The top orphan drug based on revenue in 2021 was Imbruvica.2. Top Selling Orphan Drugs (Forecast 2026)
- 2.1. Ibrutinib (Brand Name Imbruvica®)
- 2.3. Olaparib.
- 2.4. Ruxolitinib (Brand Name Jakafi®)
- 2.5. Venetoclax (Brand Name Venclexta®)
- 2.6. Acalabrutinib (Brand Name Calquence®)
- 2.7. Tafamidis (Brand Name Vyndaqel®)
As of 2019, overall development for rare diseases takes nearly four years longer than non-rare disease conditions and the success rate of orphan drugs in clinical trials is estimated to be only six percent. Despite significant progress in the fight to treat rare diseases, unmet patient needs remain.
What is the first approved orphan drug : The Food and Drug Administration (FDA) grants first marketing approval to an orphan drug, Panhematin®, for acute intermittent porphyria and other acute porphyrias.
What is an ultra orphan drug
Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening.
Are orphan drugs profitable : Orphan drugs have become a lucrative business opportunity for drug makers. Pharmaceutical companies are seeking “orphan” status to develop blockbuster drugs used to treat other common medical conditions.
Both, Humira and Lipitor, are currently the leading drugs by lifetime sales.
LIPITOR
Which Is the Most Profitable Pharmaceutical Drug Ever The most lucrative pharmaceutical drug in the industry is LIPITOR, developed, marketed, and sold by the PFIZER corporation. During its patent protection period, LIPITOR amassed a staggering $131 billion in sales.
Can orphan drugs be profitable
Sean Tu, a College of Law professor found lucrative so-called “orphan” drugs earn manufacturing tax credits, have longer patent exclusivities and face easier Food and Drug Administration review.Which country has the most orphans Several countries have larger than average orphan rates. The countries with the highest rates are Russia, Iran, Colombia, Brazil, Mexico, South Africa, and Peru.Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening.
RPI Deficiency
This is considered to be the rarest disease in the world. Ribose-5-Phosphate Isomerase (RPI), is a crucial enzyme in a metabolic process in the human body. This condition can cause muscle stiffness, seizures, and reduction of white matter in the brain.
How much do ultra orphan drugs cost : For 147 on-patent drugs with available data in 2023, monthly prices were higher for ultra-rare orphan indications than for rare or common orphan indications and non-orphan indications ($70 128 (£55 971; €63 370) v $33 313 v $16 484 v $14 508; P<0.001).
What is the most profitable drug ever : Humira – $19.9bn
Abbvie's Humira contributed almost 61% to the company's total revenue in 2018, making it the most profitable product. With a 7% growth in net sales worldwide in 2018, the drug is crucial for the treatment of rheumatoid arthritis.
What is the average cost of orphan drugs
The median treatment cost was USD 218,872 for orphan drugs (IQR = USD 231,057, range USD 237–USD 1,272,021) and USD 12,798 for non-orphan drugs (IQR = USD 57,940, range USD 44–USD 382,866, p < 0.001; Figure 1; Appendix A, Table A1). New drugs treatment cost at US market entry (USD 2021) and median WAC.
Atorvastatin
The Top 300 Drugs of 2021
| Rank | Drug Name | Total Prescriptions (2021) |
|---|---|---|
| 1 | Atorvastatin | 116,702,335 |
| 2 | Metformin | 91,151,043 |
| 3 | Levothyroxine | 89,309,050 |
| 4 | Lisinopril | 88,272,557 |
Actinium-225
An alpha-emitting isotope with a short half-life, Actinium-225 is so rare that the annual global production is less than a grain of sand. Given this scarcity, it should come as no surprise that the isotope has been dubbed 'the rarest drug on Earth.
What is a blockbuster drug : A blockbuster drug is a very popular drug that generates annual sales of $1 billion or more for the company that sells it. Common blockbuster drugs are Vioxx, Lipitor, and Zoloft.