Saturday, August 19, 2023

What is an Orphan Drug? Detail information on Orphan Drugs

Orphan Drug Names Approved by the US FDA


The development and approval of medications for rare diseases have historically presented unique challenges due to the limited patient populations and financial incentives for pharmaceutical companies. However, with the establishment of the Orphan Drug Act in 1983, the United States Food and Drug Administration (FDA) created a framework to incentivize the development of drugs for rare diseases, known as orphan drugs. In this article, we will explore the concept of orphan drugs, the significance of their unique names, and some examples of drugs approved by the FDA under this designation.

Understanding Orphan Drugs

An orphan drug is a medication specifically developed to treat rare diseases or conditions affecting a small number of people. In the United States, a rare disease is defined as a condition that affects fewer than 200,000 individuals, although this threshold may vary in other countries. The Orphan Drug Act was passed to address the significant unmet medical needs of patients with rare diseases by providing incentives to pharmaceutical companies for investing in research and development.

One of the key incentives offered to manufacturers of orphan drugs is market exclusivity for a designated period. During this time, no other company can market the same drug for the same indication, providing a unique commercial advantage for the developer. Additionally, the FDA waives certain fees and provides support for clinical trial design and drug development processes, reducing the financial burden associated with orphan drug development.

Orphan Drug Naming Conventions

To help differentiate orphan drugs and facilitate proper identification, the FDA follows specific naming conventions for these medications. Orphan drug names usually contain a suffix that distinguishes them from other drugs on the market. This suffix is composed of four lowercase letters and is added to the nonproprietary (generic) name of the drug.

The inclusion of the suffix in the orphan drug name allows healthcare providers, patients, and pharmacists to recognize the drug as an orphan product and reinforces its unique status as a medication developed for a rare disease. Moreover, this naming system enhances pharmacovigilance efforts, enabling easier tracking and monitoring of the drug's use and potential adverse effects.

Examples of Orphan Drug Names Approved by the US FDA

Lumacaftor/Ivacaftor (BRAND: Orkambi®)

Indication: Treatment of cystic fibrosis in patients aged 2 years and older who have two copies of the F508del mutation in the CFTR gene or who have at least one mutation that is responsive to treatment with lumacaftor/ivacaftor.

Eculizumab (BRAND: Soliris®)

Indication: Treatment of paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and generalized myasthenia gravis (gMG).

Ibrutinib (BRAND: Imbruvica®)

Indication: Treatment of various hematological malignancies, including mantle cell lymphoma, chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma, and Waldenström's macroglobulinemia.

Nusinersen (BRAND: Spinraza®)

Indication: Treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.


Orphan drugs play a crucial role in addressing the unmet medical needs of patients with rare diseases, offering hope and improved quality of life to those affected by these conditions. The US FDA's Orphan Drug Act has been instrumental in promoting the development of these life-changing medications, providing incentives to pharmaceutical companies and streamlining the approval process.

The unique names assigned to orphan drugs through the inclusion of a suffix help differentiate them in the marketplace and support pharmacovigilance efforts. As research and innovation continue to advance, we can expect the list of orphan drugs to grow, offering renewed hope and promising therapeutic options to individuals and families impacted by rare diseases

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