orphan drug

Rein Therapeutics prices $50M stock offering at $1.00 per share to fund LTI-03 Phase 2 trial for idiopathic pulmonary fibrosis, with cash runway extended through 2028.

Teva acquires Emalex Biosciences for $700M upfront plus $200M in milestones for first-in-class Tourette syndrome therapy, closing by Q3 2026.

FDA approves $REGN's Otarmeni, first dual AAV gene therapy for genetic hearing loss, in record 61 days under priority voucher program. 80% of patients showed improved hearing.

MeiraGTx acquires J&J's bota-vec gene therapy for rare blindness in $25M deal, targeting 2027 launch with promising Phase 3 clinical data.

FDA accepts Telix's Pixclara NDA for glioma imaging with September 2026 decision target. Orphan Drug designation provides competitive advantages.

Soligenix's HyBryte demonstrated superior efficacy versus Valchlor in treating cutaneous T-cell lymphoma, with 60% versus 20% success rates at 12 weeks, backed by $2.6M FDA grant.

SCYNEXIS completes acquisition of PXL-770 AMPK activator for ADPKD treatment. Deal includes $8M upfront, up to $188M milestones; Phase 2 study planned for late 2026.

Oculis' neuroprotective candidate Privosegtor receives EMA PRIME designation for optic neuritis, following FDA Breakthrough designation and positive Phase 2 data.

European Commission approves Sanofi's Rezurock for chronic GvHD treatment in patients 12+, based on 74% response rate from Phase 2 trials.

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.

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.

Grace Therapeutics will present at TD Cowen conference in March 2026. FDA sets April 2026 review date for GTx-104 aneurysmal subarachnoid hemorrhage treatment.

Turner Syndrome treatment market projected to double to $3.67B by 2033, driven by diagnostic innovation, increased awareness, and improved access to therapies globally.

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