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

Pharmaceutical Technology

JULY 14, 2022

It is estimated that there are currently more than 7,000 orphan diseases, many of which are considered life-threatening and most of which have a genetic basis. Despite this high number, orphan diseases are rare by definition, affecting around one in 2,000 people as defined by the European Union. Efforts have been made to change this.

Drugs 147

Drugs Medicine Marketing Development 147

pharmaphorum

JUNE 2, 2022

R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that previously did not have any. 141/2000 (the Orphan Regulation), that both acted to encourage development in the area, are regarded as crucial for changing the landscape in rare disease R&D.

Drugs 52

Drugs Sales Marketing Development 52

Delveinsight

FEBRUARY 9, 2021

In the pharmaceutical and healthcare domain, where discoveries take ample time, and success rates are unpredictable, translational research helps in ensuring better and innovative discoveries that act as the fuel for revolutionizing breakthroughs in the domain. The drug received priority review for the MZL.

FDA Approval 52

FDA Approval Marketing Research Protein 52

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. The US Food and Drug Administration (FDA) has approved a number of new rare disease drugs in recent years, including gene therapies.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

XTalks

MARCH 22, 2023

To celebrate this milestone, we’re taking a look back over the last 20 years in the pharmaceutical industry by highlighting some of the most talked-about mergers, blockbuster drugs, trends in FDA approvals and what’s next. In 2015, LabCorp acquired Covance , and Quintiles and IMS Health formed a joint venture in 2016, dubbed IQVIA.

Life Science 97

Life Science Drugs FDA Approval Vaccination 97