LEO Pharma Gains FDA Priority Review for Dersimelagon
BALLERUP, Denmark — September 18, 2026 LEO Pharma announced that the U.S. Food and Drug Administration (FDA) has accepted...
rare disease
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Hemab Doses First Participant in HMB-003 Phase 1
CAMBRIDGE, Mass. and COPENHAGEN, Denmark — September 17, 2026 Hemab Therapeutics announced that the first healthy volunteer has been...
Capricor to Present Deramiocel HOPE-3 Data at WMS 2026
SAN DIEGO, Calif. — September 17, 2026 Capricor Therapeutics announced that 24-month data from the HOPE-3 Phase 3 trial...
Novo Nordisk Gains Positive CHMP Opinion for FREHEMGO
BAGSVAERD, Denmark — September 17, 2026 Novo Nordisk announced that the Committee for Medicinal Products for Human Use (CHMP)...
Biogen Reports Positive Pediatric EMPAVELI Data in C3G
CAMBRIDGE, Mass. — September 17, 2026 Biogen announced publication of prespecified adolescent subgroup results from the Phase 3 VALIANT...
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Ultragenyx Wins FDA Approval for FAYUVI Gene Therapy
Novato, California, U.S., September 17, 2026 Ultragenyx Pharmaceutical Inc. announced that the U.S. Food and Drug Administration (FDA) has...
Rocket Pharmaceuticals Advances RP-A501 Toward Danon Disease Approval
CRANBURY, N.J. — September 15, 2026 Rocket Pharmaceuticals, Inc. announced alignment with the U.S. Food and Drug Administration (FDA)...
Lundbeck Completes Enrollment in Phase III Dravet Trial
Copenhagen, Denmark, September 15, 2026 H. Lundbeck A/S has announced that the last patient has been randomized in DEEp...
Tenaya Therapeutics Gains RMAT Designation for TN-401
SOUTH SAN FRANCISCO, Calif. — September 14, 2026 Tenaya Therapeutics, Inc. announced that the U.S. Food and Drug Administration...
Sionna Advances SION-451 and SION-2222 Into Phase 2a
WALTHAM, Mass. — September 14, 2026 Sionna Therapeutics, Inc. announced plans to advance its preferred proprietary dual combination of...
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