Brief about Orphan Drugs and examples of it

An orphan drug is a pharmacological agent produced to treat medical disorders that would be unprofitable to produce without government aid due to their rarity. Orphan diseases are the names given to these illnesses.

Orphan medications include the following:

Ivacaftor is a treatment for cystic fibrosis, a hereditary illness that affects roughly 30,000 Americans and causes respiratory and digesting issues.

Alglucerase is a Gaucher disease medication that causes discomfort and tissue damage in the liver, spleen, lungs, and bone marrow.

Coagulation factor IX is a drug that helps people with haemophilia B prevent bleeding.

Imatinib is a cancer treatment that is used to treat specific kinds of leukaemia.

Rucaparib is an ovarian cancer therapy.

According to the "Coherent Market Insights" Global Industry Insights, Trends, Outlook, and Opportunity Analysis of Orphan Drugs Market.


Orphan Drugs Market
Orphan Drugs Market


It can be expensive to develop a medicine for a rare ailment that only affects a small number of people. Congress passed the Orphan Drug Act in 1983 to encourage pharmaceutical companies to produce more orphan medications. This law incentivizes drug companies to develop and manufacture orphan medications by providing tax breaks, clinical research funding, and the exclusive right to market these drugs for seven years.

The law has had a positive impact, with the number of orphan medications continuously increasing since 1983. According to the National Organization for Rare Disorders, the FDA approved over 600 orphan medicines between 1983 and 2020. (NORD). Prior to the passage of the Orphan Drug Act, there were only 38 orphan medications.

Cystic fibrosis, Lou Gehrig's illness, and Tourette's syndrome are all examples of rare disorders. However, they also include conditions like acromegaly, sometimes known as "gigantism," and the strangely termed Jumping Frenchman of Maine, an incredibly unusual disorder that causes a strong startle reaction to unexpected sounds or images. Every year, new uncommon diseases are identified. The majority are inherited, but others might be caused by environmental or hazardous factors.

Despite the fact that each disease affects a small number of individuals, the country's population of over 30 million people is affected by one of the 7,000 rare diseases. Many rare diseases are fatal, and children make up more than half of those who are affected. Only about ten percent of all rare diseases have FDA-approved therapies.

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