Praxis Precision Medicines Gains FDA Priority Review Boost
BOSTON, March 30, 2026 Praxis Precision Medicines has achieved a major regulatory milestone as the U.S. Food and Drug...
rare disease
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OS Therapies Advances OST-HER2 Toward Phase 3 Trial
New York, March 27, 2026 OS Therapies has achieved a significant regulatory milestone by securing meetings with multiple global...
Drug Farm Secures FDA RDEP Entry for DF-003 Rare Disease Drug
Albany, USA & Shanghai, China – March 26, 2026 Drug Farm announced that the U.S. Food and Drug Administration...
Ionis Zilganersen NDA Gets FDA Priority Review
CARLSBAD, California, March 23, 2026 Ionis Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has accepted...
FDA Approves AVLAYAH for Hunter Syndrome Treatment
SOUTH SAN FRANCISCO, Calif., March 25, 2026 In a landmark regulatory milestone, Denali Therapeutics announced that the U.S. Food...
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Pharming Group Secures Japan Approval for Joenja in APDS
LEIDEN, NETHERLANDS, March 24, 2026 Pharming Group has announced that Japan’s Ministry of Health, Labour and Welfare has approved...
FDA Approves Rhythm Pharmaceuticals’ IMCIVREE for Acquired Hypothalamic Obesity
BOSTON, March 19, 2026 Rhythm Pharmaceuticals announced that the U.S. Food and Drug Administration has approved Imcivree (setmelanotide) for...
Sanofi Rilzabrutinib Wins Japan Orphan Status
Paris, March 2, 2026 Sanofi announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug...
QTORIN Rapamycin Meets Phase 3 Endpoint in MLM
WAYNE, Pa., Feb. 24, 2026 — Palvella Therapeutics announced positive topline results from its Phase 3 SELVA clinical study...
FDA CRL Delays Bitopertin Approval for Rare EPP Disease
WATERTOWN, Mass., Feb. 13, 2026 — Disc Medicine announced that the U.S. Food and Drug Administration has issued a...
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