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Regulatory Intelligence & Drug Approvals2 weeks agoFDA Approves Zanvastro (zilganersen) for the Treatment of Alexander Disease
CARLSBAD, Calif.--(BUSINESS WIRE)--September 3, 2026-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has approved Zanvastro (zilganersen) for the...
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Regulatory Intelligence & Drug Approvals2 weeks agoSTAT+: Ionis wins FDA approval for first drug for Alexander disease, a rare and deadly neurological disorder
Zanvastro is the first disease-modifying treatment for Alexander disease, a rare and fatal disorder.
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Regulatory Intelligence & Drug Approvals2 weeks agoRemembering Peter Barton Hutt, an icon of FDA law
BIOCENTURY | POLITICS, POLICY & LAWREMEMBERING PETER BARTON HUTT, AN ICON OF FDA LAWHutt shaped FDA’s approach to the law in the 1970s, helped craft food...
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Regulatory Intelligence & Drug Approvals2 weeks agoRare Disease Drug Approvals
This page describes recent rare disease drug approvals that received a CDER or FDA communication. This is not an exhaustive list of all rare disease drug...
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Regulatory Intelligence & Drug Approvals2 weeks agoNovel Drug Approvals for 2026
FDA Novel Drug Therapy Approvals for 2026
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Regulatory Intelligence & Drug Approvals2 weeks agoFDA Approves First Drug to Treat Alexander Disease
The U.S. Food and Drug Administration has approved Zanvastro (zilganersen) injection for the treatment of Alexander disease in pediatric and adult patients.
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Regulatory Intelligence & Drug Approvals2 weeks agoReplimune’s melanoma drug would have been rejected again if not for senior CBER official
The FDA’s primary review team found that Replimune hadn’t sufficiently addressed deficiencies that had been pointed out in the two prior rejection letters for the melanoma...
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Regulatory Intelligence & Drug Approvals2 weeks agoOpinion: STAT+: A surprising bright spot at the FDA on regulating unproven stem cells
“These successes are surprising, worth discussing, and even celebrating a bit,” STAT Lab Dish columnist Paul Knoepfler writes of the FDA and stem cells.
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Regulatory Intelligence & Drug Approvals2 weeks agoCapricor, Replimune illustrate why FDA must listen to rare disease specialists
Last month’s FDA advisory committee meetings for therapies developed by Capricor Therapeutics and Replimune effectively illustrate the different outcomes for patients when the agency prioritizes specialization...
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Regulatory Intelligence & Drug Approvals2 weeks agoSpinal Cord Stimulator Recall: Boston Scientific Removes Infinion CX Lead
Infinion CX Lead bodies may experience mechanical stress at the anchor site resulting in inadequate stimulation requiring lead explant and replacement.
FDA warning letter accuses Bausch & Lomb of lacking commitment to remediation
Deep dive: Psychedelics back on FDA’s doorstep
‘Patients are not a renewable resource’: Rethinking rare disease R&D
Scientists turn pig semen extract into eye drops that kill cancer in mice
Novartis to pay $2B upfront to take next-gen PI3Kα inhibitor from Synnovation
