rare disease

Catalyst Pharma settles patent litigation with Hetero Labs, blocking generic FIRDAPSE entry until 2035. Fourth major competitor settlement extends exclusivity.

AstraZeneca ($AZN) reported Q1 revenue of $15.29B, up 13% YoY, with oncology surging 20%. Shares fell 2.30% despite beating estimates.

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.

Belite Bio begins rolling NDA submission to FDA for tinlarebant, a potential breakthrough oral therapy for rare Stargardt disease, targeting 2027 commercialization.

Upsher-Smith launches digital support platforms for DMD treatment KYMBEE, enhancing patient access and clinician resources through comprehensive support program.

Viking Therapeutics to present at two major biotech conferences in April 2026, conducting investor meetings in New York and Boston.

Neurocrine Biosciences to acquire Soleno Therapeutics for $2.9B in all-cash deal, paying $53/share—a 34% premium driven by Soleno's FDA-approved Vykat XR for Prader-Willi Syndrome.

Neurocrine acquires Soleno for $2.9B to gain Vykat XR, a rare disease blockbuster generating $190M in 2025 revenue.

Priovant initiates Phase 2b/3 trial for brepocitinib in lichen planopilaris, marking fourth indication; dermatomyositis FDA decision expected Q3 2026.

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 secures EMA PRIME fast-track designation for Privosegtor, a potential first-in-class neuroprotective optic neuritis therapy, following FDA Breakthrough status in January 2026.

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

Soligenix's dusquetide (SGX945) receives EU orphan drug designation for Behçet's Disease, backed by Phase 2a data showing 40% ulcer reduction with no adverse events.

Rhythm Pharma wins FDA approval for IMCIVREE, the first therapy for acquired hypothalamic obesity, based on 18.4% placebo-adjusted BMI reduction.

Soligenix's SGX945 earns UK Promising Innovative Medicine designation for Behçet's Disease, enabling early patient access with Phase 2 data showing 40% ulcer improvement.

Kyntra Bio will report fourth quarter and full year 2025 financial results on March 16, 2026, as the biopharmaceutical company advances its oncology and rare disease pipeline.

European Commission approves every-4-weeks dosing regimen for Elfabrio, reducing patient treatment burden and triggering $25 million milestone payment to Protalix from Chiesi.

Global skeletal dysplasia market projected to grow from $3.48B (2026) to $5.37B (2032) at 7.42% CAGR, driven by diagnostic advances and innovative therapies.

Rezolute to present ersodetug antibody therapy program at Citizens Life Sciences Conference in Miami, March 10-11, 2026, with direct investor meetings.

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.