Doctors, Drugs and Gene Silencing - Clinical Research Informer

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

pharmaphorum

NOVEMBER 24, 2020

The FDA has approved Alnylam’s gene silencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. The disease is also difficult to diagnose and often takes around six years before doctors correctly identify it.

FDA Approval

FDA Approval Gene Silencing Drugs RNA

High hopes as Inventiva takes NASH contender into phase 3

pharmaphorum

JANUARY 6, 2021

Analysts are continuing to back Inventiva’s lanifibranor as a potential “best-in-class” drug for the fatty liver disease NASH, as the company prepares for the launch of a phase 3 trial in spring. There are no approved drugs for NASH – full name non-alcoholic steatohepatitis – after the FDA rejected Intercept’s obeticholic acid last year.

Gene Silencing

Gene Silencing Regulation Pharma Companies Trials

Clinical Research Informer

FDA approves Alnylam’s ultra-rare disease drug Oxlumo

High hopes as Inventiva takes NASH contender into phase 3

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