Industry News

FDA's approval of eladocagene exuparvovec-tneq (Kebilidi) makes that product the first FDA-approved gene therapy for treating AADC deficiency.

Brian Feth and Jonathan Grinstein go behind the headlines to discuss the impact of a Republican administration on Federal Trade Commission actions, tax relief, and Health and Human Services (HHS) leadership; as well as advancements in CAR-T.

The new Institute for Cell Therapy Discovery and Innovation will utilize expertise to develop cell therapies for cancer, autoimmune diseases, and infections.

A £520 million investment for manufacturing capacity was announced by Chancellor of the Exchequer, Rachel Reeves, but academic and industry leaders stress the money should be used to train personnel.

Recent hurricanes in the US close Baxter plant, shining a spotlight on supply chain fragility again.

CDER Director Patrizia Cavazzoni and CBER Director Peter Marks provided an update on the rare disease innovation hub in a new FDA Voices blog post.

With DKK 600 million (US$87.4 million) in funding from the Novo Nordisk Foundation, the new supercomputer offers the potential to accelerate innovation in drug discovery.

WHO and its partners are providing support to countries dealing with mpox outbreaks.

The report highlights international regulators’ plans for the development and availability of vaccines to prevent and drugs to treat mpox.

Laks Pernenkil, Brian Feth, and Alex Philippidis go behind the headlines to discuss the impact of recent news, including FDA’s drug shortage list update, Nobel prize winners in microRNA and AI, and a big-potential research win for RNA editing.

Redica Systems has been assisting pharmaceutical companies in determining GMP quality risks at their manufacturing sites, and with AI, it has a relatively new addition to its toolbox.

The agency’s CHMP recommended drugs to treatments for hemophilia, two biosimilars for psoriasis, and two flu vaccines at their October meeting.

The agency has reconfirmed its recommendation to not give positive opinion to Translarna (ataluren) after a reexamination of the available data.

The design of the center prioritizes integration of the entire supply chain, with the end goals of accelerating product development and autonomous production capacity in alignment with EU priorities.

The draft joint network strategy for the EU until 2028 will be open for public consultation until Nov. 30, 2024.

Pharmaceutical Technology sat down with Niloufar Salehi, advisor at Eli Lilly & Company, to talk about the session she is moderating at AAPS PharmSci 360 2024, Symposium: An Accelerated Development of Poorly Soluble Drugs Using Predictive Tool.

The CDMO will be launching its EnzeneX 2.0 continuous manufacturing for biologics technology during CPHI Milan, which is being held Oct. 8–10, 2024 in Milan, Italy.

Pharmaceutical Technology sat down with Erica Schlesinger, vice president of technical development at Serán Bioscience, to talk about her upcoming presentation at AAPS PharmSci 360 2024, “High Dose Subcutaneous Delivery of Protein Therapeutics.”

Pharmaceutical Technology sat down with Anna Jenks, director of Pharmaceutical Operations for Merck & Co., Inc. in Rahway, NJ, USA to talk about her prologue presentation for AAPS PharmSci 360 2024.

This Behind the Headlines news roundup panel discussion discusses news items such as research results from the June Lab at the University of Pennsylvania presented at the Hanson Wade 9th CAR-TCR Summit; winter vaccine recommendations, particularly for respiratory syncytial virus (RSV) and Beyfortus access and deployment; upcoming early October Nobel prize announcement predictions; digital biology analytics, quantitation, alongside point of care (POC) and distributed manufacturing. The panel also talks about the Biosecure Act and manufacturing reshoring; Federal reserve interest rate cuts of a full half a percent (1); and the general investment and financial outlook.

According to the survey results, 49% of industry respondents are “highly positive” on the contract services sector seeing growth over the next 18 months.

The new mechanism of action targets cholinergic receptors instead of the standard dopamine receptors.

The agency is recommending that the market authorization for Oxbryta (voxelotor) be suspended so that emerging data may be reviewed.

Two drugs, Miplyffa (arimoclomol) and Aqneursa (levacetylleucine), have been approved by FDA to treat neurological symptoms associated with Niemann-Pick disease, type C in adults and children.

FDA’s Darby Kozak provided commentary on the anniversary of the Drug Price Competition and Patent Term Restoration Act of 1984.

The agency is recommending the extension of the smallpox and mpox vaccine, Imvanex, to adolescents aged 12 to 17.

Approvals were recommended for treatments of ovarian cancer, lung cancer, bleeding disorders, respiratory disease, and more.

The critical APIs that this funding will contribute to are used in the treatment of asthma, diabetes, and anxiety disorders.

During a presentation at the PDA/FDA Joint Regulatory Conference, Nicholas Violand of Johnson & Johnson and Paul Palmer of Paul R. Palmer Limited discussed how aging facilities can impact the quality of pharmaceutical products.

The 2024 CPHI Pharma Awards comprise finalists spanning 14 categories and includes awards for future leaders, women of the year, and an “at the heart of pharma” category.