rare disease

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.

Rhythm Pharmaceuticals reports positive Phase 3 data for setmelanotide treating acquired hypothalamic obesity, with FDA decision expected March 2026.

EMA committee endorses mavorixafor (XOLREMDI) for WHIM syndrome treatment, marking first approved therapy in Europe. Final Commission approval expected Q2 2026.

Vertex Pharmaceuticals outperforms Centessa with profitable cystic fibrosis revenue, successful product launches, and advanced pipeline, offering lower-risk investment profile versus pre-revenue competitor.

Soligenix wins EMA orphan drug designation backing for SGX945 treating Behçet's Disease, following positive Phase 2a results and prior FDA support.

Eton Pharma gains FDA approval for DESMODA, the first liquid desmopressin formulation for central diabetes insipidus, launching March 9 with projected peak sales of $30-50 million.

Fulcrum's pociredir shows strong fetal hemoglobin gains in sickle cell Phase 1b trial, with 58% of patients reaching therapeutic levels and reduced pain crises.

ALS therapeutics market to more than double by 2035, reaching $1.96 billion. Growth driven by genetic therapies, improved diagnostics, and disease awareness.

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

ANI Pharmaceuticals will report 2025 results on February 27, 2026, reaffirming confidence in meeting or exceeding guidance for revenues, EBITDA, and earnings per share.