Through this global alliance, PackGene, Weill Cornell Medicine, and GC4K, an Australian non-profit, intend to deliver a custom-tailored gene therapy solution to treat hereditary spastic paraplegia type 56, a particularly rare neurological disease.

The HTAR, which aims to increase access to new medicines, becomes effective Jan. 12, 2025.

Patrizia Cavazzoni, director of FDA’s Center for Drug Evaluation and Research, has announced her retirement from the agency.

CNP-104 is a biodegradable nanoparticle that previously received fast track designation from FDA in January 2022, which would eventually make it eligible for accelerated approval and priority review.

Posdinemab is the second tau-directed investigational therapy by J&J that has received fast track designation from FDA this year for Alzheimer’s disease.

Vanessa Almendro, Elliot Berger, Benjamin McCloud, and Turna Ray go behind the headlines to discuss 2024’s biggest trends in the pharmaceutical industry, as well as the unforeseen outcomes that took us by surprise.

The AI Lab was launched almost exactly three years ago and has produced several programs so far, at least one of which achieved its first scientific stage gate 40% faster than the teams originally forecasted.

Using splicing technology, the new company aims to innovate safer and more effective RNA therapies for severe genetic diseases.

The final guidance is a revision of a policy that has existed in some form since at least 2009, while a separate, draft guidance will accept public comment for consideration for its final version through April 8.

With the new $94 million (€90 million) funding, the company will develop its pipeline of oral macrocycle drugs, nCycles, against validated biologic targets.

With this approval, HYQVIA [Immune Globulin Infusion 10% (human) with Recombinant Human Hyaluronidase] becomes the first and only facilitated subcutaneous immunoglobulin available in Japan to treat these disorders.

The draft guidance document provides recommendations for complying with 21 CFR 211.110.

Under the agreement, the companies will advance development of IBI3009, Innovent’s ADC candidate, which has received IND approvals in the US, China, and Australia.

The comment period for the Federal Register Notice, “Evaluating the Immunogenicity Risk of Host Cell Proteins in Follow-on Recombinant Peptide Products: Establishment of a Public Docket: Request for Information and Comments”, has been reopened by FDA until March 3, 2025.

In an interview with Pharmaceutical Technology Roger Viney from ICE Pharma highlights the importance of sustainability for the bio/pharma industry and some important future considerations for companies.

The guidance provides recommendations for those interested in participating in FDA’s Advanced Manufacturing Technologies Designation Program.

Contract development and manufacturing organizations (CDMOs) are more critical than ever as the demand for advanced therapies continues to grow. Here are the CDMOs and service providers that are poised to make the biggest impact on the pharmaceutical industry.

The company is recalling one lot of PROGRAF 0.5mg (tacrolimus) and one lot of ASTAGRAF XL 0.5mg (tacrolimus extended-release) capsules because some bottles may contain empty capsules.

The PharmTech Group editors present our most popular technical articles from 2024.

The PharmTech Group editors present our most popular interviews from 2024.

FDA's approval of Hikma Pharmaceuticals’ liraglutide injection makes this product the first generic version of Victoza, a GLP-1 receptor agonist.

Pointing to the continued circulation and evolution of COVID-19, WHO published a statement on the COVID-19 vaccine antigen composition to respond to variants of the virus.

The launch of Roche's cobas mass spectrometry solution will bring fully automated mass spec analysis to the clinical lab.

Revised guidelines published by EMA and HMA update the rules on identification of commercially confidential information and personal data used in marketing authorization applications.

Results from a Phase IIb study missed the primary endpoint by failing to achieve statistical significance.

Emer Cooke, executive director of EMA, is encouraged by the competitiveness of the European pharmaceutical market.

The company is voluntarily recalling all lots of Adrenalin Chloride Solution (EPINEPHrine nasal solution, USP) 30 mg/30 mL (1 mg/mL) 30 mL vials due to misleading labeling and its status as an unapproved drug.

The companies will use Orexo’s powder-based drug delivery technology to develop mucosal vaccines in an inhaled formulation.