Drug Discovery World

FEBRUARY 23, 2024

In a breakthrough for advanced therapies, this week saw the FDA approve the first ever cell therapy for solid tumour cancers, but there were other significant developments in the cell and gene therapy space. News round-up for 19-23 February by DDW Digital Content Editor Diana Spencer.

Gene Therapy 52

Gene Therapy Insulin FDA Approval Drugs 52

The Pharma Data

DECEMBER 13, 2020

The FDA also recommended that Gamida Cell generate additional manufacturing-related data prior to requesting a pre-Biologics License Application (BLA) meeting. Specifically, the FDA requested that Gamida Cell demonstrate analytical and clinical comparability from the company’s planned commercial manufacturing sites.

Licensing 52

Licensing Licensing Clinical Trials Clinical Trials 52

Camargo

NOVEMBER 11, 2020

Unpacking the (Black) Box: Antares Licenses Urology Product with Boxed Warning. When the FDA requires a product’s labeling to include a boxed warning (also called a “black box warning” because the text is surrounded by black border), the potential market value of the drug often drops severely. In October, the U.S.

Development 242

Development Bioequivalency Generic Drugs Production 242

Pharmaceutical Technology

SEPTEMBER 19, 2022

The US Food and Drug Administration (FDA) has awarded Accelerated Approval for bluebird bio ’s Skysona (elivaldogene autotemcel; eli-cel) for slowing neurologic dysfunction progression in early, active cerebral adrenoleukodystrophy (CALD) patients. bluebird bio was granted a rare paediatric priority review voucher following approval.

FDA Approval 246

FDA Approval In-Vivo Gene Therapy Clinical Trials 246

Drug Discovery World

SEPTEMBER 14, 2023

Also in July 2023, Alexion, AstraZeneca Rare Disease (Alexion) and Pfizer entered into an agreement for Alexion to purchase and license the assets of Pfizer’s early-stage rare disease gene therapy portfolio for a total consideration of up to $1bn, plus tiered royalties on sales. billion, a decrease of $15.0 Vaccines were up 9.1%

Drugs 59

Drugs Vaccination Vaccine Medicine 59

XTalks

APRIL 24, 2023

Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. Dr. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Drug Discovery World

OCTOBER 4, 2022

billion in 2013 to less than $3 billion in 2018) due to loss of patent protection for the five FDA approved pharmacologic treatments available for AD, none of which slow or stop the damage from the disease. . OLX-07010 has been well characterised through an extensive program of pre-clinical toxicology and metabolism testing. .

Development 52

Development In-Vivo Compound Screening Clinical Development 52