Doctors, Gene Silencing and Marketing - 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

The regulator said it wanted more long-term safety and efficacy data from the REGENERATE study before making a final decision on the drug, which had been expected to be the first entrant into a likely multi-billion dollar market niche. billion annually if approved.

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

Webinars

Stay Connected