Acsh.org2h
Rare Diseases Are Neglected. The FDA Needs A Dedicated 'Center of Excellence for Rare Diseases'.
The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive ...
Tempest Granted Fast Track Designation from the U.S. Food and Drug Administration for Amezalpat to Treat Patients with Hepatocellular Carcinoma
Fast Track Designation highlights potential of amezalpat to address unmet need for patients with HCC Amezalpat builds ...
Biohaven's troriluzole gains FDA priority review for SCA
NEW HAVEN, Conn. - Biohaven Ltd. (NYSE: BHVN), a biotechnology company with a market capitalization of $3.87 billion, announced today that the U.S. Food and Drug Administration (FDA) has accepted its ...
Moleculin Announces First European Approval for Phase 3 AML Trial
HOUSTON - Moleculin Biotech, Inc. (NASDAQ:MBRX), a late-stage pharmaceutical company currently trading near its 52-week low of $0.66, announced it has received the first European regulatory approval ...
GlobalData on MSN4d
FDA grants orphan drug status to Arbor Biotechnologies’ PH1
The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) and rare paediatric disease designation ...
Targeted Oncology3d
FDA Grants 225Ac-SSO110 Orphan Drug Status in SCLC
Ac-SSO110 has now received FDA orphan drug designation and investigational new drug application clearance for the treatment ...
PharmaTimes5d
FDA grants Orphan Drug Designation to Ariceum cancer therapy
Ariceum Therapeutics, a private biotech company based in Berlin, has announced that the US Food and Drug Administration (FDA) ...
The Pharma Letter1d
BIO applauds introduction of bipartisan ORPHAN Cures Act
The ORPHAN Cures Act (HR 946), a bipartisan bill that removes barriers to treating and curing rare diseases, was reintroduced in the US House of Representative last week.
Targeted Oncology13d
FDA Grants Orphan Drug Designation to MB-105 for R/R T-Cell Lymphoma
MB-105, a first-in-class CD5-targeted CAR T-cell therapy, received FDA orphan drug designation for relapsed/refractory T-cell ...
EURACTIV6d
Swedish drug agency’s new pricing model surprises, concerns patient and pharma groups
A new drug reimbursement principle is seen as a significant departure from value-based pricing. Despite concerns, the new pricing system could increase the availability of some orphan drugs by 35 per ...