Drug Pipeline News

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.

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.

Understand how to naviate the current shortage of pediatric ADHD medications.

The federal agency approved Perflutren Lipid Microsphere following positive data from 3 pediatric clinical trials.

A poster presentation at AAAAI from the phase 3 PEOPLE Study revealed DBV712 treatment in children with peanut allergy has a favorable safety and tolerability profile.

The Rare Pediatric Disease Designation joins the Fast Track and Orphan Designations that have already been granted to AOC 1044.

A Prescription Drug User Fee Act (PDUFA) target action date of June 21, 2024, has been set by the FDA.

ARS Pharma plans to submit data from the repeat dose study of neffy to the FDA early in the second quarter of 2024, as part of its response to the CRL issued by the federal agency for the previously submitted New Drug Application (NDA).

The approval makes omalizumab the first and only FDA-approved medicine to reduce allergic reactions in patients with one or more food allergies.

The Orphan Drug Designation follows the receipt of Rare Pediatric Disease Designation from the FDA in January 2024.