Pharmafile Logo
  • - PMLiVE
  • - PMLiVE
  • Diversity, Equity and Inclusion
  • Communiqué 53 Cover Image

Acute hepatic porphyria

- PMLiVE

The power of representation, innovation and trust in rare disease clinical trials

By Carol Pitcher-Towner

- PMLiVE

Alnylam’s RNAi therapeutic vutrisiran recommended by CHMP for rare heart disease

The committee has recommended the drug for both wild type and hereditary ATTR-CM

- PMLiVE

Alnylam scores EU approval for RNAi drug Givlaari

Company says it will work towards value-based agreements for the drug

- PMLiVE

Givlaari, Rybelsus lead crowded CHMP recommendations

EU regulator delivered 15 positive opinions and no rejections

- PMLiVE

Alnylam’s third RNAi drug clears phase 3 trial in rare kidney disease

Plans to file for approval in US and Europe in early 2020

- PMLiVE

FDA clears Alnylam’s second RNAi drug for rare disease

Second drug in the class to be cleared by regulatory authority

- PMLiVE

ACPs to lead gene therapies in haemophilia market growth

New therapies to hit existing players

- PMLiVE

Alnylam gains key NICE approval for Onpattro

Important milestone for company's first product

- PMLiVE

Speedy approval for Pfizer’s Onpattro rival

Approved in two different formulations

- PMLiVE

Akcea steals march on Alnylam with NICE approval

Advantage to Tegsedi over Onpattro

- PMLiVE

Alnylam swaps Sanofi for Regeneron as R&D partner

Novel alliance between two biotechs

- PMLiVE

NICE says no to rival Alnylam and Akcea amyliodsis drugs – but deal could be done

Room for manoeuvre after initial rejection

Subscribe to our email news alerts

Latest content

Latest intelligence

Quick links