FDA Approves Another Prolia Biosimilar, Ponlimsi

On March 30, 2026, the US Food and Drug Administration (FDA) approved Teva Pharmaceutical Industries’ Ponlimsi^™ (denosumab-adet), another biosimilar to Prolia^® (denosumab). Ponlimsi is approved for all Prolia indications, including treatment of postmenopausal women and men with osteoporosis at high risk for fracture, glucocorticoid‑induced osteoporosis, and bone loss associated with androgen deprivation therapy for nonmetastatic prostate cancer or adjuvant aromatase inhibitor therapy for breast cancer. It is administered by a healthcare provider as a 60mg subcutaneous (SC) injection once every six months. Several Prolia biosimilars have launched in the US, with additional FDA‑approved products expected to enter the market. Here is the prescribing information.

High‑Dose Spinraza Regimen for SMA Approved

The FDA approved Biogen’s Spinraza^® (nusinersen) high‑dose intrathecal regimen for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients on March 30, 2026. The updated dosing regimen is designed to deliver higher drug exposure during both the loading and maintenance phases, and to use a faster loading schedule. Under the previous dosing regimen, Spinraza was administered as four loading doses at 12mg over approximately two months, with the first three doses given at 14‑day intervals and a fourth dose 30 days later, followed by maintenance dosing three times per year. The newly approved high‑dose regimen includes two 50mg loading doses administered 14 days apart, followed by 28mg maintenance doses every four months. For patients transitioning from the low-dose regimen, a single high‑dose loading phase is recommended. Approval was supported by the phase II/III DEVOTE study, which demonstrated statistically significant improvements in motor function among treatment‑naïve, symptomatic infants receiving high‑dose Spinraza compared with a matched untreated control from the ENDEAR study. A higher incidence of pneumonia and pneumonia‑related adverse reactions was observed with the high‑dose regimen. Biogen states the 28mg vial will be priced at approximately $152,000, consistent with the current 12mg vial, while the 50mg vial will be priced at about $271,000. Both doses are expected to be available in the US in the coming weeks. The updated prescribing information can be found here.

FDA Adopts Mechanism‑Based Labeling for Alyftrek and Trikafta, Expanding Access

On April 1, 2026, the FDA expanded Vertex Pharmaceuticals’ Alyftrek^® (vanzacaftor, tezacaftor, ivacaftor) for expanded use in people with cystic fibrosis (CF) aged six years and older who have a (cystic fibrosis transmembrane conductance regulator protein) CFTR gene variant that is either responsive based on clinical or in vitro data or results in production of CFTR protein. It also applies to Trikafta^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in patients ages two and older. The label expansion broadens eligibility beyond specific variant listings (i.e., F508del) to a mechanism‑based criterion, enabling treatment regardless of variant location within the CFTR protein. The label expansion is expected to make approximately 800 additional US patients newly eligible for CFTR modulator therapy, increasing eligibility to nearly 95% of people with CF. Alyftrek and Trikafta are oral, triple‑combination CFTR modulators that restore CFTR function by targeting defects in CFTR protein processing and activity, treating the underlying cause of cystic fibrosis. Here is prescribing information for Alyftrek and Trikafta.

Tecartus Converts to Full Approval for Mantle Cell Lymphoma

On April 2, 2026, the FDA granted full approval to Gilead Sciences’ Kite for Tecartus^® (brexucabtagene autoleucel), an autologous CD19‑directed CAR T‑cell therapy, for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) after one or more prior lines of therapy, including patients who are Bruton tyrosine kinase inhibitor (BTKi)‑naïve. The approval converts its 2020 accelerated approval to traditional approval based on cohort three of the phase II ZUMA‑2 trial, which demonstrated a 91% objective response rate, 79% complete remission rate, and a median duration of response not reached at 23 months. This confirms durable clinical benefit and fulfills post‑marketing requirements. The updated prescribing information is here.

MedWatch Update

FDA Flags New Safety Concerns for Tavneos Following Reports of Serious Liver Injury

The FDA issued a safety communication on March 31, 2026, highlighting new concerns regarding Amgen’s Tavneos^® (avacopan). This is an oral complement 5a receptor (C5aR) antagonist approved as adjunctive therapy for adults with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)‑associated vasculitis (granulomatosis with polyangiitis and microscopic polyangiitis) since 2021. The agency identified 76 postmarketing cases of drug‑induced liver injury, including 74 serious outcomes, 54 hospitalizations, and eight deaths, with several cases involving vanishing bile duct syndrome, a rare but potentially irreversible condition. While hepatotoxicity was previously noted in clinical trials and included in product labeling, the FDA stated that the severity and fatal outcomes observed post‑approval represent new safety concerns. The FDA reported a median time to onset of liver injury of approximately 46 days after treatment initiation and advised clinicians to conduct baseline and ongoing liver function monitoring. The agency continues to evaluate Tavneos’ benefit‑risk profile and has indicated it will provide additional updates as more data become available. More information is available here.