Tag: Rare Disease
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Ultragenyx’s Fayuvi Wins FDA Approval as the First Treatment for Sanfilippo Syndrome Type A
The FDA approved Ultragenyx’s Fayuvi as the first treatment for pediatric MPS IIIA, supported by a 23.5-point cognitive treatment effect.
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Scholar Rock’s Isembyld Wins FDA Approval for Spinal Muscular Atrophy
The FDA approved Scholar Rock’s Isembyld for patients aged two years and older receiving SMN2-targeted therapy, adding the first muscle-directed treatment to established SMA care.
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FDA Approves Ionis’ Zanvastro as the First Treatment for Alexander Disease
The FDA approved Ionis’ Zanvastro as the first treatment for Alexander disease, validating GFAP-targeted antisense therapy in an ultra-rare neurological disorder.
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LEO Pharma Bets Up to $435 Million on Oral Therapy for Rare Sunlight-Induced Pain
LEO Pharma is acquiring global rights to dersimelagon from Tanabe Pharma in a deal worth up to $435 million. The oral MC1R agonist has completed Phase 3 development in erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) and has already been submitted to the FDA.
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Regeneron Wins FDA Approval for Pasatru in Ultra-Rare Bone Disease
The FDA approved Regeneron’s Pasatru for adults with fibrodysplasia ossificans progressiva after Phase 3 data showed a 90–94% reduction in new heterotopic ossification lesions versus placebo.
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Can New Data Rescue Capricor’s Duchenne Cell Therapy at the FDA?
Capricor is submitting new 24-month upper-limb data for deramiocel after an FDA advisory committee voted against the existing efficacy evidence, reopening the regulatory debate around the DMD cell therapy.
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Epicrispr Raises $90 Million to Silence Disease Genes Without Cutting DNA
Epicrispr raised $90 million to advance EPI-321, a single-dose AAV therapy designed to silence DUX4 without permanently editing DNA.