FDA Action Alert: Argenx, Aldeyra, Intercept and Sarepta

The FDA is set to make four important decisions this week, including the approval of Argenx’s subcutaneous efgartigimod for adults with generalized myasthenia gravis, Aldeyra’s investigational intravitreal injection ADX-2191 for primary vitreoretinal lymphoma, Intercept Pharmaceuticals’ obeticholic acid for pre-cirrhotic liver fibrosis due to non-alcoholic steatohepatitis, and Sarepta’s gene the..

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  • · 2023. 6. 20.
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FDA Braces for Looming Boom in Cell and Gene Therapy Submissions

The FDA is preparing for a surge in cell and gene therapy submissions, with up to 10 regulatory decisions expected this year alone. The establishment of the Office of Therapeutic Products (OTP) is part of the FDA's response to the growing demand for regulatory review of these therapies. However, experts have warned that modernization of both the regulatory and access and reimbursement frameworks..

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  • · 2023. 6. 13.
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FibroGen’s Duchenne Muscular Dystrophy Drug Fails Phase III Trial

FibroGen, a biotech company, experienced a setback as its leading candidate, Pamrevlumab, failed to meet the primary endpoint in a Phase III trial for non-ambulatory patients with Duchenne muscular dystrophy (DMD) on background corticosteroids. The LELANTOS-1 trial tested the candidate against a placebo and found that it missed the primary study endpoint of Performance of the Upper Limb 2.0 at t..

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  • · 2023. 6. 8.
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