The Prescription Drug User Fee Act (PDUFA) of 1992 allows the FDA to charge fees to applicants submitting drug applications or sponsors of approved prescription drugs.

Arrowhead Pharmaceuticals has submitted an application to begin an early-stage clinical trial, evaluating ARO-DM1 in patients with Myotonic Dystrophy Type 1.

HMG-CoA reductase inhibitors are a type of medication used to lower cholesterol levels in the body and are commonly referred to as statins.

Internationally recognized biotech company CSL, in partnership with Arcturus Therapeutics, has made public that the Ministry of Health, Labor and Welfare in Japan has officially sanctioned the use of ARCT-154.

Once granted by the European Commission, the centralised marketing authorisation is valid in all European Union (EU) Member States, Iceland, Norway and Liechtenstein.

Rallybio Reveals Initial Multi-Dose Results from Finalized Phase 1 Evaluation on Safety and Drug Dynamics for RLYB212, a Monoclonal Antibody Targeting HPA-1a to Avert Thrombocytopenia in Infants and Newborns.

Type of AE: This refers to any unfavorable and unintended sign (including abnormal laboratory findings not meeting the criteria for an AE) that occurs during or after treatment with the test article.

IDH1 inhibitors have made great progress in the treatment of acute myeloid leukemia and glioma, but further research is needed to confirm their clinical efficacy in other tumors.

The choice of a radiolabeled molecule for a radiopharmaceutical drug depends on several factors, including the target tissue or organ, the specific disease being studied, and the desired properties of the radiotracer.

Tectonic Therapeutics, known for GPCR-targeted therapies, has successfully dosed the first cohort in human trials for its Fc-relaxin RXFP1 receptor project.

Generic drugs are medications that have been approved by the FDA as equivalent to brand-name drugs in terms of safety, efficacy, and quality.

Hsp90 inhibitors are substances that inhibit the activity of the Hsp90 heat shock protein. They have therapeutic benefit in treating various types of malignancies, as Hsp90 stabilizes a variety of proteins required for cancer cell survival.