Pharmaceutical Technology

SEPTEMBER 30, 2022

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. bluebird then broke its own pricing record when the $3m Skysona was approved, and now faces the unique challenge of selling the world's two most expensive drugs.

Gene Therapy 289

Gene Therapy FDA Approval Gene Genetic Disease 289

Drug Discovery World

OCTOBER 31, 2022

The last month has seen huge strides forward in our understanding of cancers, particularly in how they develop resistance to therapies and how we can ‘outsmart’ them using gene editing or different therapeutic pathways, but also how we can better target drugs to individuals and accurately predict treatment outcomes.

Drugs 52

Drugs Research Gene Editing Protein 52

pharmaphorum

NOVEMBER 16, 2021

NHS patients in England and Wales with drug-resistant epilepsy are in line for access to another treatment option, after NICE recommended routine use of Angelini group company Arvelle Pharma’s Ontozry. The drug was eventually cleared in the EU in April, with the UK giving it a green light in June.

Drugs 96

Drugs Medicine Clinical Trials Clinical Trials 96

XTalks

NOVEMBER 30, 2022

The therapy is now officially the world’s most expensive drug at $3.5 Hear more about the therapy and how the high price tag of the new drug is actually lower than the cost of treating Hemophilia B patients over their lifetimes with current conventional treatments. million per dose.

Gene Therapy 52

Gene Therapy Life Science Gene FDA Approval 52

pharmaphorum

JANUARY 5, 2022

The FDA thinks an antibody-drug conjugate (ADC) developed by AbbVie could set new standards in treatment for certain patients with lung cancer, awarding the drug breakthrough status. Hutchmed’s drug is also being paired with Tagrisso and AZ’s PD-L1 blocker Imfinzi (durvalumab) in global registrational trials.

Antibody 110

Antibody Drugs Protein Gene 110

pharmaphorum

DECEMBER 23, 2021

Daiichi Sankyo has been granted breakthrough status by the FDA for patritumab deruxtecan, a HER3-targeted antibody-drug conjugate (ADC) in clinical trials for lung cancer. HER3 is expressed in more than 80% of EGFR-mutant NSCLC, and overexpression is associated with poor outcomes, but no HER3-targeted drugs have been approved to date.

Drugs 71

Drugs Antibody Clinical Trials Clinical Trials 71

Rethinking Clinical Trials

FEBRUARY 28, 2024

But there are concerns to developing DCTs including lack of standardization and validation, regulatory and ethical uncertainties, engagement vs. coercion, data security and privacy issues, technological literacy and access, resistance to change and adoption, and lack of “safe” sharing. Balance with not adding barriers for participation.

Clinical Trials 141

Clinical Trials Clinical Trials Trials Clinical Research 141