Setmelanotide (Imcivree) is approved for chronic weight loss and weight management in patients ages six years and older with rare genetic conditions that lead to obesity. These conditions are pro-opiomelanocortin deficiency, proprotein subtilisin/kexin type 1 deficiency, and leptin receptor deficiency. People with these conditions become severely obese very young owing to low metabolism rates and impairment of the ability to feel full or satiated. Setmelanotide stimulates areas of the brain that regulate appetite and fullness. It does not correct the underlying genetic variation. The drug is given by subcutaneous injection.

The most common adverse effects of setmelanotide are injection site reactions, skin hyperpigmentation, nausea, headache, diarrhea, abdominal pain, back pain, fatigue, vomiting, depression, upper respiratory tract infection, and spontaneous penile erection.

For complete prescribing information for setmelanotide, go to http://www.accessdata.fda.gov/drugsatfda_docs/label/2020/213793s000lbl.pdf.

Lumasiran (Oxlumo) is the first drug approved to treat primary hyperoxaluria type 1, a rare genetic disorder that leads to overproduction of oxalate; in combination with calcium, oxalate can create renal calculi that can be deposited in the kidneys and lead to progressive kidney damage. High levels of oxalate can also damage other organs such as the heart, bones, and the eyes. Lumasiran decreases oxalate production.

Nurses should teach patients how to administer lumasiran subcutaneously and about its most common adverse effects of injection site reaction and abdominal pain.

For complete prescribing information for lumasiran, go to http://www.alnylam.com/wp-content/uploads/pdfs/OXLUMO-Prescribing-Information.pd.

Lonafarnib (Zokinvy) is the first drug approved to treat Hutchinson-Gilford progeria syndrome and progeroid laminopathies, rare genetic diseases leading to accelerated premature aging and death. Children with these disorders are born with normal appearance, but by nine to 24 months characteristic facial abnormalities begin to show, and alopecia develops. The child has delays in growth, a short stature, and low weight. These children develop widespread arteriosclerosis resulting in the normal complications of this condition (hypertension, stroke, angina, enlarged heart, and heart failure), leading to death. The average age of death from arteriosclerosis is 13 years.

Lonafarnib inhibits farnesyltransferase, preventing accumulation of progerin and progerin-like proteins in the inner nuclear membrane. The most common adverse reactions are vomiting, diarrhea, infection, nausea, decreased appetite, fatigue, upper respiratory tract infection, abdominal pain, musculoskeletal pain, electrolyte abnormalities, decreased weight, headache, myelosuppression, increased liver enzymes, decreased blood bicarbonate, cough, and hypertension.

Nurses should teach families to give the child the oral capsule twice a day with meals. Lonafarnib capsules can be either swallowed whole with water or opened and mixed with Ora Blend SF, Ora-Plus, orange juice, or applesauce. Juices that contain grapefruit or Seville orange juice should be avoided to prevent drug interactions that can increase the risk of adverse effects.

For complete prescribing information for lonafarnib, go to https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/213969s000lbl.pdf.