Drug Pipeline News

The once daily extended-release oral suspension with nighttime dosing is approved to treat ADHD as a monotherapy or as an adjunctive therapy to approved CNS stimulant medications.

Approximately 40% of patients who receive PN in the United States as an intravenous source of nourishment are under 18 years of age.

The federal agency has accepted the sBLA for Priority Review designation, with a target action date of September 15, 2024.

The extended label approval is based on full evidence from the XTEND-Kids study.

Data from the ongoing phase 1/2 CHORD trial was presented at the ASGCT annual conference.

The new data, published in The Lancet, showed that nirsevimab substantially reduced RSV hospitalizations.

The resubmission was announced in a first quarter, 2024 earnings news release from Lilly, which expects "regulatory action in the second half of 2024."

The rare genetic disease causes the body's immune system to not function properly.

A decision from the federal agency is expected in the fourth quarter of 2024.

With the approval comes the immediate availability of 5 mg, 7.5 mg, 10 mg, 12.5 mg, and 15 mg doses for patients between 2 to 5 years of age.

This decision from the federal agency marks the first FDA approval of a systemic therapy to treat patients with pediatric LGG with BRAF rearrangements, including fusions.

The biosimilar to ustekinumab is approved for patients 6 years and up, and is expected to be marketed on or after February 21, 2025.

Abeona's pz-cel is up for indicated use to treat patients with recessive dystrophic epidermolysis bullosa.

The 5-in-1 vaccine candidate has the potential to reduce shots and simplify the immunization process.

Juanita Mora, MD, breaks down the FDA approval of benralizumab as an add-on maintenance therapy among patients with severe asthma aged 6 to 11 years.

The treatment is approved for adolescents aged 12 years and older.

The pediatric approval to treat CABP is 1 of 3 indications approved by the FDA.

The phase 3 portion of the AMPLITUDE trial will include adolescents aged 10 to 17 years.

The planned phase 1/2 INSPIRE Duchenne trial (NCT06138639) is a first in-human, open-label, multicenter trial to evaluate tolerability and safety of SGT-003.

If approved, govorestat would be the first medication indicated for the treatment of galactosemia, a rare genetic metabolic disease resulting in an inability to metabolize simple sugar galactose.

Tenofovir alafenamide was approved in 2022 to treat pediatric patients aged 12 years and older.

IXINITY is now approved to treat all patients with the hemophilia B to help control bleeding episodes, as well as reduce the frequency of them.

The FTD will allow the development and review timeline of nipocalimab to accelerate. It is granted to drugs with the potential to treat severe conditions and provide unmet vital medical needs.

TNX-2900 was previously granted Orphan Drug designation by the federal agency in 2022 for the treatment of PWS.

The initial dose of pemivibart is 4500 mg, which is administered as a single intravenous infusion. A repeat 4500 mg dose should be administered every 3 months if ongoing protection is needed, stated the FDA.

The decision makes givinostat the first nonsteroidal drug approved to treat patients with all genetic variants of DMD.

Administration of the HIV-1 specific, non-nucleoside reverse transcriptase inhibitor (NNRTI) to the indicated population can be done via 25 mg oral tablets or new 2.5 mg oral tablets, developed to help administration and weight-adjusted dosing for children.

The decision makes atidarsagene autotemcel the first FDA-approved gene therapy for MLD, a rare genetic disease that affects the brain and nervous system.

The approval is based on data from a phase 3, multicenter, randomized trial that evaluated patients aged 8 to 17 years with HeFH who had LDL-C levels of 130 mg / dL or greater who were being treated with lipid-lowering medications.