Nexviazyme-Avalglucosidase Alfa-Ngpt
Enzyme replacement transformed Pompe disease, but the original therapy reached skeletal muscle inefficiently, limiting benefit for patients with predominantly muscular disease. Nexviazyme (avalglucosidase alfa) was engineered to improve that delivery. It carries roughly fifteen-fold more mannose-6-phosphate than the earlier enzyme, improving uptake into muscle cells where it clears the glycogen accumulation caused by acid alpha-glucosidase […]
Saphnelo-Anifrolumab-Fnia
Systemic lupus erythematosus had seen only one new biologic approved in over fifty years before Saphnelo (anifrolumab), and the type I interferon pathway had long been a suspected but unproven target. Unlike therapies aimed at a single cytokine, Saphnelo blocks signaling from all type I interferons by antagonizing their shared receptor. The intravenous antibody is […]
Rylaze-Asparaginase Erwinia Chrysanthemi (Recombinant)-Rywn
Asparaginase is essential to modern leukemia regimens, but hypersensitivity to E. coli-derived products forces a switch, and supply of the Erwinia alternative had been unreliable. Rylaze addressed both problems. It depletes asparagine, an amino acid leukemia cells cannot synthesize but depend on to survive. Approved as a component of a chemotherapy regimen, it serves patients […]
Aduhelm-Aducanumab-Avwa
Aduhelm (aducanumab) became the first anti-amyloid antibody to reach the market, in one of the most contested approvals in FDA history, after decades of failed trials built on the amyloid hypothesis. Given as a monthly intravenous infusion, it targeted aggregated amyloid beta and held accelerated approval based on plaque reduction alone. That approval proceeded despite […]
Rybrevant-Amivantamab-Vmjw
EGFR exon 20 insertion mutations resist conventional EGFR inhibitors, leaving a subset of lung cancer patients with few targeted options. Rybrevant (amivantamab) was built to reach them. Amivantamab binds both EGFR and MET, blocking parallel growth signals while also recruiting immune effector cells to the tumor. The intravenous bispecific antibody was first approved for advanced […]
Zynlonta-Loncastuximab Tesirine-Lpyl
Relapsed large B-cell lymphoma remains difficult to treat after multiple lines, and antibody-drug conjugates offer a way to concentrate cytotoxic payloads on B cells. Zynlonta (loncastuximab tesirine) targets CD19. Once internalized, it releases a pyrrolobenzodiazepine dimer that cross-links DNA and kills the lymphoma cell. The CD19-directed antibody-drug conjugate is infused for adults with relapsed or […]
Jemperli-Dostarlimab-Gxly
Checkpoint inhibitors reshaped gynecologic oncology, and mismatch repair status became one of the strongest predictors of response. Jemperli (dostarlimab) was developed around that biomarker. By preventing PD-1 from restraining T cells, Jemperli allows the immune system to recognize and attack tumor cells. The intravenous antibody was first approved for adults with mismatch repair-deficient recurrent or […]
Evkeeza-Evinacumab-Dgnb
Homozygous familial hypercholesterolemia produces extreme LDL levels from birth, and patients often respond poorly to therapies that depend on a working LDL receptor. Evkeeza (evinacumab) offered a route around that limitation. Unlike statins, PCSK9 inhibitors, and most other lipid therapies, Evkeeza works independently of LDL receptor activity, which makes it effective even in receptor-negative patients. […]
Pasatru-Garetosmab-grts
For people living with fibrodysplasia ossificans progressiva (FOP), the body gradually forms bone in places where bone should not exist. This can severely restrict movement and progressively affect everyday life. Pasatru (garetosmab-grts) is designed to help address this process by reducing new heterotopic bone formation and disease flare-ups in adults with FOP. Its approval provides […]
Lytenava-Bevacizumab-vikg
For years, bevacizumab has been widely used in ophthalmology through repurposed oncology formulations. Lytenava (bevacizumab-vikg) changes that by becoming the first FDA-approved bevacizumab specifically developed for intravitreal use in patients with wet age-related macular degeneration. By inhibiting VEGF, Lytenava reduces abnormal blood vessel growth and leakage that can lead to progressive vision loss. Having a […]
