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The U.S. FDA has approved Imcivree® (setmelanotide – Rhythm Pharmaceuticals) to treat of obesity and control hunger in patients who are at least six years old and have Bardet-Biedl syndrome.

• Imcivree first received FDA approval in 2020. It is also indicated for chronic weight management in patients at least six years old who have obesity due to POMC, PCSK1, or LEPR deficiency confirmed by genetic testing in which variants in POMC, PCSK1, or LEPR genes are interpreted as pathogenic, likely pathogenic, or of uncertain significance.
• Bardet-Biedl syndrome is a rare, complex genetic disorder that can cause hyperphagia, an abnormally strong feeling of hunger or desire to eat. Hyperphagia can lead to obesity and may be present as early as the patient’s first year of life.
• Recommended dosing under the new indication is based on the patient’s age, renal health, and clinical response to treatment. Imcivree is administered by subcutaneous injection.

Eli Lilly has obtained FDA approval for Olumiant® (baricitinib) oral tablets to treat severe alopecia areata in adults. Olumiant is now the first FDA-approved systemic treatment for the condition.

• Alopecia occurs when the immune system attacks hair follicles, causing hair loss. Alopecia areata describes the loss of hair in patches, usually on the face and scalp.
• Recommended Olumiant dosing for alopecia is 2mg once daily, increased to 4mg once daily if the response to treatment is not adequate. Patients who have complete or nearly complete scalp hair loss, with or without substantial eyelash or eyebrow hair loss, may start treatment at 4mg once daily. In all patients taking 4mg daily, the dose should be reduced to 2mg once daily when an adequate response has been achieved.
• Olumiant first received FDA approval in 2018. It is also indicated to treat moderately to severely active rheumatoid arthritis in adults who have had an inadequate response to one or more tumor necrosis factor blockers.

Amvuttra™ (vutrisiran) has been approved to treat polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

• A rapidly progressing disease with a poor prognosis, hATTR causes abnormal proteins to accumulate in the body’s tissues. This can lead to neuropathy, which occurs when damage or dysfunction of the nerves leads to numbness, tingling, weakness, and pain.
• Recommended dosing for Amvuttra is 25mg administered via subcutaneous injection by a healthcare professional once every three months.
• Manufacturer Alnylam Pharmaceuticals has launched Amvuttra at a wholesale acquisition cost (WAC) of $115,875 per syringe.

[Sanofi and Regeneron] The FDA approved Dupixent^® (dupilumab) for children aged 6 months to 5 years with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. A regulatory filing for this age group is under review by the European Medicines Agency and submissions to regulatory authorities in additional countries are underway.

• The approval is based on data that includes a Phase 3 trial evaluating Dupixent every four weeks (200 mg or 300 mg, based on body weight) plus low-potency topical corticosteroids (TCS) or TCS alone (placebo).
• Dupixent is a pre-filled syringe and is administered every four weeks based on weight as an injection under the skin.

CellCept® (mycophenolate mofetil) has received an expanded indication for use in recipients of allogenic heart, kidney, or liver transplants who are at least three months old. It is used in combination with other immunosuppressants to prevent organ rejection.

• The updated indication allows for CellCept’s use in pediatric recipients of allogenic heart and liver transplants. The drug, which first received FDA approval in 1995, was previously indicated for these uses in adults only.
• CellCept is available in oral form as tablets, capsules, and an oral suspension. It is also available as an intravenous (IV) infusion that can be used up to 14 days for patients unable to take one of the oral formulations, though patients should be switched to oral dosing as soon as possible. Recommended dosing is based factors that include the patient’s age and body surface area, the type of transplant received, and the dosage form being used.

Regeneron’s Dupixent® (dupilumab) is now indicated to treat patients at least six months of age who have moderate-to-severe atopic dermatitis that is not adequately controlled with topical prescription therapies or for whom those therapies are not advisable. It was previously approved to treat atopic dermatitis only in patients at least six years of age.

• First FDA approved in 2017, Dupixent is also indicated to treat asthma, eosinophilic esophagitis, and chronic rhinosinusitis with nasal polyposis.
• Recommended dosing for atopic dermatitis in children is based on the patient’s age and weight. Maintenance dosing can range from 200mg once every four weeks to 300mg once every two weeks. Dupixent is given by subcutaneous injection and can be administered at home.

The U.S. FDA has approved an expanded indication for Sanofi’s Lantus® (insulin glargine) to improve glycemic control in adult and pediatric patients who have diabetes mellitus (type 1 or type 2).

• Prior to this expansion, Lantus was indicated to improve glycemic control in adults who had type 1 or type 2 diabetes mellitus, and in pediatric patients who had type 1 diabetes mellitus.
• Lantus dosing is individualized based on the patient’s metabolic needs, blood glucose monitoring, glycemic control, type of diabetes, and prior insulin use.
• The FDA first approved Lantus in 2000.

Amgen’s Riabni™ (rituximab-arrx), a biosimilar for Rituxan® (rituximab – Genentech and Biogen), is now FDA approved to for use with methotrexate to treat moderately to severely active rheumatoid arthritis (RA) in adults who have had an inadequate response to one or more tumor necrosis factor antagonist therapies.

• The FDA first approved Riabni in 2020 to treat certain adults diagnosed with non-Hodgkin lymphoma, chronic lymphocytic leukemia, granulomatosis with polyangiitis (Wegener’s granulomatosis), and microscopic polyangiitis.
• Riabni’s recommended dosing for RA is two 1,000mg intravenous infusions separated by two weeks (one treatment course) every 24 weeks. If appropriate based on clinical evaluation, the time between treatment courses can be shortened to as little as 16 weeks.

GSK’s Priorix® (measles, mumps, and rubella vaccine, live) has gained FDA approval to provide active immunization for the prevention of measles, mumps, and rubella (MMR) in individuals who are at least 12 months old.

• Recommended dosing is one 0.5mL subcutaneous injection given at 12-15 months of age, followed by a second 0.5mL injection administered at 4-6 years of age.
  • If Priorix is not given on this schedule, the patient should receive the first and second doses at least four weeks apart.
  • Priorix can also be used to provide a second MMR vaccine dose for patients who have received a different MMR vaccine for their first dose.
• Priorix has launched at wholesale acquisition cost (WAC) of $87.31 per dose.

TG Therapeutics is removing Ukoniq® (umbralisib) from the U.S. market due to a potential increase in the risk of patient death.

• In 2021, the U.S. FDA granted accelerated approval for Ukoniq to treat marginal zone lymphoma and follicular lymphoma. Continued approval was contingent on the results of confirmatory trials.
• The FDA announced in February 2022 that it was investigating a possible increased risk of death in patients treated with Ukoniq. The agency advised healthcare professionals and patients to talk about the potential risks and benefits of the medication versus other available treatments.
• As of June 2022, the FDA concluded that the risks of treatment with Ukoniq outweigh the potential benefits. TG Therapeutics chose to voluntarily withdraw the drug from the market following this determination.