Drug Pipeline News

The application indication is for adults and pediatric patients aged 12 years and older with CSU whose disease is not adequately controlled with H1 antihistamines.

With the decision, Kebilidi becomes the first FDA-approved gene therapy for AADC deficiency, and is indicated for adult and pediatric patients.

A target action date for potential approval of prademagene zamikeracel (pz-cel) has been set for April 29, 2025.

Data stems from the first 4 patients dosed in a low-dose cohort of an ongoing phase 1/2 open-label trial.

The initiation of a phase 2 study of the investigational 31-valent pneumococcal conjugate vaccine is expected by the end of January 2025.

The federal agency has not raised any concerns regarding safety and efficacy of tapinarof cream, 1%.

Application acceptance was based on positive results with monthly and bi-monthly dosing data from a phase 3 trial.

The approval makes methotrexate, currently, the only only oral liquid methotrexate on the market approved for both adult and pediatric indications.

The fibrin sealant solution is now available in 18 countries.

Look back at several quick video interviews discussing the expanding role of artificial intelligence, how RSV treatment has evolved, and much more.

The TrustTSC trial did not achieve its primary endpoint of percent reduction in 28-day frequency of TSC-associated seizures.

Interim analysis of the phase 3 REGAL Trial is anticipated in this quarter of 2024.

There were no treatment or RSV-related deaths during the study.

The approved indication is for patients aged 7 years and up with narcolepsy to treat cataplexy or excessive daytime sleepiness.

Approval is based on a study in 116 adult and pediatric male patients with either severe hemophilia A or severe hemophilia B, both without inhibitors.

This decision marks the second time the FDA has issued a complete response letter (CRL) for dasiglucagon to treat hypoglycemia in patients 7 days and up with congenital hyperinsulinism.

On average, participants treated with levacetylleucine for 12 weeks had better fSARA scores compared to when they received a placebo.

The needle-free option to treat type 1 allergic reactions, including anaphylaxis, was approved for adults and children who weigh 66 lbs or more on August 9, 2024.

With this acceptance, the federal agency has assigned a PDUFA date of May 22, 2025 for roflumilast foam (Zoryve) 0.3%.

With this approval, FluMist becomes the first vaccine to prevent influenza that does not need to be administered by a health care provider.

The approval is indicated for adults and children aged 2 years and older, making arimoclomol (Miplyffa) the first drug approved by the FDA to treat NPC.

Infigratinib has also received Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation from the FDA.

Get caught up with our journal! Review some of the top stories from the Contemporary Pediatrics website over the last week, and catch up on anything you may have missed.

The landmark indication is supported by a couple of pivotal trials assessing dupilumab for patients with chronic rhinosinusitis with nasal polyps, as well as a pediatric severe asthma trial assessing the biologic.

Biogen's Phase 2/3 study showed a higher dose of nusinersen improves motor function in SMA infants, with plans for regulatory submission to advance treatment.

Availability for the type 1 allergic reactions treatment approved by the FDA in August is expected later this month.

The investigational MEK inhibitor has been assigned a Prescription Drug User Fee Act (PDUFA) date of February 28, 2025.

Efficacy of roflumilast cream 0.05% improved over time among children aged 2 to 5 years with mild to moderate atopic dermatitis.

Following the FDA's acceptance of the NDA, a PDUFA date of December 26, 2024 has been set for setmelanotide in patients as young as 2 years of age.

The federal agency advised manufacturers in June that 2024-2025 COVID-19 vaccines should be monovalent JN.1, with the preferred lineage being the KP.2 strain.