FDA approves Zevra’s rare disease drug after resubmission

After a second review, the FDA approved Zevra Therapeutics’ treatment for Niemann-Pick disease type C, a progressive rare genetic disease caused by the accumulation of …

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Marks: FDA open to using single-arm studies for accelerated approval of rare disease gene therapies

Peter Marks, the FDA official who oversees gene therapies, is again advocating for the use of accelerated approvals for gene therapies that treat rare diseases.

November 28, 2023

Sanofi’s $1.7B rare disease gambit; CAR-T boxed warning saga; New biotechs on Nasdaq; A seller’s market for M&A?; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit

January 28, 2024

Gillian Anderson’s Anatomy Lesson; ‘Rolling’ Into Retirement; HCPs Self-Diagnose

The following contains links to social media websites including X, TikTok, Instagram, Facebook, and LinkedIn. At the 2024 Golden Globes, actor Gillian Anderson made a

January 13, 2024

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