Pharma5: 5 Program Updates After the Recent FDA Overhaul
Here are five program updates after recent overturn at the U.S. Food and Drug Administration (FDA).
FDA Approves Gene Therapy for Rare Immune Disorder (2026)
FDA-approved Kresladi (marnetegragene autotemcel) is the first gene therapy treating leukocyte adhesion deficiency type I (LADI).
FDA Approves Drug to Treat Hunter Syndrome
Avlayah (tividenofusp alfa-eknm) has been FDA-approved to treat some individuals with Hunter Syndrome (Mucopolysaccharidosis type II or MPS II).
Uplizna Approved to Treat IgG4-Related Disease
Amgen’s UPLIZNA has been approved by the U.S. Food and Drug Administration as the first and only treatment for adults living with Immunoglobulin G4-related disease.
GSK’s Penmenvy Meningococcal Vaccine Approved by FDA
GSK’s Penmenvy (meningococcal groups A, B, C, W and Y vaccine) has been approved by the U.S. Food and Drug Administration for use in individuals aged 10 through 25 years.
FDA Approves Dupixent for Chronic Obstructive Pulmonary Disease
The U.S. Food and Drug Administration (FDA) has expanded its approval of Dupixent to chronic obstructive pulmonary disease (COPD).
UNLOXCYT Approved to Treat Metastatic cSCC
Checkpoint Therapeutics’ UNLOXCYT (cosibelimab-ipdl) has been approved by the U.S. Food and Drug Administration to treat adults with metastatic cutaneous squamous cell carcinoma or locally advanced cSCC who are not candidates for curative surgery or curative radiation.
FDA Approves Nipocalimab to Treat Sjögren’s Disease
Johnson & Johnson’s anti-FcRn antibody nipocalimab is the first investigational therapy to be granted breakthrough therapy designation by the U.S. Food and Drug Administration as a treatment for adults with moderate-to-severe Sjögren’s disease.
Updated COVID Vaccines Approved by FDA
FDA has approved and granted emergency use authorization for updated mRNA COVID-19 vaccines (2024-2025 formula) to include a monovalent (single) component that corresponds to the Omicron variant KP.2 strain of SARSCoV-2.
FDA Approves Epinephrine Nasal Spray
The U.S. Food and Drug Administration (FDA) has approved the first needle-free alternative to the EpiPen.