Pharmacological Profile of FDA-Approved Orphan Drugs in the Year 2022

This manuscript aimed to provide a scientific report of recently FDA-approved orphan drugs for helping patients, researchers, clinicians, and academicians engaged in rare diseases.

The development of an orphan drug is really challenging (due to the small number of potential patients and the poor business potential despite substantial financial investment and the use of other resources) but vital (as it creates new hope for patients and families affected by rare diseases) process. FDA approved about 54% (20 out of 37) novel orphan drug therapies for varying rare diseases such as refractory follicular lymphoma, acid sphingomyelinase deficiency, amyotrophic lateral sclerosis, and generalized pustular psoriasis. It has been reported that 50% of these orphan drugs (e.g., adagrasib, futibatinib, pacritinib, olutasidenib, and tebentafusp-tebn) are recommended for uncommon or care cancers such as KRAS G12C mutated form of non-small cell lung cancer, locally advanced or metastatic intrahepatic cholangiocarcinoma (cancer of the intrahepatic part of the bile duct), intermediate or high-risk primary or secondary myelofibrosis, relapsed or refractory acute myeloid leukemia with susceptible IDH1 mutation, unresectable or metastatic uveal melanoma. This manuscript, describing the pharmacological aspects such as therapeutic applications, mechanisms of action, pharmacokinetics, adverse effects, doses, and special cases particularly in pediatrics, geriatrics, pregnant women, and lactating mothers, summarized twenty orphan drugs approved by the FDA in the year 2022 and price (for determining the cost of therapy), shall serve as a helpful document for concerned patients and clinicians.

This manuscript, the pharmacological report of recently FDA-approved twenty orphan drugs depicting the pharmacological profile, serves as an essential handy document for health professionals as well as patients concerned with rare diseases.