Drug Pipeline News

Improvement in the left ventricular ejection fraction (LVEF) and statistically significant benefit in the Performance of the Upper Limb (PUL v2.0) scale were observed following 2 years of CAP-1002 treatment in Duchenne muscular dystrophy (DMD) patients.

According to Italfarmaco Group, a New Drug Application (NDA) for givinostat has been accepted and granted priority review by the FDA to treat Duchenne muscular dystrophy (DMD) patients, following positive topline phase 3 trial results.

Indicated for growth hormone deficiency (GHD) patients aged 3 years and older, somatrogon-ghla is now FDA-approved to treat GHD following positive phase 3 data demonstrating non-inferiority, measured by annual height velocity at 12 months, compared to somatropin.

After receiving accelerated approval, delandistrogene moxeparvovec-rokl is the first gene therapy to treat Duchenne muscular dystrophy (DMD) in ambulatory pediatric patients aged 4 to 5 years.

Children 10 years and older with type 2 diabetes (T2D) can now be treated with empagliflozin and empagliflozin plus metformin hydrochloride to improve blood sugar control, per a recent approval by the FDA.

With a newly FDA-approved indication, odevixibat can now be used to treat cholestatic pruritus in patients 12 months and up with Alagille syndrome (ALGS).

Kirk Thame, MD, associate professor, pediatric gastroenterology, hepatology, and nutrition, Monroe Carell Jr. Children’s Hospital at Vanderbilt, Nashville, Tennessee, explains how linaclotide differs from other treatment options for pediatric functional constipation, and why children will benefit from its recent FDA approval.

This decision marks the first functional constipation treatment approved by the FDA for this patient population.

The newly FDA-approved prescription product delivers nalmefene to the nasal cavity to reverse the effects of opioid overdose.

Patients using adalimumab-adbm for chronic inflammatory disease treatment such as juvenile idiopathic arthritis (JIA), Crohn’s disease, and others, will have an autoinjector option beginning July 1, 2023, according to Boehringer Ingelheim.

Following the FDA approval of beremagene geperpavec (B-VEC) (Vyjuvek; Krystal Biotech) to treat dystrophic epidermolysis bullosa (DEB) patients aged 6 months and older, Bernard A. Cohen, MD, discusses its safety and efficacy and how it can change the treatment landscape for DEB.

The FDA has approved Krystal Biotech's beremagene geperpavec (B-VEC) (Vyjuvek; Krystal Biotech) to treat the wounds of dystrophic epidermolysis bullosa (DEB) patients aged 6 months and older. B-VEC is the first FDA-approved gene therapy treatment for DEB, according to the agency. In this Contemporary Pediatrics interview, Raj Chovatiya, MD, PhD, breaks down this much-anticipated FDA approval.

The FDA Advisory Committee recently voted in support of approval for Pfizer's maternal immunization vaccine to help prevent RSV in infants. In this Contemporary Pediatrics® interview, Tina Tan, MD, FAAP, FIDSA, FPIDS, pediatric infectious diseases attending, Ann & Robert H. Lurie Children's Hospital, Chicago, explains what this means for this patient population ahead of an expected FDA decision in August, 2023.

This formulation of ibuprofen is the only non-opioid product approved to treat pain in infants that is delivered through injection, according to Cumberland Pharmaceuticals.

The once-daily topical cream for the treatment of atopic dermatitis (AD) has demonstrated positive topline results in 2 phase 3 studies in adults and children aged 2 years and up. According to Dermavant, a supplemental new drug application (sNDA) filed with the FDA is anticipated in Q1 of 2024.

The REFLECT Scoliosis Correction System from Globus Medical is intended to correct progressive scoliosis in pediatric patients while simultaneously maintaining stability and motion as well as allowing for future modulated growth.

Nirsevimab’s efficacy against infant hospitalizations due to respiratory syncytial virus was reinforced after a phase 3b clinical trial, demonstrating an 83% reduction in infant hospitalizations.

With the recent FDA-approved indication for children aged 2.5 years and older, somapacitan-beco is the first and only once-weekly growth hormone (GH) for children and adults.

According to Pfizer, the company’s pneumococcal conjugate vaccine Prevnar 20 has been approved by the FDA to treat infants and children aged 6 weeks to 17 years for the prevention of invasive pneumococcal disease (IPD).

According to a phase 2 study, remission of macrophage activation syndrome (MAS) secondary to systemic juvenile idiopathic arthritis (sJIA) or adult-onset Still’s disease (AOSD) was achieved in patients taking emapalumab who failed high-dose glucocorticoids.

Ritlecitinib could be a suitable treatment option for alopecia areata patients aged 12 years and older following positive results from a recent trial.

Roflumilast is a once-daily, topical phosphodiesterase type 4 (PDE4) inhibitor under development for treatment of inflammatory dermatoses, focusing on the hair-bearing areas including the face, scalp, and trunk.

Following voluntary market withdrawal from Covis Pharma, the FDA has decided to withdraw the approval of Makena and its generics for reducing the risk of preterm birth.

Data suggest confirmatory trials conducted for drugs granted accelerated approval by the FDA have taken a median 3.5 years to complete since 2012.

Leniolisib is the first and only treatment approved in the United States for APDS, a rare primary immunodeficiency.

Pediatric patients with BRAF V600E low-grade glioma as young as 1 year can now be treated with dabrafenib plus trametinib following its FDA approval.

Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor indicated for patients as young at 5 years to control high levels of low-density lipoprotein cholesterol (LDL-C) as a result of HoFH.

A recent study indicated that lebrikizumab treatment for 16 weeks was effective for adolescents and adults with moderate-to-severe atopic dermatitis.

Results from a recent phase 3 clinical trial demonstrated topline safety and efficacy results for tapinarof cream, 1% (VTAMA; Dermavant) as a potential treatment for atopic dermatitis in patients 2 years and older.

If approved by the FDA, this 5-in-1 investigational candidate could provide the broadest meningococcal serogroup coverage.