Drug Pipeline News

MNV-201 is being evaluated in phase 2 clinical trials for treatment of the ultra-rare, life-threatening mitochondrial disorder affecting children.

Roflumilast cream 0.05% is being studied for mild-to-moderate AD in infants aged 3 months to under 2 years in a new phase 2 trial.

The FDA has approved belimumab (Benlysta; GSK) autoinjector for children with lupus nephritis, expanding at-home treatment.

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The FDA has extended the review of ruxolitinib for atopic dermatitis in children 2 to 11 years to September 2025 to evaluate new CMC data.

Lenacapavir is the first twice-yearly option available in the US for those who need or want PrEP, according to Gilead Sciences.

Currently, omaveloxolone (Skyclarys) is approved to treat adults and adolescents aged 16 years and older impacted by FA.

The federal agency has assigned a target action date of January 31, 2026, for a decision.

Garadacimab-gxii is the only treatment to target factor Xlla for prophylactic use to prevent attacks of HAE in patients aged 12 years and older.

Though the decision will not meet the June 17, 2025, target date, the federal agency anticipates a decision within approximately 4 weeks.

The approval for ustekinumab-stba now offers all dosage forms to the reference product, ustekinumab, and is indicated for patients aged 6 to 17 years.

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Currently, Vimkunya is FDA-approved for use among individuals aged 12 years and older.

Glecaprevir/pibrentasvir was a "highly efficacious treatment for people with acute HCV," with 96.2% of patients analyzed having sustained virological response 12 weeks post-treatment.

The phase 2 trial will be 4 weeks and evaluate investigational, once-daily roflumilast cream 0.05% in infants 3 months to less than 2 years of age.

The BLA for clesrovimab was based on results demonstrated in the phase 2b/3 CLEVER trial among healthy pre-term and full-term infants.

Results at 24 weeks revealed that 76% of individuals achieved a 75% or more improvement in overall disease severity (EASI-75), trial's primary endpoint.

The combination therapy yielded boosted treatment benefits of navepegritide, including improvement in mean ACH Z-score and mean annualized growth.

Six-month results from the phase 2 VLA1553-221 trial aligned with initial data and demonstrated immune response at day 180 among patients aged 1 to 11 years.

The AI-enabled, wearable device is the first FDA-approved, drug-free solution for nasal congestion and allergies in children ages 12 and up.

With the new drug application accepted with priority review, the FDA has assigned a date of November 20, 2025, to complete its review for potential approval.

The hyperpolarized contrast agent for oral inhalation can now be used in patients 6 years and older, lowering the approved indication from 12 years of age.

The decision makes hydrocortisone oral solution (Khindivi; Eton Pharmaceuticals) the only FDA-approved oral formulation of hydrocortisone.

Review some of the reactions from experts about the recent FDA approval of roflumilast foam 0.3% to treat scalp and body psoriasis in patients aged 12 years or older.

The approved indication now includes children down to 6 weeks of age to protect against invasive meningococcal disease via serogroups A, C, W, and Y.

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The once-daily, steroid-free topical is now approved to treat plaque psoriasis of the scalp and body in patients aged 12 years and older.

The expanded indication follows original approval in August 2018 for use in previously-treated patients 12 years and older.

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NVX-CoV2705 is indicated for individuals aged 12 to 64 years with an underlying condition that poses high risk for severe COVID-19 outcomes.