Rare Diseases
Alkermes Acquires Avadel Pharmaceuticals for $2.1B, Gaining Sleep Drug LUMRYZ and Setting Strategic Evolution
Alkermes; Avadel Pharmaceuticals; acquisition; LUMRYZ; narcolepsy; sleep medicine; strategic portfolio; biopharmaceuticals; rare disease; deal premium
Sanofi’s efdoralprin alfa (INBRX-101) achieves all endpoints in Phase 2 alpha-1 antitrypsin deficiency (AATD) trial
Sanofi; efdoralprin alfa; INBRX-101; AATD; alpha-1 antitrypsin deficiency; Phase 2; Inhibrx; acquisition; rare disease; plasma-derived therapy; Zemaira
Novo throws weight behind budding rare disease pipeline
Novo Nordisk; rare disease; pipeline; Omeros; Zaltenibart; MASP-3 inhibitor; PNH; complement system; milestone payments
Gene Therapy Delivers Long-Term Immune Protection in Children With Rare Immune Disorders
gene therapy; long-term immune protection; ADA-SCID; severe combined immunodeficiency; LAD-I; leukocyte adhesion deficiency; rare disease; pediatric immunology; clinical trial; curative treatment
BioCryst Snaps Up Fellow Angioedema Player Astria for $700M
BioCryst; Astria Therapeutics; acquisition; hereditary angioedema (HAE); navenibart; rare disease; pharmaceutical industry; cash and stock deal; Jill Milne; $700 million
FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful
PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure
Diagonal Therapeutics Secures $128 Million Series A Funding through Innovative Antibody Agonists
Diagonal Therapeutics, Series A funding, Antibody agonism, Cellular “on” switches, Rare diseases