X4 Pharmaceuticals (XFOR) announced that its Marketing Authorization Application for mavorixafor for the treatment of WHIM syndrome, a rare ...

Submission supported by positive results from global, Phase 3 4WHIM clinical trial; U.S. regulatory approval in WHIM syndrome ...

Paris: Following the adoption of a positive opinion by the European Medicines Agency's (EMA) Committee for Medicinal Products ...

EMA validates X4 Pharma’s marketing authorization application for mavorixafor to treat WHIM syndrome: Boston Monday, January 27, 2025, 11:00 Hrs [IST] X4 Pharmaceuticals, a comp ...

Committee for Medicinal Products for Human Use (CHMP). The EMA previously granted orphan designation to mavorixafor in WHIM syndrome. In April 2024, mavorixafor received U.S. Food and Drug ...

Bristol Myers on Friday said the European Medicines Agency's Committee for Medicinal Products for Human Use recommended approval of Breyanzi for the treatment of adults with relapsed or refractory ...

Mereo’s alvelestat, which targets alpha-1 antitrypsin deficiency-associated lung disease, has also gained positive recognition from the EMA’s Committee for Orphan Medicinal Products ...

The European Medicines Agency (EMA)’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on the Company’s application for Orphan Designation for alvelestat.

Despite some market exclusivity concerns, GERN still has many years of sales ahead, unencumbered by generic competition.

In a heartwarming reunion tale, six-year-old Benjamin Joseph has finally found a forever home with his biological family, courtesy of the innovative Children First ...