From genetically modified pig kidney transplants to breakthrough antibiotics for drug-resistant infections, February’s FDA approvals highlight cutting-edge advancements across multiple specialties. Here is a comprehensive overview of the month's key FDA decisions.
Cardiology
FDA Approves Trials for Genetically Modified Pig Kidney Transplants
The FDA approved the first clinical trials of genetically modified porcine kidney transplants for patients with end-stage renal disease. United Therapeutics and eGenesis will assess safety, immunogenicity, and viability, with modifications designed to reduce rejection and enhance compatibility. Patients will be monitored for 24 weeks post-transplant, with lifelong follow-ups. This approval advances xenotransplantation as a potential solution to the organ shortage.
Infectious Diseases
FDA Approves Novel Antibiotic for Complicated Intra-Abdominal Infections
The FDA approved aztreonam-avibactam (EMBLAVEO) with metronidazole for treating complicated intra-abdominal infections in adults with limited options. This marks the first monobactam/β-lactamase inhibitor combination therapy. The phase III REVISIT trial demonstrated its efficacy against resistant gram-negative bacteria, offering a new treatment alternative for severe infections.
Neurology
FDA Approves Medtronic's Adaptive Deep Brain Stimulation System
The FDA approved Medtronic's BrainSense Adaptive deep brain stimulation (DBS) system for Parkinson's disease treatment. This closed-loop neuromodulation therapy automatically adjusts stimulation parameters in real-time based on individual brain activity, enhancing symptom management. The approval follows the Adaptive DBS Algorithm for Personalized Therapy in Parkinson’s Disease trial, which demonstrated the system's safety and efficacy.
FDA Approves Mirdametinib for NF1-Associated Plexiform Neurofibromas
Mirdametinib (Gomekli) has been approved for treating inoperable neurofibromatosis type 1-associated plexiform neurofibromas in patients aged 2 years and older. The phase III ReNeu trial reported an overall response rate of 41% in adults and 52% in pediatric patients, with all responses being partial.
FDA Approves New Drug-Device Combo for Parkinson’s Symptoms
The FDA approved Onapgo, a continuous apomorphine hydrochloride infusion, for treating motor fluctuations in adults with advanced Parkinson's disease. The phase III TOLEDO trial demonstrated that Onapgo significantly reduced daily "off episode" duration by a mean of 2.6 hours compared to 0.9 hours with placebo. This subcutaneous infusion offers an alternative to intermittent apomorphine injections, providing more consistent symptom management.
Rare Disorders
FDA Approves First Treatment for Cerebrotendinous Xanthomatosis
Ctexli (chenodiol) has been approved as the first treatment for cerebrotendinous xanthomatosis (CTX), a rare lipid storage disorder. Clinical trials showed that Ctexli significantly reduced plasma cholestanol and urine 23S-pentol levels, biomarkers associated with CTX's metabolic dysfunction.
Rheumatology
FDA Approves Tocilizumab Biosimilar
The FDA approved AVTOZMA (tocilizumab-anoh), a biosimilar to ACTEMRA, for treating rheumatoid arthritis and other inflammatory conditions. A phase III trial confirmed equivalence in efficacy, safety, and pharmacokinetics between AVTOZMA and the reference product.
Spinal Muscular Atrophy
FDA Approves Evrysdi Tablet for SMA Treatment
The FDA approved a tablet formulation of Evrysdi® (risdiplam) for treating spinal muscular atrophy in patients aged 2 years and older weighing at least 20 kg. This tablet offers the same efficacy and safety profile as the existing oral solution but provides the convenience of room-temperature storage.
Regulatory Updates
FDA Regulations Could Impact AI Integration in EMRs
New FDA regulations may classify certain artificial intelligence (AI) tools used in electronic medical records as medical devices, necessitating premarket review. The FDA's new regulations could influence AI integration in clinical practice by addressing concerns about automation bias and the timing of AI-assisted clinical decisions.
FDA Warns of Potential Failures in Diabetes Device Alerts
The FDA issued a warning regarding safety risks associated with smartphone-connected diabetes devices. Missed alerts from continuous glucose monitors and insulin pumps have led to severe complications, including hypoglycemia and diabetic ketoacidosis. Users are advised to regularly verify alert configurations and ensure device compatibility after software updates.
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