The U.S. Food and Drug Administration (FDA) approved Alnylam Pharmaceuticals’ Oxlumo (lumasiran) for primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in children and adults.
The U.S. Food and Drug Administration’s PDUFA dates for the last week of November 2020 include a review of Liquidia Technologies’ NDA for LIQ861 for the treatment of pulmonary arterial hypertension (PAH).
While the world has largely been focused on the development of vaccines and therapeutics for Covid-19, the U.S. Food and Drug Administration has remained busy lining up potential approvals of medications for other diseases and illnesses.
Syneos Health announced an agreement to acquire Synteract, a full-service contract research organization (CRO) focused on the biopharmaceutical industry.
Alnylam Pharmaceuticals presented complete results from the company’s ILLUMINATE-B Phase III study in children under 6 years with primary hyperoxaluria type 1 (PH1), a rare disorder affecting the kidneys.
San Diego-based Retrophin is buying Orphan Technologies for $90 million in cash up front and up to $427 million in milestone payments.
Novartis said new interim data from an ongoing phase clinical trial for the company’s $2.1 million-per-patient gene therapy Zolgensma showed spinal muscular atrophy patients experienced significant therapeutic benefit.
The U.S. Food and Drug Administration accepted Copenhagen, Denmark-based Orphazyme’s New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick disease Type C.
Shares of Reata Pharmaceuticals plunged more than 33 percent after the company announced a potential delay in plans to seek approval of omaveloxolone, an investigational treatment of Friedreich’s ataxia (FA).
Alexion Pharmaceuticals shuttered the company’s oral small molecule Factor D inhibitor program for a rare kidney disease following disappointing clinical data.