Fayuvi scored 23.5 points higher than untreated peers on cognitive testing, backing the first-ever approval for a Sanfilippo syndrome A gene therapy.
Feature|Articles|August 25, 2023
Discussing the State of Pharma with Congressman Adam Smith
Author(s)Grant Playter
Adam Smith, a House of Representatives member representing Washington's 9th congressional district, discusses the state of U.S. healthcare and pharma.
Advertisement
Adam Smith, a House of Representatives member representing Washington's 9th congressional district, chats with Grant Playter, associate editor, to discuss the state of U.S. healthcare and pharma. Topics of discussion include Adam's memoir and own struggles with the healthcare system, balancing the competing demands of innovation and pricing, and the role of the judicial system with regards to FDA oversight.
Advertisement
Related to this article
FDA compliance officers detailed 2026 guidance updates, online drug sale enforcement, and a first-of-its-kind AI-related warning letter at the PDA/FDA Joint Regulatory Conference.
Inebilizumab (Uplizna) cut MG-ADL scores by 1.9 points versus placebo, supporting its approval in Japan for myasthenia gravis.
Lundbeck's asedebart, which blocks ACTH signaling at its source, has won FDA orphan status for endogenous Cushingsyndrome.
Satralizumab cut MOGAD relapse risk 68% in a phase 3 trial, positioning it as the first potential treatment for this rare disease.
Ersodetug missed its phase 3 primary endpoint in congenital HI, but FDA is reviewing CGM data showing 50%+ hypoglycemia reductions.
Panelists from FDA, Genentech, and Minaris discussed raw material risk, supplier oversight, and at-risk release strategies for advanced therapies at the PDA/FDA Joint Regulatory Conference.
FDA's Francis R.W. Godwin (CDER) and Kathleen Jones, PhD (CBER) detail a record pace of drug warning letters and the CGMP deficiencies most commonly cited in biologics facility inspections.
Advertisement
Advertisement