Drug Pipeline News

Bluebird bio has announced that their SKYSONA therapy has been approved for pediatric use by the FDA.

Aro Biotherapeutics’ ABX1100, used to treat Pompe disease, was granted a Rare Pediatric Drug designation by the FDA.

In a recent article, researchers discussed current atopic dermatitis treatments, along with the need for safe and effective long-term treatments.

Improvements in atopic dermatitis symptoms were seen in children taking dupilumab, with minimal adverse events.

It was recently announced that oral Otezla is efficient in treating pediatric patients with moderate to severe plaque psoriasis.

Olipudase alfa-rpcp (Xenpozyme; Sanofi) is the first disease-specific, and now, only approved therapy for the treatment of acid sphingomyelinase deficiencies in pediatric and adult patients.

Ibrutinib (IMBRUVICA; AbbVie) was approved by the US Food and Drug Administration for use in pediatric patients with chronic graft versus host disease.

A phase 3 trial of dupilumab (Dupixent; Sanofi and Regeneron) has shown positive results for use in children aged 1 to 11 years afflicted with eosinophilic esophagitis.

After a recent FDA approval, Mirena can be used to prevent pregnancy for up to 8 years.

Following its FDA approval, Zynteglo will be used to treat beta-thalassemia in adults and pediatric patients who require a regular red blood cell transfusion.

Xofluza is now the first single-dose oral medication for influenza approved for children aged less than 12 years.

The FDA has granted Emergency Use Authorization (EUA) of the JYNNEOS vaccine for pediatric patients <18 years who are at high risk of monkeypox infection.

If approved, ibrexafungerp would be the first and only approved therapy for both the treatment of vulvovaginal candidiasis (VVC) and the prevention of recurrent vulvovaginal candidiasis (RVVC).

This decision by the US Food and Drug Administration marks the first approved treatment for pediatric lupus nephritis.

What we know about monkeypox in pregnancy, including treatment recommendations, vaccination, and postpartum care.

VIVUS LLC announced the US Food and Drug Administration's approval of QSYMIA (phentermine and topiramate extended-release capsules) for the treatment of obesity in adolescents ages 12 to 17 years.

The approval represents an adjunctive therapy option for pediatric patients older than age 16, as well as adults.

If approved, trofinetide has the potential to be a new treatment for Rett syndrome in pediatric and adult patients 2 years and older.

Opzelura (ruxolitinib) cream is the first and only FDA-approved treatment for adult and pediatric patients 12 years of age or older with vitiligo.

Nonstimulant drugs and even nonmedication options are available for use.

Cynthia J. Trickett, PA-C, MPAS, discusses the personal and clinical effects of atopic dermatitis in adult and pediatric patients at the 2022 Maui Dermatology Conference for PAs & NPs.

Ruling comes in cases of physicians serving in prison for convictions over prescription drugs.

Recently, the FDA announced the approval of supplemental indication for phentermine and topiramate extended-release capsules for chronic weight management in children aged 12 years and older.

Entrepreneur beats prices of supply chain wholesalers, PBMs, pharmacies, and insurers.

Data from a recent study of once weekly somapacitan injection shows positive results for prepubertal children with growth hormone deficiency (GHD).

Currently approved for the treatment of vulvovaginal candidiasis, and if the sNDA is approved, ibrexafungerp would be the first and only oral non-azole medication for the prevention of recurrent VVC.

The FDA recently announced the acceptance of a supplemental New Drug Application for relugolix 40 mg, estradiol 1 mg, and norethindrone acetate .5 mg in the treatment of heavy menstrual bleeding associated with uterine fibroids in premenopausal women.

In a video for Contemporary Pediatrics®, Tina Tan, MD, FAAP, FIDSA, FPIDS, discusses an expansion of vaccine access likely to come with the FDA approval of the PRIORIX vaccine.

Verrica received a Complete Response Letter from the FDA regarding their New Drug Application for the topical molluscum contagiosum treatment, VP-102.

Evinacumab phase 3 results show a 48% LDL-C reduction in children with homozygous familial hypercholesterolemia.