GlobalData on MSN5d
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 ...
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 ...
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) ...
Targeted Oncology4d
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 ...
Targeted Oncology4h
FDA Fast Tracks Amezalpat for HCC Treatment
Amezalpat received fast track designation from the FDA for the treatment of patients with hepatocellular carcinoma.
Moleculin Biotech receives first approval in Europe for AML trial recruiting
Moleculin Biotech (MBRX) has received first country regulatory approval in Europe to begin recruiting for its Phase 3 pivotal trial protocol ...
News Medical on MSN1d
U.S. FDA Grants Orphan Drug Designation to Ariceum Therapeutics’ Proprietary Radiopharmaceutical Cancer Therapy
Ariceum Therapeutics (Ariceum), a private biotech company developing radiopharmaceutical products for the diagnosis and treatment of certain hard-to-treat cancers, today announced that the U.S. Food ...
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 ...
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.
Acsh.org8h
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 ...