The FDA approved Johnson & Johnson's FcRn blocker IMAAVY (nipocalimab-aahu) on August 24 for warm autoimmune hemolytic anemia (wAIHA) in adults and children aged 12 and older. It is the first therapy approved specifically for wAIHA, a disease in which pathogenic IgG autoantibodies destroy red blood cells, causing severe anemia, extreme fatigue and significant morbidity and mortality risk. The report forecasts annual sales exceeding $5 billion.
The FDA granted accelerated approval to Ultragenyx Pharmaceutical's AAV gene therapy Genglycos (pariglasgene brecaparvovec-opnr, DTX401) for adults and children aged 8 and older with glycogen storage disease type Ia (GSDIa). GSDIa is an ultra-rare inherited metabolic disorder caused by G6PC gene mutations leading to glucose-6-phosphatase deficiency. It is the second AAV drug the FDA has approved recently.
The FDA has approved Johnson & Johnson's IMAAVY (nipocalimab-aahu) for warm antibody autoimmune hemolytic anemia in adults and children aged 12 and older who are receiving or have previously received corticosteroid treatment, an announcement made on August 24. wAIHA is a rare, life-threatening disease in which pathogenic IgG autoantibodies attach to and destroy red blood cells, causing severe anemia, extreme fatigue and increased morbidity and mortality risk.
Johnson & Johnson's FcRn-targeting monoclonal antibody nipocalimab (IMAAVY) received FDA approval on August 24 for warm autoimmune hemolytic anemia in adults and children aged 12 and older who have received glucocorticoid therapy before or currently. Nipocalimab selectively blocks FcRn to reduce circulating IgG antibodies, including pathogenic autoantibodies. Johnson & Johnson obtained the drug through its $6.5 billion acquisition of Momenta in August 2020.
The FDA has expanded the label for Johnson & Johnson's monoclonal antibody Imaavy to include warm autoimmune hemolytic anemia, a rare disorder that destroys red blood cells. The approval comes sixteen months after Imaavy's initial FDA approval.
Regenxbio announced on August 24 that the FDA has again placed its gene therapy trial for MPS II (Hunter syndrome) on hold after small masses were found on the spines of five patients; the company said the patients have no related symptoms. The pause comes seven months after Regenxbio disclosed that a boy receiving a similar MPS I therapy developed a brain tumor, described as the first case conclusively linked to AAV gene therapy.
The Centers for Medicare and Medicaid Services issued a final rule barring federal Medicaid and CHIP funding for pediatric gender-affirming medications and surgery, effective Oct. 13. Medicaid experts say the Trump administration's approach could set a precedent for denying coverage of drugs beyond gender-affirming care, challenging the expectation that Medicaid covers most FDA-approved outpatient drugs when manufacturers pay rebates.
Why it matters: A Medicaid coverage rule could upend the assumption that FDA-approved drugs with rebates must be covered, with broad implications for drugmakers.
On August 19, 2026, the FDA granted accelerated approval to Genglycos (pariglasgene brecaparvovec-opnr) for patients aged 8 and older with glycogen storage disease type Ia (GSDIa). It is the first approved therapy for this rare inherited disease and a one-time AAV8 gene therapy approved on a surrogate endpoint. The approval comes as the FDA tightens its review standards for cell and gene therapies.
The US FDA has approved Regeneron's BLA for garetosmab, a monoclonal antibody, to treat adults with fibrodysplasia ossificans progressiva (FOP), a progressive ultra-rare genetic disease characterised by heterotopic ossification. The approval was announced on August 19. Per the Insight database, garetosmab is the first approved activin A-targeted therapy worldwide.
FDA has approved OncoSil Medical's phosphorus-32 (P-32) radioactive implant device through the Humanitarian Device Exemption pathway for unresectable, non-metastatic distal bile duct cancer. It is described as the first and so far only Class III medical device approved specifically for this rare, aggressive bile duct cancer subtype. The approval adds a new radiotherapy option delivered via an established isotope rather than newer agents such as lutetium-177 or actinium-225.
The FDA granted accelerated approval to Ultragenyx's AAV gene therapy pariglasgene brecaparvovec-opnr, brand name Genglycos, for adults and children over 8 with glycogen storage disease type Ia. In the phase 3 GlucoGene study, the one-time AAV8 therapy cut daily cornstarch intake by a mean 31% from baseline versus placebo over 48 weeks. Ultragenyx, which also received a priority review voucher, must provide two years of safety and efficacy data on 50 treated and 20 control patients; it awaits a Sept. 19 decision on UX1111.
The FDA has approved the AAV gene therapy Genglycos (pariglasgene brecaparvovec-opnr, development code DTX401) for glycogen storage disease type Ia, a rare inherited metabolic disorder caused by G6PC gene mutations leading to glucose-6-phosphatase deficiency. It is the first gene therapy the FDA has approved for this disease.
The FDA granted accelerated approval to Ultragenyx Pharmaceutical's Genglycos (pariglasgene brecaparvovec-opnr), a one-time AAV8 gene therapy and the first approved treatment for glycogen storage disease type Ia in patients aged 8 and older. In a 48-week randomized placebo-controlled study, patients achieved a statistically significant 31% mean reduction in daily cornstarch intake, the primary endpoint. The manufacturer must complete additional trials to confirm effectiveness, and the label warns of anaphylaxis, liver toxicity, adrenal insufficiency and tumorigenicity risk.
The FDA has approved Regeneron Pharmaceuticals' Pasatru for fibrodysplasia ossificans progressiva, an ultra-rare disease in which bone grows where it should not. The approval caps a three-decade effort and follows a pivotal trial in which the medicine drastically cut the risk of new bone formation. Patients with FOP typically rely on wheelchairs by age 25, and investigators hope Pasatru can help maintain mobility.
On August 6, 2026, the FDA granted accelerated approval to Replimune's oncolytic virus therapy Tudriqev in combination with a PD-1 inhibitor for patients with PD-1-resistant advanced melanoma. It is the first approved oncolytic virus combination therapy worldwide and the first oncolytic virus approval by the FDA in 11 years.
President Donald Trump has named White House aide Heidi Overton, M.D., Ph.D., as his nominee for FDA commissioner, ending three months without a permanent leader since Marty Makary departed in May. Overton, currently deputy director of the White House Domestic Policy Council, previously directed the Center for a Healthy America at the America First Policy Institute. She faces Senate confirmation before the Health, Education, Labor and Pensions Committee chaired by Sen. Bill Cassidy.
The Trump administration has nominated Heidi Overton, a Johns Hopkins-trained physician and former chief policy officer at Center for a Healthy America before joining the White House, as FDA's next commissioner. She would succeed Marty Makary, whose term was marked by conflicts with the administration and controversial regulatory decisions. Her selection is expected to advance the MAHA agenda of HHS Secretary Robert F. Kennedy Jr. and raises questions about FDA independence from political influence.
FDA has approved Lantheus's tau PET tracer TAUKLARIFY. The approval rests on two blinded-read studies covering PET images from more than 500 subjects across three trials of cognitively normal, mildly cognitively impaired and mild Alzheimer's disease participants. Safety was assessed in 1734 subjects, with headache (0.7%) the most common adverse reaction at rates of 0.1% or more. In 2023, Lantheus's partner Xontong Pharmaceutical out-licensed all ex-China rights to the tracer, then code-named XTR006/MK-6240, through a US joint venture while retaining exclusive rights to develop, manufacture and commercialize it in mainland China, where it is now in Phase III trials.
The FDA has approved Regeneron's Pasatru (garetosmab), an intravenous anti-activin A monoclonal antibody given every four weeks, for fibrodysplasia ossificans progressiva in adults. The approval rests on the phase 3 Optima trial, in which 63 adults were randomized and the 10- and 3-mg/kg doses cut new bone lesions by 90% and 94% versus placebo over 56 weeks, with high dose reducing flare-ups by 89%. Pasatru becomes the second FOP drug after Ipsen's Sohonos and will reach roughly 220 U.S. adult patients; Regeneron declined to state a list price but plans co-pay support and free medicine for eligible patients.
JAMA reports that the FDA has approved a gene therapy for younger children with sickle cell disease. Only the headline was available; the full text was not captured. DOI: 10.1001/jama.2026.9488.
