From the FDA Drug Label
POMALYST is a thalidomide analogue Thalidomide is a human teratogen, inducing a high frequency of severe and life-threatening birth defects such as amelia (absence of limbs), phocomelia (short limbs), hypoplasticity of the bones, absence of bones, external ear abnormalities (including anotia, micropinna, small or absent external auditory canals), facial palsy, eye abnormalities (anophthalmos, microphthalmos), and congenital heart defects. The thalidomide tragedy refers to the severe birth defects caused by thalidomide, a human teratogen.
- Thalidomide can cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy.
- The drug can cause severe and life-threatening birth defects, including amelia, phocomelia, and congenital heart defects.
- Due to its teratogenic effects, thalidomide is only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) 1, 1.
From the Research
The thalidomide tragedy was a devastating pharmaceutical disaster that occurred in the late 1950s and early 1960s, causing severe birth defects in thousands of children worldwide, and its use today is strictly controlled due to its known teratogenic effects, particularly in pregnant women 2.
Key Points
- The thalidomide tragedy led to fundamental changes in drug approval processes worldwide, with much stricter requirements for testing medications, particularly regarding their effects during pregnancy 3.
- Thalidomide is used under strict controls for conditions like multiple myeloma and leprosy complications, but pregnant women must absolutely avoid it due to its known teratogenic effects 4.
- The medication caused phocomelia, a condition where babies were born with severely underdeveloped or absent limbs, along with other congenital abnormalities affecting the ears, eyes, heart, and internal organs 5.
- Approximately 10,000-20,000 children were affected globally before the connection was established, and the FDA's refusal to approve thalidomide in the United States prevented a wider disaster 6.
Current Use of Thalidomide
- Thalidomide is approved for use in acute erythema nodosum leprosum and, in combination with dexamethasone, in newly diagnosed myeloma 6.
- It is also used in the treatment of multiple myeloma, leprosy complications, and other conditions, but its use is limited by toxicity and restricted access 4, 3.
Important Considerations
- Thalidomide has several targets and mechanisms of action, including a hypnosedative effect, immunomodulatory properties, and antiangiogenic action 4.
- Its use is associated with several side effects, including teratogenicity, peripheral neuropathy, and deep vein thrombosis, and close monitoring is recommended to prevent these adverse effects 4, 5.