Pfizer to Retire Low-Dose Vyndaqel
On Aug. 29, 2025, Pfizer announced plans to discontinue Vyndaqel® (tafamidis meglumine 20mg), by the end of 2025. Approved in 2019 alongside high-dose Vyndamax® (tafamidis 61mg), both drugs treat transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and progressive heart condition. The decision aims to streamline treatment as Vyndamax offers the same clinical benefits in a single daily capsule compared with Vyndaqel’s four-capsule regimen. Vyndaqel currently accounts for less than 20% of tafamidis prescriptions in the US. For Vyndamax prescribing information, see here.
Leqembi Iqlik Approved for Alzheimer’s At-Home Injection
On Aug. 29, 2025, the US Food and Drug Administration (FDA) approved Biogen and Eisai’s Leqembi® Iqlik™ (lecanemab-irmb) 360mg/1.8mL subcutaneous (SC) autoinjector for maintenance dosing in early Alzheimer’s disease (AD), including patients with mild cognitive impairment or mild dementia. This is the first anti-amyloid therapy allowing for at-home administration, following an initial 18-month intravenous (IV) treatment phase at 10mg/kg every two weeks over one hour. Patients may transition to either monthly IV infusions at 10mg/kg or weekly 360mg SC injections via the autoinjector, which delivers the dose in approximately 15 seconds. The autoinjector is designed to reduce systemic reactions showing less than 1% versus 26% with IV infusions in studies. Coinciding with the approval, the FDA updated its safety guidance for amyloid-related imaging abnormalities with edema (ARIA-E), a known side effect involving brain swelling or fluid buildup. Previously, MRIs were recommended before the fifth, seventh, and fourteenth infusions, but the agency now advises imaging prior to the third infusion to enable earlier detection. Leqembi Iqlik is expected to be available on Oct. 6, 2025, at a cost of $19,500, which is slightly higher than the IV maintenance dose of approximately $13,316 (cost varies by patient weight). Eisai also plans to file for FDA approval of a 500mg SC initiation dose. Here is the prescribing information.
More Prolia and Xgeva Biosimilars
On Sept. 2, 2025, the FDA approved Henlius and Organon’s biosimilars Bildyos® (denosumab-nxxp, 60 mg/mL) and Bilprevada® (denosumab-nxxp, 120mg/1.7mL) as alternatives to Amgen’s Prolia® (denosumab) and Xgeva® (denosumab), respectively. Bildyos is indicated for the treatment of osteoporosis for those at high risk for fracture in certain populations like men, post-menopausal women and those taking glucocorticoids or certain drugs for prostate and breast cancer. It is administered by a healthcare provider as a 60mg SC injection once every six months. A boxed warning and a risk evaluation and mitigation strategy (REMS) caution that using Bildyos may raise the risk of dangerous hypocalcemia (low levels of calcium in the blood) for patients who have chronic kidney disease (CKD). Bilprevada is indicated to prevent skeletal-related events for some patients who have multiple myeloma, bone metastases from solid tumors, giant cell tumors of the bone or hypercalcemia of malignancy. After loading doses, it is administered SC at 120mg once every month by a healthcare provider. Jubbonti® (denosumab-bbdz – Sandoz), the first biosimilar to Prolia, and Wyost® (denosumab-bbdz – Sandoz), the first biosimilar to Xgeva, launched on May 31, 2025, and will hold interchangeability exclusivity until Oct. 29, 2025. Other biosimilars are expected to launch this year. Prescribing information can be found here for Bildyos and Bilprevada.
Risperdal Consta Gets First Generic
On Sept. 4, 2025, the FDA approved Amneal Pharmaceuticals’ generic to Johnson & Johnson Innovative Medicine’s Risperdal Consta® risperidone extended-release injectable suspension (12.5mg, 25mg, 37.5mg, and 50mg vials) for the treatment of schizophrenia and as maintenance therapy for bipolar I disorder, either alone or in combination with lithium or valproate. Approval was granted under the Competitive Generic Therapy (CGT) pathway, securing 180-day exclusivity. The product is expected to launch in the fourth quarter of 2025. US annual sales for the reference product totaled approximately $194 million for the 12 months ending July 2025.
Sobi Withdraws Synagis
On Aug. 12, 2025, Sobi announced that it will discontinue Synagis® (palivizumab) injection by the end of the year. Approved in 1998, Synagis requires monthly administration during the respiratory syncytial virus (RSV) season and was limited to select pediatric populations. In contrast, Beyfortus (nirsevimab, Sanofi/AstraZeneca) and Enflonsia™ (clesrovimab, Merck) are recommended for infants under eight months entering their first RSV season, with Beyfortus also approved for high-risk children aged 8–19 months entering their second season. Both offer broader coverage and longer-lasting protection with a single dose. The American Academy of Pediatrics (AAP) recently updated its guidance to no longer routinely recommend Synagis and is finalizing new recommendations incorporating Enflonsia.
Policy Updates
FDA Introduces Rare Disease Evidence Principles to Accelerate Ultra-Rare Drug Approvals
On Sept. 3, 2025, the FDA unveiled its Rare Disease Evidence Principles (RDEP) framework, a new regulatory pathway designed to expedite the approval of therapies for ultra-rare genetic diseases. RDEP allows single-arm trials to serve as pivotal evidence when supported by confirmatory data. This supporting evidence can include the drug’s activity at the target site and relevant preclinical findings that reinforce the observed treatment effect. To qualify, candidate therapies must target a known genetic defect, treat diseases affecting fewer than 1,000 individuals in the US, and address conditions with rapid functional decline and no adequate alternatives. Analysts’ views on the FDA’s new RDEP framework are divided. Some see it as a continuation of existing regulatory flexibility, offering limited practical change due to its narrow eligibility criteria. Others interpret it as a positive signal of the FDA’s commitment to supporting innovation in ultra-rare disease drug development. However, there are concerns that relying on mechanistic plausibility and limited data could weaken evidentiary standards and complicate reimbursement decisions.
Recall
Sterile Water and Saline Irrigation Recalled
On Aug. 26, 2025, B. Braun recalled Sterile Water for Injection and Sodium Chloride 0.9% Irrigation Solution due to the chance of fluid leaking from the port site. If product leakage is not visibly apparent and the product becomes contaminated with microorganisms, there is a risk that the final mixed product may also be contaminated. This could potentially result in a bloodstream infection requiring additional medical intervention. In certain patients, such infections may pose a life-threatening risk. Sterile Water for Injection is used as a diluent in the preparation of parenteral solutions and must be mixed with appropriate solutes before intravenous administration. Sodium Chloride 0.9% Irrigation Solution is used for general irrigation of body cavities, tissues, wounds and surgical instruments, but is not intended for injection. For more information about the recall, see here.
Cyclobenzaprine Recalled
On Aug. 27, 2025, Unichem Pharmaceuticals issued a recall for one lot of cyclobenzaprine hydrochloride 10mg tablets due to a labeling error in which bottles labeled as cyclobenzaprine 10mg actually contain meloxicam 7.5mg tablets. Cyclobenzaprine is a muscle relaxant indicated as an adjunct to rest and physical therapy for relief of muscle spasm associated with acute, painful musculoskeletal conditions, while meloxicam is a non-steroidal anti-inflammatory drug (NSAID) indicated for osteoarthritis, rheumatoid arthritis, and juvenile rheumatoid arthritis. Inadvertent administration of meloxicam may result in serious adverse events including cardiovascular, gastrointestinal, renal, anaphylactic, and dermatologic reactions, especially in patients with NSAID allergies, those on anticoagulants, or with underlying comorbidities. Full recall details are available here.