Drug Pipeline News

An advisory panel for the US Food and Drug Administration voted 14-1 to withdraw 17α-hydroxyprogesterone caproate (Makena; Covis Pharma) from the market.

Dr. Hyams, MD, discusses functional constipation in children and how positive results from a phase 3 trial on linaclotide will change treatment.

Moderna has announced that their latest BA.4/BA.5 Omicron-targeting bivalent COVID-19 booster vaccine, mRNA-1273.222, has been granted Emergency Use Authorization by the US Food and Drug Administration.

Eagle Pharmaceuticals and Enalare Therapeutics have announced that their ENA-001 drug for the treatment of Apnea of Prematurity has been granted an Orphan Drug Designation by the US Food and Drug Administration.

The US Food & Drug Administration approved tetanus toxoid, reduced diptheria toxoid and acellular pertussis vaccine, adsorbed [Tdap] (Boostrix, GSK) today for immunization during the third trimester of pregnancy to prevent pertussis—or whooping cough—in infants under 2 months of age.

The approval, awarded to Alnylam Pharmaceuticals, is based on the results of the ILLUMINATE-C phase 3 trial.

Pfizer and BioNTech have applied to the FDA for Emergency Use Authorization of their Omicron BA.4/BA.5-adapted bivalent vaccine booster for use in pediatric patients aged 3 to 11 years.

AVROBIO’s AVR-RD-04 therapy has been granted rare pediatric disease designation to treat cystinosis in pediatric patients.

In 2 recent phase 3 trials, safety and efficacy was proven in the use of roflumilast cream 0.3% for the treatment of plaque psoriasis.

Sodium thiosulfate has been approved by the FDA to treat ototoxicity in pediatric patients with localized, non-metastatic solid tumors.

After promising outcomes in an ongoing phase 2 trial, the FDA has given Pfizer’s GBS6 vaccine Breakthrough Therapy Designation against Group B Streptococcus in infants.

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.