A Quarterly Review of NDAs – January - March 2025

This quarterly review on New Drug Applications (NDAs) contains data for applications approved for the first time during the first quarter of 2025, which includes New Molecular Entities (NMEs) and new biologics. A total of 38 applications were approved by the FDA during these three months. Based on submission classification, they can be divided as – Type 1 (New molecular entity): 18.4%, Type 2 (New active ingredient): 2.6%, Type 3 (New dosage form): 15.8%, Type 4 (New combination): 5.3%, Type 5 (New formulation or manufacturer): 21.1%, Biologics License Agreement (BLA): 34.2% and medical gases: 2.6%. Clearly, the focus was on developing new biological products.

The approvals in the first quarter of 2025 were divided into seven different dosage forms. Following are the percentages of those dosage forms – 2.6% oral solution, 21.1% tablets, 55.3% injectables, 2.6% gas inhalation products, 7.9% oral suspensions, 2.6% intrauterine and 7.9% capsules. All the BLAs were injectable products.

FDA approved Symbravo for the acute treatment of migraine headaches in adults, with or without aura. It combines two active ingredients - Meloxicam and Rizatriptan. It’s not intended for migraine prevention.

Ctexli by Mirum Pharmaceuticals Inc. is approved by the FDA for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Ctexli is the first FDA-approved drug to treat CTX, a very rare lipid storage disease. CTX is a genetic metabolic disorder caused by a mutation in a gene called CYP27A1 resulting in a deficiency of the enzyme that is important in the body’s ability to break down fats.

Sebela Women's Health Inc. received approval for MIUDELLA® (copper intrauterine system) for the prevention of pregnancy for up to three years. MIUDELLA is a next-generation, hormone-free, low-dose copper intrauterine device or IUD, the first to be approved by the FDA in the United States in over 40 years.

Blujepa (gepotidacin) is a new class of antibiotics called triazaacenaphthylene, which has a lower potential for antibiotic resistance. It works by targeting and blocking two essential bacterial enzymes, DNA gyrase and topoisomerase IV, which are needed for bacterial replication. It is used in women to treat an uncomplicated urinary tract infection affecting the lower urinary tract. Gomekli (mirdametinib) is used to treat neurofibromatosis type 1 (NF 1). It is from the drug class MEK inhibitors. NF 1 is a rare genetic disorder due to a mutation in the NF 1 gene. NF 1 patients have tumor growth along the peripheral nerve sheath, which cause disfigurement and functional impairment.

Qfitlia (fitusiran) is the first therapy in the US to treat hemophilia A or B. It has a unique mechanism to help reduce the frequency of bleeding episodes for people with hemophilia.

Author Details

Neelam Sharma, Lavanya Kundurthy and Hemant N. Joshi, PhD, MBA, Tara Innovations LLC, [email protected]

Publication Details