Top 5 Costliest Drugs in the World

Why cost is higher?

The development of any drug requires lots of time, effort, and crores of rupees. If the disease condition is rare, also referred to as an ‘órphan disease’, which affects only a small percentage of the population. Then the development of highly specialized ‘órphan drugs’ is even more complicated because they are not profitable enough to produce. That’s why the fewer the cases the higher its price will be.

Here’s the list of Top 5 Costliest drugs in the world. The price is converted from US Dollars to Indian Rupees.

5. Luxturna

Company: Spark Therapeutics
FDA Approval: 2017
Use: LUXTURNA (voretigene neparvovec-rzyl) is a prescription gene therapy product used for the treatment of patients with inherited retinal disease due to mutations in both copies of the RPE65 gene.
How it works: LUXTURNA uses the adeno-associated viral vector serotype 2 (AAV2) to carry a functional copy of the RPE65 gene into the retinal pigment epithelial (RPE) cells to compensate for the RPE65 mutation.
Dosage form: It is one-time gene therapy. It is administered via subretinal injection following a vitrectomy.
Cost in India: 60-80 lacs (30-40 lacs per eye)

4. Myalept (Metreleptin)

Company: AstraZeneca. Rights to the drug were divested to Aegerion Pharmaceuticals later in 2014
FDA Approval Year: 2014
Use: Myalept is used in leptin-deficient people who have Generalized lipodystrophy, a rare disease that results in having little to no fat tissue all over the body. Leptin is important for regulating key metabolic processes. Its deficiency may lead to high triglycerides (fat in the blood) and high blood sugar.
How it works: Myalept treats problems caused by not having enough leptin in the body (leptin deficiency). Because Myalept acts similarly to the body’s naturally occurring leptin, it can help treat problems in people with Generalized lipodystrophy that result from not having enough leptin.
Dosage form: Powder for reconstitution; injected S.C.
Cost in India: 5-6 Crores yearly

3. Danyelza (naxitamab-gqgk)

Company: Y-mAbs Therapeutics, Inc.
FDA Approval: 2020
Use: for the Treatment of Neuroblastoma
How it works: GD2 is a disialoganglioside that is overexpressed on neuroblastoma cells and other cells of neuroectodermal origin, including the central nervous system and peripheral nerves. Danyelza binds to the glycolipid GD2 and induces complement-dependent cytotoxicity (CDC) and antibody-dependent cell-mediated cytotoxicity (ADCC).
Dosage Forms: 3 mg/kg/day (up to 150 mg/day), administered as an intravenous infusion after dilution on Days 1, 3, and 5 of each treatment cycle.
Cost in India: 7-8 Crores yearly

2. Zokivny ( Lonafarnib)

Company: Eiger BioPharmaceuticals
FDA Approval: 2020
Use: Zokinvy is the first and only treatment to target the cause and symptoms of progeria, also known as Hutchinson-Gilford progeria syndrome, and processing-deficient progeroid laminopathies (PDPL) in patients ages 1 and above. These illnesses are caused when the body makes harmful proteins called progerin and progerin-like proteins.
How it works: It interrupts a key step in the disease process. It is a farnesyltransferase inhibitor, helping to block progerin and progerin-like proteins from building up and damaging cells.
Dosage forms: It is available in 50-mg and 75-mg capsules to provide administration options for starting dosage and maintenance dosing. Zokinvy capsules are taken twice a day with meals.
Cost in India: 7-7.7 Crores Yearly

1. Zolgensma (onasemnogene abeparvovec-xioi)

Company: Novartis
FDA Approval: 2019
Use: It is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA).
How it works: It targets the genetic root cause of SMA by replacing the function of the missing or nonworking gene, called the SMN1 gene. This gene is critical to making SMN protein. SMN protein is essential to motor neuron cell survival. These cells control muscle function. Without SMN protein, motor neuron cells die, causing muscles to become so weak that breathing, eating, and moving becomes difficult, and the condition is likely to become life-threatening in its most severe forms.
Dosage forms: Suspension for Intravenous Infusion
Cost in India: 18 Crores

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