Safety and effectiveness in sickle cell disease were evaluated in a clinical trial of 11 patients aged 5 years to younger than 12 years. All 8 patients who were evaluable for efficacy achieved the primary efficacy outcome of VF12, defined as no protocol-defined severe vaso-occlusive crises for at least 12 consecutive months within the first 24 months after infusion.
July 1
Casgevy
On July 1, the FDA issued a supplemental approval for exagamglogene autotemcel (Casgevy, Vertex Pharmaceuticals, Inc) for patients aged 2 years and older with sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent β-thalassemia. Exagamglogene autotemcel had previously been approved for patients aged 12 years and older with sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent β-thalassemia. According to the FDA, this is the first gene therapy approved for patients aged 2 years and older with sickle cell disease.
July 7
Trutakna
On July 7, the FDA approved atacicept-vymj (Trutakna, Vera Therapeutics, Inc.) to reduce proteinuria in adults with primary immunoglobulin A nephropathy who are at risk for disease progression.
A randomized, double-blind, placebo-controlled clinical trial in adults with biopsy-confirmed IgA nephropathy supported the approval. Patients were randomly assigned to receive atacicept-vymj 150 mg by subcutaneous injection once weekly or a placebo. The primary efficacy endpoint was change from baseline in proteinuria, measured by urine protein-to-creatinine ratio from a 24-hour urine collection, after 9 months of treatment in the first 203 patients eligible for the month 9 visit. At 9 months, patients receiving atacicept-vymj had an average 46% reduction in proteinuria compared with placebo.
July 9
Sarclisa Escena
On July 9, the FDA approved isatuximab-irfc (Sarclisa Escena, Sanofi-Aventis US LLC) for subcutaneous injection for multiple myeloma indications.
The approval covers 3 multiple myeloma regimens. Isatuximab-irfc may be used with pomalidomide and dexamethasone in adults who have received at least 1 prior line of therapy, including lenalidomide and a proteasome inhibitor; with carfilzomib and dexamethasone in adults with relapsed or refractory multiple myeloma after 1 to 3 prior lines of therapy; and with bortezomib, lenalidomide, and dexamethasone in adults with newly diagnosed multiple myeloma who are not eligible for autologous stem cell transplant.
July 10
Keytruda and Padcev
On July 10, the FDA approved pembrolizumab (Keytruda) or pembrolizumab and berahyaluronidase alfa-pmph (Keytruda Qlex), each in combination with enfortumab vedotin-ejfv (Padcev), as neoadjuvant treatment before surgery followed by adjuvant treatment after cystectomy for adults with muscle-invasive bladder cancer. The approval extends the prior indication from cisplatin-ineligible patients to all patients with muscle-invasive bladder cancer who are candidates for cystectomy.
The approval was based on KEYNOTE-B15/EV-304, an open-label, randomized, active-controlled, multicenter trial of 808 patients with previously untreated muscle-invasive bladder cancer who were candidates for radical cystectomy with pelvic lymph node dissection and eligible for cisplatin-based chemotherapy.
July 14
Revtorpyk
On July 14, the FDA approved gedatolisib (Revtorpyk, Celcuity Inc) in combination with fulvestrant, with or without palbociclib, for adults with hormone receptor-positive, HER2-negative locally advanced or metastatic breast cancer without a PIK3CA mutation detected following progression on or after at least 1 line of hormone therapy in the metastatic setting.
Efficacy was evaluated in study 1 of VIKTORIA-1, an open-label, randomized, multicenter trial of 392 adults with locally advanced, inoperable, or metastatic HR-positive, HER2-negative breast cancer. Patients were randomly assigned 1:1:1 to receive gedatolisib with fulvestrant and palbociclib, gedatolisib with fulvestrant, or fulvestrant alone. Patients received treatment until there was disease progression or unacceptable toxicity.
July 16
Lipfendra
On July 16, the FDA approved enlicitide (Lipfendra, Merck Sharp & Dohme, LLC) for use with diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in adults with high cholesterol or heterozygous familial hypercholesterolemia (HeFH), an inherited form of high cholesterol. Enlicitide is the first oral therapy to inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9) and is administered once daily as a tablet.
The approval was supported by 2 randomized, double-blind, placebo-controlled trials, NCT05952856 and NCT05952869, that evaluated the efficacy and safety of enlicitide in 3,207 adults with severe hypercholesterolemia, including patients with and without HeFH, who were already receiving maximally tolerated statin therapy.
July 24
Tylenol with Naproxen
On July 24, the FDA approved Tylenol with Naproxen (acetaminophen 325 mg/naproxen sodium 110 mg), the first nonprescription fixed-dose product to combine acetaminophen and naproxen sodium in a single tablet. The medication is indicated for the temporary relief of minor aches and pains due to headache, backache, muscular aches, toothache, menstrual cramps, and minor pain of arthritis in adults and children aged 12 years or older.
The approval was supported by evidence establishing the effectiveness and safety of acetaminophen/naproxen sodium and demonstrating the contribution of each active ingredient to its overall pain-relieving effect. According to the FDA, combining the 2 active ingredients in a single tablet provides consumers with “a new nonprescription option for up to 12 hours of pain relief.”