Your source for the latest industry trends and drug information news.
Volume 7 | Issue 16
August 15, 2025
Contributors:
Chief Authors: Rebecca Waite, PharmD & Christina Ramsay, PharmD
Contributing Authors: Xintian Wu, PharmD/MBA Candidate, and Jessica Lin PharmD/MBA Candidate
On July 28, 2025, the U.S. Food and Drug Administration (FDA) approved Sephience™ (sepiapterin) for the treatment of hyperphenylalaninemia in adult and pediatric patients 1 month and older with sepiapterin-responsive phenylketonuria (PKU). PKU is a rare genetic disorder that causes a dangerous buildup of phenylalanine (an amino acid found in many foods, like meat, eggs, dairy, nuts and grains) in the blood. High levels of phenylalanine can be toxic and lead to seizures and other serious health problems, including brain disorders and behavioral disabilities. The primary treatment of PKU is following a strict diet low in phenylalanine. However, in some cases additional pharmacologic intervention is needed to lower blood phenylalanine levels. There are currently three FDA-approved medications for treatment of PKU — Kuvan® (sapropterin dihydrochloride), Palynziq® (pegvaliase) and Sephience™ (sepiapterin).
FDA approval of Sephience™ is based on the results of the Phase 3 APHENITY trial, which showed two-thirds of study participants had at least a 30% reduction in their blood phenylalanine levels. Sephience™ dose is based on age and body weight and is taken once daily with food and used in conjunction with a phenylalanine restricted diet. The powder packets can be mixed with water or apple juice to create a liquid mixture, or doses greater than 1,000mg may also be mixed with strawberry jam or applesauce to create a soft food mixture per the manufacturer's package instructions. Warnings and precautions for Sephience™ include increased risk of bleeding and risk of a drug-drug interaction with levodopa, a medication used for the treatment of Parkinson’s disease. Based on clinical findings, three patients taking levodopa and Sephience™ experienced neurological changes, including seizures. Additional drug interaction precautions include avoiding taking Sephience™ with certain types of other drugs like methotrexate and sulfasalazine. Side effects of Sephience™ include diarrhea, headache and hypophenylalaninemia (low levels of phenylalanine). Continued monitoring of blood phenylalanine levels along with consistent diet can help minimize the risk of hypophenylalaninemia.
On July 31, 2025, the FDA released a drug safety communication announcing that drug manufacturers of prescription opioid medications must make required updates to their medication’s package labeling in the next 30 days. This announcement comes after the FDA held a joint meeting with the Drug Safety and Risk Management Advisory Committee and the Anesthetic and Analgesic Drug Products Advisory Committee in May 2025 for discussion and expert analyses of two long-term opioid observational studies. The groups reviewed data from the studies regarding the risks of long-term opioid use, including potential for abuse, addiction, misuse and overdose.
The required updates to the drug package labeling include removing the phrase “extended treatment period” from the Indications and Usage section of the labeling. While there is no clearly defined number of days to determine “extended use,” there is a lack of clinical safety and efficacy data to support continued use of opioid medications over an indefinite time. In fact, the risks of opioids can increase when used for more than just a few days. The package labeling must also highlight that higher doses of opioids are associated with greater risks and that these risks persist over the entire course of treatment. In addition, there are updates required to clarify that extended-release opioid medications should be considered only when other types of pain medications, such as ibuprofen and acetaminophen (Tylenol), as well as immediate-release opioids, have been tried and failed, when clinically appropriate. Guidance that stopping or discontinuing opioids should be done gradually because opioids can cause physical dependence and abrupt discontinuation can lead to symptoms of withdrawal (such as nausea, vomiting, shaking, sweating and feeling anxious), along with information on opioid overdose reversal agents like Narcan (naloxone), is also required as part of the update. The goal of updating package labeling to include expanded safety information for all prescription opioid medications is to help educate health care providers when prescribing and patients who may be prescribed these medications about the serious risks associated with opioids and to better understand and evaluate the benefits of therapy compared to these risks.
On July 28, 2025, the Centers for Medicare & Medicaid Services (CMS) released Medicare Part D bid information (detailed instructions from CMS to plan sponsors outlining the requirements for them to submit their bids for approval by CMS to provide Part D drug coverage) for the 2026 plan year in preparation for the Medicare Open Enrollment period, which begins on October 15, 2025. Medicare Part D provides beneficiaries with coverage which helps pay for their prescription drugs. Private health insurance companies can contract with CMS to offer prescription drug plans (PDPs) or offer Medicare Advantage (MA) plans that include prescription drug coverage (MA-PDs). Before being modified through legislation in the Inflation Reduction Act (IRA), introduced in 2022, the Part D benefit included four phases (deductible phase, initial coverage phase, coverage gap also known as the “donut hole,” catastrophic phase). The IRA changed the Part D benefit phases by getting rid of the coverage gap phase and lowering the cost share amounts previously required by the Part D beneficiary and the amount paid by Medicare in the catastrophic phase, thereby shifting these additional prescription drug costs to the plan sponsor. In an effort to minimize the amount of the increased drug costs that are passed to the beneficiaries in the form of increased monthly premiums (monthly payments made by beneficiaries who are enrolled in Part D plans), the IRA included a cap of 6% to the base beneficiary premium (BBP).
Last year, in July 2024, as part of the annual CMS Part D bid information process, CMS announced a new nationwide voluntary three-year Part D Premium Stabilization Demonstration for PDPs (including Employer Group Waiver Plans or EGWP plans) which began in 2025. The goal of the program is to help stabilize Part D premiums as plans adjust to the changing environment and new legislation. For 2025, the three components of the program included a $15 reduction to the BBP, year-over-year premiums capped at $35, and narrowed risk corridors, which are cost ranges designed to share financial risk between Medicare and the plan sponsor. For 2026, the reduction to the BBP is being lowered to $10, year-over-year premiums are capped at $50, and CMS is discontinuing the narrowed risk corridors.
New Drug: Anzupgo® (delgocitinib)
Topical cream approved for the treatment of moderate to severe chronic hand eczema in adults when topical corticosteroids are not clinically appropriate or have been tried and failed. [7/23/2025 – LEO Pharma, Inc.]
Dificid® (fidaxomicin)
Indication: For the treatment of diarrhea caused by C. difficile in adult and pediatric patients 6 months and older who can swallow tablets and weigh at least 28 pounds.
Dosage Form/Strength: 200mg tablet
Average Wholesale Price (AWP): Generic = $5,940 | Brand = $6,240
