Palynziq Use Now Approved in Adolescents With PKU
On Feb. 27, 2026, the US Food and Drug Administration (FDA) approved BioMarin Pharmaceutical’s Palynziq® (pegvaliase‑pqpz) for use in adolescents aged 12 years and older with phenylketonuria (PKU). Initially approved for adults only, Palynziq is an enzyme substitution therapy designed to reduce blood phenylalanine (Phe) concentrations in individuals with PKU, a rare inherited metabolic disorder that can lead to serious neurocognitive complications if inadequately controlled. Approval in the adolescent population was based on results from the phase III study, which demonstrated statistically significant reductions in blood Phe levels compared with dietary management alone in adolescents with uncontrolled Phe concentrations. Palynziq is administered by subcutaneous (SC) injection following an induction, titration and maintenance dosing schedule based on blood Phe concentrations. Due to the chance that Palynziq may cause anaphylaxis (severe allergic responses), it has both a boxed warning and a risk evaluation and management strategy (REMS). Here is the updated prescribing information.
FDA Expands Sogroya’s Pediatric Use with Three New Growth Disorder Indications
On Feb. 27, 2026, the FDA approved Novo Nordisk’s Sogroya® (somapacitan‑beco), a long‑acting growth hormone injection, for three additional pediatric indications: idiopathic short stature (ISS), short stature in children born small for gestational age (SGA) without catch‑up growth by age two and growth failure associated with Noonan syndrome (NS). Sogroya is now approved for children two and a half years and older across these conditions, in addition to its existing approvals for pediatric and adult growth hormone deficiency. The long‑acting formulation allows for once‑weekly SC administration, offering an alternative to daily injections. Approval was based on data from the phase III REAL8 basket study, which demonstrated that once‑weekly Sogroya was noninferior to daily growth hormone therapy for mean annualized height velocity at week 52 across all three indications. The recommended dose is 0.24mg/kg body weight once weekly for treatment-naïve patients and patients switching from daily growth hormone. Sogroya is available in 5mg, 10mg and 15mg doses. Updated prescribing information is available here.
Juxtapid Label Expanded to Include Children as Young as Two with HoFH
The FDA approved Chiesi Global Rare Diseases’ Juxtapid® (lomitapide) oral capsules for use in pediatric patients aged two years and older with homozygous familial hypercholesterolemia (HoFH) on Feb. 25, 2026. This approval expands Juxtapid’s indication beyond adults, for whom it has been approved since 2012, and supports its use alongside a low‑fat diet and other lipid‑lowering therapies. Juxtapid is a microsomal triglyceride transfer protein (MTP) inhibitor that reduces the production of low‑density lipoprotein cholesterol (LDL‑C). FDA approval was based on results from a phase III, open‑label, multicenter study evaluating 43 pediatric patients aged five to 17 years of age, in which treatment with Juxtapid resulted in clinically meaningful LDL‑C reductions over a 24‑week period while patients remained on standard‑of‑care therapy. Juxtapid dosing is individualized and titrated to the highest tolerated level, with a recommended starting dose of 2mg for patients ages two to 15 years and 5mg for patients 16 years and older. The drug requires careful monitoring because of known hepatic safety risks, including elevations in liver enzymes. Juxtapid was approved with a REMS that includes a restricted distribution program due to the risk of hepatotoxicity. Here is the updated prescribing information.
First Generic Fluticasone Inhaler Approved & Will Launch Soon
On March 4, 2026, the FDA approved Glenmark Pharmaceuticals’ fluticasone propionate inhalation, 44mcg per actuation, a generic equivalent of Flovent® HFA (GlaxoSmithKline), for the maintenance treatment of asthma in patients requiring inhaled corticosteroid therapy. Glenmark’s product is the first approved and, therefore, received 180 days of market exclusivity upon commercial launch, which is expected in March 2026. According to IQVIA sales data, Flovent HFA 44mcg had annual sales of approximately $520.1 million in 2025.
3rd CNPV Approval Granted to Tecvayli with Darzalex Faspro for Earlier-Line Multiple Myeloma
The third FDA Commissioner’s National Priority Review Voucher (CNPRV) Pilot Program approval was granted on March 5, 2026, to Johnson & Johnson’s Tecvayli® (teclistamab‑cqyv) in combination with Darzalex Faspro® (daratumumab and hyaluronidase‑fihj) for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy, including a proteasome inhibitor and lenalidomide. This approval expands Tecvayli, a bispecific BCMA‑directed T‑cell engager, for use in second-line therapy, and combining it with SC Darzalex Faspro, a CD38‑directed monoclonal antibody, enables outpatient dosing while leveraging dual immune targeting. Approval was based on statistically significant and clinically meaningful improvements in progression‑free survival and overall survival versus standard‑of‑care regimens, with an 83% reduction in the risk of disease progression or death and 83% of patients alive at three years compared to 65% in the control group. Also, among patients alive and relapse‑free at six months, 90% remained free of cancer progression three years after the study began. As part of the CNPRV pilot program, this approval was issued 55 days after filing. The FDA also announced conversion of Tecvayli’s 2022 accelerated approval to traditional approval as a single‑agent treatment for adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy. The prescribing information for Tecvayli includes a Boxed Warning for cytokine release syndrome (CRS) and neurologic toxicities and requires REMS enrollment. Here is the updated prescribing information.
Recall
Udenyca Recalled
On Feb. 26, 2026, the FDA issued a class I recall for multiple lots of Accord BioPharma’s Udenyca® (pegfilgrastim‑cbqv) injection, 6mg/0.6mL single‑dose prefilled syringes. The recall applies to 116 cartons that were prompted by temperature abuse, as the affected units were stored at controlled room temperature rather than the required refrigerated conditions, which may result in reduced product potency. Loss of potency in pegfilgrastim products is considered medically significant, as it may increase the risk of severe neutropenia and febrile neutropenia, potentially leading to serious infections, hospitalization or death in vulnerable patients. Udenyca is a leukocyte growth factor FDA-approved biosimilar to Neulasta® (pegfilgrastim – Amgen) and is indicated to decrease the incidence of infection, as manifested by febrile neutropenia in patients with non‑myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant risk of febrile neutropenia. It is also approved for increasing survival in patients acutely exposed to myelosuppressive doses of radiation (hematopoietic sub-syndrome of acute radiation syndrome). For more information about the recall, see here.
Webcol Alcohol Prep Pads Recalled On March 3, 2026, Cardinal Health issued a voluntary recall of select lots of Webcol™ Large Alcohol Prep Pads after routine sterilization testing identified microbial contamination with Paenibacillus phoenicis, indicating the products may not be sterile. The contamination poses a potential infection risk, particularly for critically ill, immunocompromised, neonatal and pediatric patients. To date, there have been no reported adverse events or patient harm, and the probability of detection is low. Webcol Alcohol Prep Pads are intended to cleanse the skin to reduce bacteria before injections or minor procedures. To read about the recall, see here.