MarketPulse

orphan drug

5 articles
GlobeNewswire Inc.GlobeNewswire Inc.··Pharming Group N.V.

Pharming Wins Japan Approval for Joenja, First APDS Treatment for Young Children

Pharming secures Japan approval for Joenja, first APDS treatment for children aged 4-11 globally, based on positive Phase III data showing lymphadenopathy reduction and immune function improvement.
PHARpediatric treatmentorphan drug
BenzingaBenzinga··Globe Newswire

Eton Pharmaceuticals Bolsters Rare Disease Portfolio With HEMANGEOL Acquisition

Eton Pharmaceuticals acquires HEMANGEOL commercialization rights, the only FDA-approved infantile hemangioma treatment, expanding its portfolio to ten commercial products with 2026 earnings accretion expected.
ETONrare diseaseorphan drug
GlobeNewswire Inc.GlobeNewswire Inc.··Na

Grace Therapeutics to Present at TD Cowen Conference; FDA Sets GTx-104 Review Date

Grace Therapeutics will present at TD Cowen conference in March 2026. FDA sets April 2026 review date for GTx-104 aneurysmal subarachnoid hemorrhage treatment.
GRCEFDA approvalclinical-stage
GlobeNewswire Inc.GlobeNewswire Inc.··Sns Insider

Turner Syndrome Treatment Market Set to Double by 2033 on Diagnostic Innovation

Turner Syndrome treatment market projected to double to $3.67B by 2033, driven by diagnostic innovation, increased awareness, and improved access to therapies globally.
PFELLYNVSSNYNVO+2healthcarerare disease
BenzingaBenzinga··Prnewswire

Citius Pharma Posts Inaugural Revenue on LYMPHIR™ Commercial Launch

Citius Pharma reports first revenue of $3.9M from LYMPHIR™ launch, raises $20.9M to support growth and advance pipeline candidates.
CTXRCTORorphan drugimmunotherapy