Regulations

CSL Behring up as FDA approves hemophilia B gene therapy

With a list price of $3.5 million, Hemgenix (etranacogene dezaparvovec) becomes the most expensive single-dose drug and the first gene therapy approved for hemophilia B. The US Food and Drug Administration (FDA) gave CSL Behring’s one-time gene therapy the thumbs up yesterday to treat adults with hemophilia B, a rare bleeding disorder caused by a gene defect that results in insufficient production of blood clotting protein factor IX. “We are thrilled to witness this milestone in hemophilia B treatment,” said…

FDA discusses the ‘must-have’ CMC info to advance gene therapies

Having qualified assays and appropriate cGMPs in place is crucial to support product quality when bringing a gene therapy product into late-phase studies, the US FDA says. This week, the US Food and Drug Administration (FDA) hosted the first in a series of virtual town halls aimed at engaging with product development stakeholders and researchers to discuss topics related to advanced therapies. The event run by the Office of Tissues and Advanced Therapies (OTAT) – soon to become the Office…

Emergent: Compliance underway at Camden, but ‘it’s a long road ahead’

Emergent has invested in quality enhancement and improvement initiatives in response to an FDA warning letter but says there is still much to do. For Emergent BioSolution’s third quarter results, revenues from its contract development and manufacturing organization (CDMO) business unit dropped $76.4 million year-on-year. While much of this was due to lowered sales from COVID-19 vaccine clients AstraZeneca and J&J, the decrease also reflected reduced production at the firm’s Camden facility in Baltimore, Maryland, which recently received a warning…

Bristol Myers hit with warning letter at AZ fill/finish plant

The US FDA highlighted contamination issues at a facility that produces cancer drug Abraxane but has been earmarked by Bristol Myers Squibb to become a biologics drug product plant. The US Food and Drug Administration (FDA) inspected the facility in Phoenix, Arizona from March 28 to April 6, 2022 and issued a warning letter on October 31. The violations noted in the letter included failures to sufficiently investigate contamination issues and to effectively implement corrective actions and preventive actions (CAPA).…

Instil Bio assesses manufacturing process as it pauses cell therapy trials

A decrease in manufacturing success rates has led Instil Bio to pause trials and assess the production processes for its tumor infiltrating lymphocyte (TIL) cell therapy candidates. Dallas, Texas-based cell therapy developer Instil’s lead candidate ITIL-168 is an autologous cell therapy product derived from a participant’s own TILs. The product is on Phase II/III trials for melanoma but after reporting a decrease in the rate of successful manufacturing of the product resulting in the inability to dose some patients in…

Another FDA inspection ends in 483 for Catalent in Belgium

A US FDA inspection of a fill/finish facility in Brussels has landed Catalent with another Form 483, this time with nine observations. The Form 483 followed the inspection of the drug product, run by contract development and manufacturing organization (CDMO) Catalent, in August and September this year. Deficient air supply in aseptic areas, unexplained batch discrepancies, unestablished control procedures, and inappropriately designed equipment make up the first four observations cited by the US Food and Drug Administration (FDA). “Control over…

Zip lipped on sustainability but that doesn’t mean biomanufacturers aren’t working on it

According to industry experts at BWB, sustainability should be so ingrained in the industry’s everyday activities that firms should not have to shout about it.  Sustainability is the hot topic of the moment and a continuous talking point in the biopharma industry. Environmental, social, and governance (ESG) documents are published and sent out across the news wires, but more-often-than-not these are seen as box-ticking exercises or examples of ‘greenwashing.’   But last month, GlaxoSmithKline (GSK) launched a sustainability program calling on its…

US Gov removes WuXi Bio site from Unverified List

A China-based subsidiary of CDMO WuXi Biologics can again receive certain imported bioprocess equipment after being removed from the US Government’s Unverified List (UVL). Back in February, the US Commerce Department included two subsidiaries of WuXi Biologics based in Shanghai and Wuxi – among 33 Chinese companies – to the list, restricting their ability to import certain items subject to the Export Administration Regulations (EAR) from the US. At the time, the contract development and manufacturing organization (CDMO) said the…

NJ plant woes continue for Lilly with reports of FDA 483

Eli Lilly has received an FDA Form 483 citing further manufacturing issues at its Branchburg, New Jersey facility, Reuters reports. The Reuters’ exclusive was based on a redacted copy of the Form 483 – obtained under a Freedom of Information Act request – which followed an inspection at the Branchburg facility by the US Food and Drug Administration (FDA) in July. Among the issues highlighted, Reuters said: “Lilly personnel found ‘unknown debris’ in a production area and that raw drug…

FDA creates advanced therapies ‘super office’ to meet higher demand

The US FDA office tasked with overseeing cell and gene therapies has been renamed and reorganized in response to increased submissions. The changes – details of which were published in the Federal Register last week – saw the ‘Office of Tissues and Advanced Therapies’ become the ‘Office of Therapeutic Products (OTP)’ and will be designated as a ‘super office’ that will manage its program at a macro level. The changes – which came into effect on September 16 – will…