Perrigo agrees to sell rare disease unit to Esteve for €275 million
The use of CRISPR-Cas9 gene editing technology has potential to transform the field of gene therapy through its simple and cost-effective delivery mechanism, said Peter Marks, director of the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER), during a 23 April webinar sponsored by the Alliance for a Stronger FDA. He also addressed FDA’s forthcoming guidance on accelerated approval for gene therapies for treating rare diseases.
Vertex’s cystic fibrosis triple combo is poised to become the top-selling orphan drug in terms of net present value, amid shifting sentiments in the pharma industry for rare disease drugs, according to Evaluate.
Neurocrine Biosciences’ newly-debuted campaign acknowledges the frustration that congenital adrenal hyperplasia (CAH) patients, families and endocrinologists often feel.
The FDA’s Center for Biologics Evaluation and Research is planning to release some crucial guidance soon for CRISPR and gene therapy developers on platform technologies, accelerated approvals for rare diseases, and potentially in the “not-too-distant future,” a platform technology guidance specifically for genome editing, CBER Director Peter Marks said Tuesday in an Alliance for a Stronger FDA webinar.
Recognition given for achievement in commercializing Joenja® (leniolisib), a first-in-class medication brought to market 10 years after disease state, APDS, was first characterized Leiden, the...
KKR and Impilo Announce Strategic Partnership Together With Management in Rare Disease Platform Immedica Pharma
An acute competition is emerging among drug developers looking at a rare neurological disorder known as chronic inflammatory demyelinating polyneuropathy, or CIDP, and Wall Street analysts are taking note.
Mere weeks after earning the FDA’s signoff on new drug Winrevair to treat PAH, Merck has kicked off an educational campaign around the rare disease.