rare disease

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.