Pharmaceutical Technology

JUNE 29, 2022

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

Drugs 246

Drugs FDA Approval Marketing Development 246

pharmaphorum

OCTOBER 3, 2022

Amylyx Pharmaceuticals’ Relyvrio – a new treatment for amyotrophic lateral sclerosis (ALS) approved by the FDA on Thursday, after review of the data from its phase 2 trial – was the next day set at a list price of $158,000 per year in the US, sparking outcry. ALS affects over 30,000 people in the US.

Drugs 52

Drugs Generic Drugs FDA Approval Trials 52

pharmaphorum

OCTOBER 21, 2021

Vounatsos also insisted that Aduhelm’s price tag has not been a factor discouraging treatment with the drug. Pushback from doctors in particular is causing anxiety for investors, as that may not be affected much if CMS opts to approve reimbursement, and shares in the company fell a little over 6% following the results announcement.

Sales 119

Sales Doctors Doctor Gene Therapy 119

Delveinsight

FEBRUARY 23, 2021

The company plans to use the capital to expand its pipeline of protein-based vaccines and biologic cancer therapies utilizing its innovative and proprietary Trimer-Tag technology platform. The company develops transformative biologics including vaccines and therapeutics for the world’s most debilitating diseases.

In-Vitro 52

In-Vitro Vaccination Vaccine Licensing 52