MOST EXPENSIVE PHARMACEUTICALS APPROVED BY THE US FDA
1. Hemgenix (etranacogene dezaparvovec)
Cost of therapy: $3.5 million
Manufacturer: CSL Behring
Indication: Hemophilia B
Hemgenix is indicated for treating haemophilia B patients under Factor IX prophylaxis and has life-threatening historical bleeding.
Fifty-four adults with moderately severe or severe haemophilia B were included in the clinical trials. 37% of patients treated with Hemgenix had sustained average factor IX activity at two years, and there was a more excellent bleed protection (54% reduction of all bleeds). Further, gene therapy reduced the need for prophylaxis; 94% of patients remained free of prophylaxis.
Hemgenix utilizes a gene therapy technique known as gene transfer, which involves introducing a functional gene into the body. This approach aims to enable the production of functional factor IX, a protein necessary for normal blood clotting, thereby enhancing the body's natural clotting ability.
2. Skysona (elivaldogene autotemcel)
Cost of therapy: $3.0 million
Manufacturer: bluebird bio
Indication: Cerebral adrenoleukodystrophy (CALD)
Skysona was approved for treating early, active cerebral adrenoleukodystrophy (CALD). It is indicated for children of 4 to 17 years of age.
The efficacy of Skysona was established in an open-label, single-arm study. At 24 months, MFD-free survival was 72% in patients on Skysona versus 43% in patients in untreated patients.
3. Zynteglo (betibeglogene autotemcel)
Cost of therapy: $2.8 million
Manufacturer: bluebird bio
Indication: ß-thalassemia
Zynteglo was approved for treating adults and paediatrics with ß-thalassemia, requiring red blood cell transfusions.
The efficacy was established in two Phase 3 studies; the trial was conducted for 24 months initially to determine the independence from transfusions.
89% of patients in the clinical trials stopped transfusions. The average haemoglobin levels of patients who achieved transfusion independence were 11.5 g/dL.
4. Roctavian (valoctocogene roxaparvovec)
Cost of therapy: $2.9 million
Manufacturer: BioMarin Pharmaceuticals
Indication: Haemophilia A
BioMarin Pharmaceuticals' Roctavian was approved for treating severe haemophilia A, costing $2.9 million per patient in the US. This is the first gene therapy developed for treating haemophilia A patients.
The approval was based on the GENEr8-1 study, a Phase 3 trial demonstrating the efficacy in 134 patients. The mean annualized bleed rate was decreased by 52% in patients treated with Roctavian. The trial also demonstrated that the efficacy was persistent beyond three years.
5. Zolgensma (onasemnogene abeparvovec)
Cost of therapy: $2.1 million
Manufacturer: Novartis
Indication: Spinal muscular atrophy (SMA)
Developed by Novartis, Zolgensma is a gene therapy designed to treat spinal muscular atrophy (SMA), a rare genetic disease that impacts muscle strength and movement. With an estimated cost of around $2.1 million per dose, Zolgensma is is one of the most expensive drugs worldwide.
The efficacy of Zolgensma is established in multiple studies, STR1VE and START enrolled children with SMA. In the STR1VE study, the average age of dosing is 3.7 months. After 14 months, 91% of patients were alive and did not require breathing support. At 18 months, nearly 59% of children could sit without support for more than 30 seconds. It further helped patients to thrive; 55% of patients could swallow thin liquids, 86% had freedom from non-oral feeding support, and 64% could maintain weight.
6. Zokinvy (lonafarnib)
Cost of therapy: $1.0 million
Manufacturer: Eiger Biopharmaceuticals
Indication: Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient Progeroid Laminopathies
Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient Progeroid Laminopathies are genetic disorders characterised by the accumulation of harmful proteins in the body, which leads to premature ageing. Zokinvy acts by blocking the building up of progerin and progerin-like proteins.
After an average follow-up of 11 years, treatment with Zokinvy increased the average life span by 2.5 years.
7. Danyelza (naxitamab)
Cost of therapy: $1.0 million
Manufacturer: Y-mAbs Therapeutics
Indication: Relapsed or refractory high-risk neuroblastoma
Danyelza is indicated for treating children one year of age and older and adults with high-risk neuroblastoma in the bone or bone marrow.
The FDA approval was based on Study 201 and Study 12-230. In Study 201, 45% (10/22) of patients showed an objective response rate, and 36% showed a complete response. 30% of patients showed no growth of cancer or spreading for six months.
8. Myalept (metreleptin)
Cost of therapy: $930,000
Manufacturer: Amryt Pharmaceuticals
Indication: Generalized lipodystrophy
Myalept is a leptin replacement therapy used for treating patients with generalized lipodystrophy. The efficacy is demonstrated in an open-label, single-arm study.
9. Luxturna (voretigene neparvovec)
Cost of therapy: $850,000
Manufacturer: Spark Therapeutics
Indication: Inherited retinal disease (IRD)
In 2017, Luxturna was approved by the US Food and Drug Administration (FDA) for treating adult patients and children with inherited vision loss.
The efficacy was established by a trial including 41 patients aged between 4 and 44 years. The primary endpoint is assessing the patient's ability to navigate the obstacle at different light levels. Patients administered with Luxturna showed significantly improved navigation compared to the control group.
10. Brineura (cerliponase alfa)
Cost of therapy: $750,000
Manufacturer: BioMarin Pharmaceuticals
Indication: Ceroid lipofuscinosis type 2 (CLN2)
Brineura is indicated for treating pediatric patients three and older with ceroid lipofuscinosis type 2 (CLN2). It is an enzyme replacement therapy and is directly administered to cerebrospinal fluid.
The efficacy is administered in 24 children; 95% of patients on Brineura did not experience a decline in their ability to walk.