Pulse Newsletter | Volume 7 - Issue 24

FDA Approves Drug to Treat Adults with Relapsed or Refractory Acute Myeloid Leukemia

On November 13, 2025, the U.S. Food and Drug Administration (FDA) approved Komzifti™ (ziftomenib) capsules for the treatment of adult patients with relapsed (cancer that returned after being treated) or refractory (cancer that did not respond to treatment) acute myeloid leukemia (AML) with a specific type of mutation for which other treatment options are not available.  AML is a type of blood cancer that affects bone marrow. Komzifti™ is supplied as a 200mg capsule and should be stored at room temperature. The recommended dose of Komzifti™ is 600mg (three 200mg capsules) taken by mouth once daily on an empty stomach. Warnings and precautions of Komzifti™ include risk of harm to an unborn baby and QTc prolongation, which causes the heart rhythm to become abnormal and can be life-threatening. The most common side effects associated with therapy include diarrhea, nausea, fatigue, muscle pain, damage to blood vessels, swelling, infection, itching, fever and differentiation syndrome, which is a severe reaction to therapy that causes life-threatening symptoms including fever, swelling, shortness of breath and kidney damage.

The safety and efficacy of Komzifti™ was determined from a single-arm, open-label phase 2 clinical trial. The primary clinical endpoint was complete remission, which was achieved in 21% of patients. Differentiation syndrome occurred in 29 out of 112 patients in the clinical trial, with 2 cases causing a fatal adverse reaction. The manufacturer recommends that patients taking Komzifti™ avoid taking antacids, such as H2 receptor blockers like ranitidine and famotidine, as well as proton pump inhibitors (PPIs) like omeprazole and pantoprazole. The cost of Komzifti™ is $48,500 for a one-month supply.

FDA Approves Reformulated Ranitidine in the U.S. Following Safety Review

On November 24, 2025, the FDA announced that the generic drug, ranitidine, which was previously sold over the counter and by prescription and is indicated for the treatment of acid reflux, will be returning to the U.S. market after an extensive safety review. Ranitidine (Zantac®) is a histamine type 2-receptor antagonist (H2RA), which works by decreasing the amount of acid produced by the stomach. Ranitidine was ordered by the FDA to be taken off the market in April 2020 due to the detection of a probable human carcinogenic (cancer-causing) substance, N-nitrosodimethylamine (NDMA), that was found to increase in the drug product over time. Patients in the U.S. were advised to discontinue taking ranitidine and speak to their health care professional about clinical alternatives for the treatment of acid reflux, such as famotidine (Pepcid®) or omeprazole (Prilosec®), which are also available over the counter and by prescription.

The FDA advised patients that the newly reformulated ranitidine will have the same clinical efficacy as before and will be safe for patient use. Updated product labeling will include recommendations for storage and handling. These include keeping ranitidine in the original bottle in a cool, dry place to protect from heat and moisture. Bottles should be kept closed tightly, and it is recommended that any unused tablets be discarded within 90 days after opening the bottle, or by the product’s expiration date, if that is sooner. An exact date for when ranitidine tablets will be available to patients in the U.S. has not yet been announced.

Medicare Announces Price Reductions for 15 Part D Drugs That Will Take Effect 1/1/2027

On November 25, 2025, the Centers for Medicare & Medicaid Services (CMS) announced an expected net savings of $12 billion annually because of the government’s negotiations with pharmaceutical companies for maximum fair prices (MFP) for 15 additional drugs (Ozempic®/Wegovy®/Rybelsus®, Trelegy Ellipta, Xtandi®, Pomalyst®, Ofev®, Ibrance®, Linzess®, Calquence®, Austedo®/Austedo XR®, Breo Ellipta, Xifaxan®, Vraylar®, Tradjenta®, Janumet®/Janumet® XR, Otezla®/ Otezla XR™). These drugs are used to treat a variety of disease states, including diabetes, chronic obstructive pulmonary disease (COPD), irritable bowel disease and cancer. The MFPs for the 15 drugs will be effective on January 1, 2027. This is in addition to the 10 original MFP drugs (Eliquis®, Xarelto®, Jardiance®, Januvia®, Farxiga®, Entresto®, Enbrel®, Imbruvica®, Stelara®, Novolog®/Fiasp®) which are due to take effect on January 1, 2026.

The Inflation Reduction Act (IRA), which was signed into law in 2022, is intended to lower drug costs for seniors and help sustain Medicare benefits over time by controlling rising drug costs. The original 10 MFP drugs are required to be covered on Medicare Part D formularies for the duration of the year in 2026. A Part D formulary is a list of covered prescription drugs organized into formulary tiers that determine the cost share paid by the Medicare beneficiary. The intent is to keep negotiating additional MFP drugs, with another round of 15 drugs that will be selected for 2028 and 20 additional drugs for 2029. The federal government is also utilizing an executive order intended to allow the U.S. “most favored nation (MFN)” drug pricing, which sets benchmark drug prices based on the prices other similar developed countries, such as the United Kingdom and Canada, pay for prescription drugs. 

New FDA Approvals

New Drug: Hyrnuo® (sevabertinib)

Oral tablets approved for the treatment of adult patients with a specific type of locally advanced or metastatic non-squamous non-small cell lung cancer who received prior systemic therapy. [11/19/2025 – Bayer HealthCare Pharmaceuticals, Inc.]

New Generics Entering the Marketplace

Mavenclad® (cladribine)

Indication: For treatment of relapsing forms of multiple sclerosis (MS) in adult patients.

Dosage Form/Strength: 10mg tablet

Average Wholesale Price (AWP): Generic = $12,600/tablet | Brand = $13,300/tablet