To understand how to succeed in launching an orphan medicine in the coming years, companies must learn from past launches and apply an orphan medicines-focused launch excellence framework for success.

In 1983, Congress passed the Orphan Drug Act (“ODA”) as part of an effort to provide market incentives for pharmaceutical manufacturers to produce “orphan drugs,” drugs for diseases that impact less ...

Korro Bio (NASDAQ:KRRO) said on Friday that it received U.S. FDA's orphan drug designation for investigational medicine KRRO-110 to treat alpha-1 antitrypsin deficiency. Stock is up about 10% ...

Setmelanotide is a melanocortin-4 receptor (MC4R) agonist designed to treat hyperphagia and obesity. Rhythm is evaluating setmelanotide in a global Phase 3 trial in patients with acquired hypothalamic ...

Epygenix Therapeutics is a precision medicine biotech company developing ... as potential treatments for Dravet syndrome, an ultra-orphan disease that causes severe convulsions and typically ...

BOSTON--(BUSINESS WIRE)--Porosome Therapeutics, Inc. (Porosome Therapeutics) today announced the Orphan Drug Designation ... Wayne State University School of Medicine. “It’s heartening to ...

It is often the case that a receptor may be identified before its ligand — a situation that leads to the use of the term 'orphan' G protein coupled receptor. Receptor antagonists for GPR14 are ...

Nagoya University Graduate School of Medicine. “Setmelanotide could provide new hope to these patients and their families.” In Japan, drugs can be designated as orphan drugs if they treat ...