma in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated.
Zolgensma is given as a one-time, single-dose, intravenous (IV) infusion therapy with a total cost at approval of $2.125 million in the U.S. It works by replacing the defective or missing survival motor neuron 1 (SMN1) gene to treat the root cause of SMA and halt disease progression. It is considered potentially curative.
Spinal Muscular Atrophy (SMA) is a rare genetic motor neuron disease that results in problems with breathing, holding up your head, and swallowing. The majority of children with SMA do not survive past early childhood due to an inability to breathe (respiratory failure).
En estudios, se demostró la efectividad en 36 pacientes que tenían entre 2 semanas y 8 meses de edad al ingresar al estudio. De los 21 pacientes tratados con Zolgensma, quedaron 19 pacientes en el estudio que tenían entre 9.4 meses y 18.5 meses, y 13 pacientes tienen al menos 14 meses de edad. También se alcanzaron los hitos del desarrollo motor.
Zolgensma labeling carries a Boxed Warning that acute liver failure and fatal outcomes have been reported and may can occur. Acute serious liver injury and elevated liver enzymes (aminotransferases) may also occur.
Advertencias y precauciones incluyen Respuesta Inmune Sistémica, Trombocitopenia, Microangiopatía Trombótica (TMA), Troponina I Elevada, Integración del Vector AAV y Riesgo de Tumorigénesis, y Reacciones Relacionadas con la Infusión.
In studies, common side effects included elevated liver enzymes and vomiting.
7. Myalept
- Estimated Cost: $1.3 million annually
- Manufacturer: Chiesi Farmaceutici S.p.A
- Use: Lipodystrophy / Leptin deficiency
- Initial FDA Approval Date: February 24, 2014
Myalept (metreleptina para inyección) fue aprobado por la FDA como una terapia de reemplazo para tratar las complicaciones de la deficiencia de leptina, además de la dieta, en pacientes con lipodistrofia generalizada congénita o adquirida. La lipodistrofia puede ser hereditaria a través de los genes o adquirida sin una causa conocida.
Lipodystrophy is a rare condition that affects how the body stores and use fat. Fat may build in places where it should not be, such as the blood and organs. Patients with generalized lipodystrophy often develop severe insulin resistance, diabetes, or high levels of triglycerides (hypertriglyceridemia) that can lead to pancreas inflammation.
Myalept is given as a subcutaneous (under the skin) injection once a day and patients can be taught to self-administer the medicine at home. One 11.3 mg vial runs about $6,188 and doses are weight-based. As one example, a patient might use about 18 vials per month, therefore annual drug cost might exceed $1.3 million.
In clinical trials, Myalept led to reductions in HbA1c (a measure of blood sugar control), fasting glucose, and triglycerides.
El etiquetado de Myalleft lleva una ** advertencia en recuadro ** por el riesgo de anticuerpos anti-metreleptina con actividad neutralizante y el riesgo de linfoma de células T; Myalept solo está disponible a través del Programa de Estrategia de Evaluación y Mitigación de Riesgos (REMS) de Myalept debido a estos riesgos.
8. Danyelza
- Costo Estimado: $1.2 millones anuales
- Manufacturer: Y-mAbs Therapeutics
- Use: Neuroblastoma
- Initial FDA Approval Date: November 25, 2020
Danyelza (naxitamab-gqgk) está aprobado para tratar a adultos y niños de 1 año de edad y mayores con neuroblastoma de alto riesgo que ha recidivado o es refractario en el hueso o la médula ósea, en combinación con el factor estimulante de colonias de granulocitos-macrófagos (GM-CSF), que han demostrado una respuesta parcial, una respuesta menor o una enfermedad estable a la terapia previa. Este medicamento innovador se utiliza en pacientes que han progresado (recidivado) o que han tenido una respuesta incompleta a la terapia (refractarios), ofreciendo una nueva esperanza en el tratamiento de esta grave enfermedad. Danyelza actúa aprovechando el sistema inmunitario del paciente para atacar las células cancerosas, lo que representa un enfoque terapéutico que busca mejorar la calidad y la expectativa de vida de los pacientes afectados. Además, la administración de GM-CSF como complemento a Danyelza fomenta un ambiente propicio para que las células inmunitarias evolucionen y cumplan su función de erradicar las células malignas, optimizando así los resultados clínicos y brindando nuevas perspectivas en la lucha contra el neuroblastoma de alto riesgo.
- Danyelza was approved by the FDA on an «accelerated» basis. In clinical studies, some people responded to this medicine, but further studies are needed for a full approval for this use.
Each vial of Danyelza costs about $24,300. The length of treatment depends on response, and dosing is weight based, so it’s hard to estimate an exact cost. As an example, if patient used 48 vials of medicine the drug cost would run about $1.2 million annually. Danyelza is also used in combination with GM-CSF to help your body to produce certain blood cells that help fight the cancer and help prevent infections, which adds to the cost of therapy.
Neuroblastoma is a solid tumor that occurs in the nervous system, outside of the brain, most often in infants and young children. These tumor cells can spread (metastasize) to other sites in the body, such as the bone or bone marrow. Some tumors can be treated, but the majority are very aggressive.
In 2 studies, patients had an overall response rate (partial response + complete response rate) of 34% and 45%. In these studies, 23% and 34% of patients continued to respond to treatment without the tumor growing or spreading for at least 6 months after initial response. Treatment may be stopped due to disease progression or intolerable side effects. Your doctor will decide how long to treat you with this medicine.
Danyelza is classified as a GD2-binding monoclonal antibody immunotherapy. It works by attaching to tumor cells and sends a signal to the immune system to kill the cancer. It is administered as an intravenous (IV) infusion in cycles. Discontinue treatment for disease progression or unacceptable toxicity.
Danyelza contains a Boxed Warning for serious infusion-related reactions and neurotoxicity.
Common side effects include infusion-related reaction, pain, fast heart rate, vomiting, cough, nausea, diarrhea, decreased appetite, hypertension, fatigue, erythema multiforme, peripheral neuropathy, urticaria, pyrexia, headache, injection site reaction, edema, anxiety, localized edema, and irritability.
9. Zokinvy
- Estimated Cost: $1.2 million annually
- Manufacturer: Eiger BioPharmaceuticals, Inc.
- Use: Progeria and Progeroid Laminopathies
- Initial FDA Approval Date: November 20, 2020
Zokinvy (lonafarnib) es un medicamento oral utilizado para el tratamiento del Síndrome de Progeria de Hutchinson-Gilford (HGPS o Progeria) y las Laminopatías Progeroides Deficientes en Procesamiento (PL), ciertas condiciones genéticas raras que provocan un envejecimiento prematuro y rápido.
Zokinvy está indicado en pacientes adultos y pediátricos de 12 meses de edad o más con una superficie corporal (BSA) de 0.39 m2 y superior. Zokinvy ofrece un beneficio de supervivencia para los pacientes con enfermedades genéticas de envejecimiento prematuro.
Specifically, Zokinvy is used:
- Para reducir el riesgo de mortalidad en el síndrome de Hutchinson-Gilford Progeria.
- For treatment of processing-deficient Progeroid Laminopathies with either:
- Heterozygous LMNA mutation with progerin-like protein accumulation
- Homozygous or compound heterozygous ZMPSTE24 mutations.
Zokinvy no está indicado para otros síndromes progeroides o laminopatías progeroides con procesamiento competente. Basado en su mecanismo de acción, no se esperaría que fuera efectivo en estas poblaciones.
Progeria y Laminopatías Progeroides son enfermedades genéticas muy raras de envejecimiento prematuro que aceleran la muerte en pacientes jóvenes. Los niños con Progeria no tratados mueren de enfermedades cardíacas a una edad promedio de 14.5 años. Aproximadamente 20 niños y jóvenes adultos se conocen como portadores de la enfermedad en los EE. UU.
La dosificación de Zokinvy se basa en el área de superficie corporal (BSA). El costo de Zokinvy es de aproximadamente $26,000 por 30 cápsulas de 50 mg y $39,000 por 30 cápsulas de 75 mg. Como ejemplo, en un paciente que toma cuatro cápsulas de 50 mg al día, el costo sería aproximadamente $1.2 millones por año. Un médico determinará la dosis adecuada para cada paciente de forma individual.
Las advertencias y precauciones incluyen el riesgo de prolongación del QTc (una posible alteración grave del ritmo cardíaco), reducción de la eficacia o reacciones adversas debido a interacciones medicamentosas; anomalías de laboratorio, nefotoxicidad (daño renal), toxicidad retiniana (daño ocular), fertilidad afectada, toxicidad embrio-fetal (daño a un bebé no nacido).
Los efectos secundarios más comúnmente informados fueron eventos gastrointestinales (del tracto digestivo) leves o moderados (como vómitos, diarrea, náuseas), infección, disminución del apetito, fatiga e infecciones del tracto respiratorio superior, entre otros efectos secundarios.
10. Kimmtrak
- Estimated Cost: $ 1.1 million annually
- Manufacturer: Immunocore
- Use: Uveal Melanoma (a type of eye cancer)
- Initial FDA Approval Date: January 25, 2022
Kimmtrak (tebentafusp-tebn) es una inmunoterapia innovadora diseñada específicamente para tratar a pacientes adultos que padecen melanoma uveal metastásico (HLA-A*02:01). Esta forma de cáncer ocular, aunque poco común, es conocida por su agresividad y su capacidad para diseminarse a otras partes del cuerpo o por no ser operable. De hecho, se estima que hasta el 50 % de los pacientes diagnosticados con melanoma uveal desarrollarán metástasis, lo que subraya la importancia de contar con opciones de tratamiento eficaces.
Kimmtrak pertenece a la categoría de los activadores biespecíficos de células T (BiTE). Su mecanismo de acción es fascinante; se une al complejo HLA-A*02:01/gp100, un antígeno de linaje que se expresa con frecuencia en las células del melanoma uveal. A través de este vínculo, Kimmtrak desencadena una respuesta del sistema inmunitario propio del paciente, ayudando al cuerpo a reconocer y combatir las células cancerosas de manera más eficaz.
Además, es importante destacar que Kimmtrak se convirtió en el primer tratamiento que utiliza un receptor de células T (TCR) en recibir la aprobación de la FDA, lo que representa un avance significativo en el tratamiento del melanoma uveal. Este hito no solo aporta nuevas esperanzas a los pacientes diagnosticados con esta enfermedad, sino que también abre un camino para la investigación y el desarrollo de terapias inmunológicas que podrían beneficiar a quienes padecen otros tipos de cáncer en el futuro.
La inmunoterapia, y en particular Kimmtrak, ha cambiado la manera en que se aborda el tratamiento del melanoma uveal, proporcionando a los médicos y pacientes una herramienta valiosa en la lucha contra esta enfermedad compleja y a menudo mortal.
El costo de la solución intravenosa de Kimmtrak (100 mcg/0,5 mL) es de aproximadamente $20.764, lo que representa un costo anual de alrededor de $1,1 millones. Por lo general, los pacientes reciben tratamiento hasta que se produce una toxicidad inaceptable o la enfermedad progresa.
En los estudios de fase 3, la supervivencia global (SG) de Kimmtrak se comparó con la del tratamiento elegido por el investigador (pembrolizumab, ipilimumab o dacarbazina) en pacientes adultos con melanoma uveal no tratado previamente y con HLA-A*02:01 positivo. Kimmtrak demostró resultados estadísticamente y clínicamente significativos, con una mediana de supervivencia global de casi 22 meses, en comparación con los 16 meses de los otros tratamientos (una mediana de 6 meses de beneficio en la supervivencia global). La mediana indica que el 50 % de los pacientes tuvo un beneficio en la supervivencia superior a 6 meses, y el 50 % inferior a 6 meses.
Kimmtrak incluye una advertencia destacada sobre el síndrome de liberación de citoquinas (SLC), que puede ser grave o potencialmente mortal. Los efectos secundarios más comunes (que se presentan en ≥ 30 % de los pacientes) son el síndrome de liberación de citoquinas (SLC), erupción cutánea, fiebre, picazón, fatiga, náuseas, escalofríos, dolor abdominal, edema, hipotensión, piel seca, dolor de cabeza y vómitos. También se han notificado anomalías de laboratorio comunes (que se presentan en ≥ 50 %).
Nota : Esta lista no es exhaustiva en cuanto a medicamentos, efectos secundarios o precios. Los costos de los medicamentos que se muestran son estimados y están sujetos a cambios en cualquier momento. La aprobación de los medicamentos puede estar sujeta a la decisión de la FDA o del fabricante. Los precios pueden variar según la duración del tratamiento, las dosis, los factores del mercado, la ayuda financiera del fabricante u otros factores.
