Regulatory Oversight and Compliance
FDA Approves First Generics of Rivaroxaban Tablets
March 12, 2025
Rivaroxaban, long known under the brand name Xarelto, is an anticoagulant, a classification that is among the most-prescribed medications in the United States.
FDA Makes Class-Wide Labeling Changes to Testosterone Products
March 06, 2025
The alterations are the result of an agency review of findings from a clinical trial, and data from required postmarket studies.
Novartis Treatment for C3G Gets Positive Opinion from EMA
March 04, 2025
CHMP gave its opinion and a recommendation for marketing authorization to Fabhalta (iptacopan), an oral, Factor B inhibitor of the alternative complement pathway.
Rare Disease Day Celebrated on February 28
March 01, 2025
More than 6000 rare diseases—those which affect fewer than one in 2000 people each—are acknowledged in the medical community, with 72% of them being genetic.
EMA Recommends Four Medicines for Approval in February
February 28, 2025
Treatments for autoimmune diseases, multiple cancers, and skin blistering have been recommended for marketing approval.
FDA Approves First Treatment for Rare Lipid Storage Disease Cerebrotendinous Xanthomatosis
February 24, 2025
Cerebrotendinous xanthomatosis, or CTX, is a metabolic disorder caused by a gene mutation that results in a deficiency of an important enzyme in the ability of the human body to break down fats.
Gilead Treatment for PBC Granted EC Conditional Marketing Authorization
February 21, 2025
Seladelpar, which is marketed in the US as Livdelzi, was recommended for an orphan drug designation by CHMP in December 2024.
FDA Grants Full Approval for Symptomatic Tenosynovial Giant Cell Tumor Treatment
February 18, 2025
Tenosynovial giant cell tumor (TGCT) is rare and non-malignant, forming in or near joints, but which if left untreated can cause damage to joints and surrounding tissue, potentially leading to significant disability.
FDA Approves Evrysdi Tablet from Roche for Spinal Muscular Atrophy
February 17, 2025
The approval makes Evrysdi the first and only tablet for treating spinal muscular atrophy.
FDA Approves SpringWorks Therapeutics Treatment for Adult and Pediatric Patients with NF1-PN
February 13, 2025
With the approval, FDA granted SpringWorks a rare pediatric disease priority review voucher.