Your source for the latest industry trends and drug information news.
Volume 7 | Issue 17
September 1, 2025
Contributors:
Chief Authors: Rebecca Waite, PharmD & Christina Ramsay, PharmD
Contributing Authors: Xintian Wu, PharmD/MBA Candidate, and Jessica Lin PharmD/MBA Candidate
On August 12, 2025, the U.S. Food and Drug Administration (FDA) approved Brinsupri™ (brensocatib) tablets for the treatment of patients 12 and older with non-cystic fibrosis bronchiectasis (NCFB), a chronic inflammatory lung disease that leads to excessive mucus production, widened and damaged airways and persistent cough. NCFB puts affected patients at an increased risk of serious lung infections. Bronchiectasis may develop in patients with cystic fibrosis, which is a rare genetic disorder affecting approximately 40,000 patients in the United States, that causes a thickening of mucus in the lungs and digestive system and can lead to permanent lung damage if left untreated. NCFB, which is more common and affects about 500,000 patients in the United States, can be caused by other chronic respiratory and inflammatory diseases, like chronic obstructive pulmonary disease (COPD), as well as history of previous lung infections, or other inflammatory autoimmune conditions, like inflammatory bowel disease. Standard management of NCFB has been limited to treatment of any underlying lung infection and disease flares with antibiotics and supplemental oxygen.
Brinsupri™ is the first and only FDA-approved drug therapy available for NCFB. The medication is available as an oral tablet that is taken once daily by mouth with or without food. Brinsupri™ works by reducing the body’s inflammatory response, specifically targeting neutrophils, a type of white blood cell in the body that work as part of the immune system to fight infection. It was approved based on the results of two clinical trials: a 52-week Phase 3 randomized, double-blind, placebo-controlled trial called ASPEN (1,721 patients), and a 24-week Phase 2 double-blind, placebo-controlled trial called WILLOW (256 patients). In the ASPEN clinical trial, patients taking Brinsupri™ had a 20% reduction in flares compared to patients taking placebo. Other clinical findings of the studies included more time without flares and less decline in lung function. Warnings and precautions for Brinsupri™ recommend monitoring for adverse skin and dental reactions and avoidance of live vaccines. Side effects observed during clinical trials with Brinsupri™ treatment included upper respiratory tract infection, headache, rash, dry skin, thickening of skin and increases in blood pressure. Brinsupri™ will be available through a limited distribution network of specialty pharmacies.
On August 5, 2025, the FDA approved Ajovy® (fremanezumab-vfrm) for the preventative treatment of episodic migraine (defined as having less than 15 migraine headache days per month) in pediatric patients aged 6 to 17 years old who weigh at least 99 pounds. According to the National Institutes of Health, approximately 10% of children experience migraines. Migraines can cause pain, nausea, vomiting and sensitivity to light and noise. Genetic predisposition appears to play a role in pediatric migraines, along with certain chemicals in the brain, however, the exact cause of recurrent migraines in pediatric patients is still unknown. Migraines can occur randomly in children or may be brought on by triggers, like stress, certain foods and lack of sleep. Preventative strategies for pediatric migraine include lifestyle modifications to help ensure that children have a healthy diet, engage in exercise throughout the day, get enough sleep and manage their stress levels.
Ajovy® was originally approved by the FDA in 2018 for use in adult patients for the prevention of migraine. Ajovy® is administered as a dose of 225mg injected once a month subcutaneously (under the skin) in the stomach, thigh or upper arm. Ajovy® can be self-administered in patients 13 years and older. Per the medication’s package labeling, patients ages 6 to 12 must have Ajovy® administered by their health care provider or adult caregiver only. Ajovy® is a type of medication called a calcitonin gene-related peptide antagonist (CGRP). Other available CGRPs include Aimovig, Emgality®, Nurtec®, Ubrelvy®, and Qulipta®. Ajovy® is the only CGRP currently FDA-approved for use in pediatric patients for episodic migraine headaches.
Ajovy® was evaluated for safety and efficacy in a 12-week, multi-center, randomized, double-blind, placebo-controlled clinical trial called the SPACE trial of 225 pediatric patients 6 to 17 years old with episodic migraine. Based on the results of the SPACE trial, Ajovy® showed a greater reduction in the number of migraine headache days per month (2.5 less migraine headache days per month) compared to placebo (1.4 less migraine headache days per month). In pediatric patients weighing less than 99 pounds, the SPACE trial did not conclude an appropriate dosage that was safe and effective. In addition, the safety of Ajovy® has not been established in pediatric patients less than 6 years old or in the treatment of pediatric patients diagnosed with chronic migraine headaches (defined as having 15 or more headache days per month). Based on the results of the SPACE trial, the safety profile of Ajovy® in pediatric patients was observed to be similar to that of adult patients. Warnings and precautions of Ajovy® use include injection site reactions, high blood pressure and increased risk for Raynaud’s syndrome, which is a condition causing the small blood vessels in the fingers and toes to narrow and restrict blood flow.
On August 7, 2025, the FDA announced a new program called FDA PreCheck. The program is designed to make it easier for pharmaceutical companies to build their manufacturing facilities and produce more prescription drugs in the United States rather than overseas in other countries. According to the FDA, the U.S. currently manufactures less than half of all pharmaceutical drugs, including both prescription and over-the-counter medicines, that are distributed in the country. In addition, the FDA indicates U.S. based manufacturers represent only a minority (about 11%) of all manufacturers of active pharmaceutical ingredients (or APIs) used in FDA-approved medications. Prescription and over-the-counter drugs contain both active and inactive ingredients. APIs are the component of a drug responsible for its therapeutic effect, while the other inactive ingredients may be used for things like absorption, stability, binding or drug delivery in the body.
In May 2025, President Donald J. Trump issued an Executive Order called “Regulatory Relief to Promote Domestic Production of Critical Medicines”. This Executive Order is intended to streamline regulatory requirements for pharmaceutical companies to facilitate efficiency and reduce barriers in the FDA review and approval process for new U.S.-based drug manufacturing sites. According to the federal government, the current timeline for a pharmaceutical company to successfully build and receive FDA approval for a new manufacturing facility in the U.S. is 5-10 years. The PreCheck program has measures in place to expedite this timeline.
The PreCheck program has two phases. The first phase is called the “Facility Readiness Phase”, which is designed to help pharmaceutical companies communicate more often with the FDA during the early stages of planning and production, including facility design and construction details to ensure compliance with FDA standards. The second phase, called the “Application Submission Phase”, is designed to streamline the approval process at later stages of development, including ensuring that standards for drug production and quality assurance requirements are met, through additional communication, feedback and meetings with the FDA. The new FDA PreCheck program, along with the Trump administration’s implementation of international tariffs (taxes placed on imported goods, including prescription drugs), is designed to boost production of pharmaceutical drugs in the U.S. Several large pharmaceutical companies, including Johnson & Johnson, AstraZeneca, Roche and Eli Lilly have already announced plans to invest billions of dollars over the next few years into U.S. based manufacturing facilities.
New Drug: Hernexeos® (zongertinib)
Oral tablets approved for the treatment of adults with non-squamous non-small cell lung cancer (NSCLC) that is unresectable (cannot be removed by surgery) or metastatic (spread to other parts of the body) and has a specific genetic mutation, and who have already received prior systemic treatment [8/8/2025 – Boehringer Ingelheim Pharmaceuticals, Inc.]
Vuity® (pilocarpine hydrochloride)
Indication: For the treatment of presbyopia (a gradual loss of close-up vision associated with normal aging) in adults.
Dosage Form/Strength: 1.25% ophthalmic solution
Average Wholesale Price (AWP): Generic = $135/5mL bottle | Brand = $140/5mL bottle
