This quarterly review of New Drug Applications contains data for applications approved during the third quarter of 2024, which includes New Molecular Entities (NMEs) and new biologics. A total of 39 applications were approved by the FDA during these three months.
The NDAs approved during the third quarter of 2024 have been divided into seven different dosage forms. The following are the percentages – injection (51.3%), tablets (17.9%), capsules (10.3%), gas (5.1%), and 2.6% each of oral films, oral solution, oral suspension, oral pellets, nasal spray, and inhalation powder. Recently, there has been more focus on injectable formulations. Following are the submission classifications: BLA (27.5%), Type 1 (new molecular entity, 22.5%), Type 2 (new active ingredient, 2.5%), Type 3 (new dosage form, 22.5%), Type 5 (new formulation or manufacturer, 17.5%), Type 9 (new indication submitted as distinct NDA, consolidated with original NDA upon approval, 2.5% and gas (5.0%).
Eli Lilly’s antibody therapy donanemab, now Kisunla, is approved for certain adults with Alzheimer’s disease. Kisunla is indicated for patients with early symptomatic Alzheimer’s, and includes individuals with mild cognitive impairment or mild dementia, and confirmed evidence of amyloid plaques, the toxic protein aggregates, or clumps, that accumulate in the brain of those with the neurodegenerative disease.
Voquezna (vonoprazan), by Phathom Pharmaceuticals, is approved by the FDA to treat heartburn associated with the most common type of gastroesophageal reflux (GERD) in adults. Voquezna is a potassium-competitive acid blocker (PCAB) that binds to the potassium channel of the H+/K+ ATPase enzyme and inactivates it.
Aqneursa by Intrabio is the only FDA-approved stand-alone therapy for the treatment of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. Niemann-Pick disease is a group of rare conditions passed down in families. The conditions affect the body's ability to break down and use fats, such as cholesterol and lipids, inside cells. Because of the buildup of fats, these cells don't work as they should, and, over time, the cells die. Niemann-Pick disease can affect the brain, nerves, liver, spleen, and bone marrow.
On September 26, the FDA approved Cobenfy -the first new schizophrenia drug in decades, a muscarinic agonist. Cobenfy (xanomeline and trospium chloride) is not a dopamine agonist or antagonist but it has a different mechanism. The problem with developing a drug to activate them in the brain is that they can trigger receptors in the gastrointestinal tract. Patients couldn’t tolerate it. Trospium was already used for overactive bladder and was added to shut down the gastrointestinal receptors. It can’t cross into the brain from the blood. That means it shuts down the muscarinic receptors in the body but doesn’t stop the first drug from doing its job in the brain.
In the same month, the FDA also approved Flyrcado™ (flurpiridaz F 18) injection. Flyrcado delivers higher diagnostic efficacy compared to single-photon emission computed tomography (SPECT) MPI. It is the first of its kind positron emission tomography myocardial perfusion imaging (PET MPI) agent, for the detection of coronary artery disease (CAD). Flyrcado can be manufactured in an offsite pharmacy and delivered as a ready-to-use unit dose. Flyrcado removes the need for on-site tracer production and generator maintenance because of its half-life (109 minutes), which is significantly longer than existing PET MPI tracers.
Author Details
Neelam Sharma MS; Lavanya Kundurthy BE; and Hemant N. Joshi, Ph.D., MBA# - Tara Innovations LLC, # [email protected], www.tara-marketing.com
Publication Details
This article appeared in Pharmaceutical Outsourcing:Vol. 25, No.4 Oct/Nov/Dec 2024Pages: 33-35