Regulations

Catalent hit with FDA 483 at Bloomington plant

A US FDA Form 483 has revealed visible foreign particles in certain batches of drug product at Catalent’s Bloomington, Indiana facility. The Bloomington plant, which offers fill and finish services and commercial scale production, was handed a Form 483 from the US Drug and Food Administration (FDA) following an inspection at the facility between August 1 and September 1. The 483 detailed that the FDA identified 179 occasions where particles, like black specks, foreign matter, particular matter, and foreign objects…

Bluebird Skysona approval: ‘A resurgence for HSC gene therapy’

The US approval of bluebird bio’s Skysona (eli-cel) marks a resurgence for the wider hematopoietic stem cell (HSC) gene therapy sector, says AVROBIO. Following the endorsement by an Advisory Committee in June, the US Food and Drug Administration (FDA) granted approval of bluebird bio’s Skysona (elivaldogene autotemcel, or eli-cel) Friday for the treatment of active cerebral adrenoleukodystrophy (CALD). Skysona uses ex vivo transduction with a Lenti-D lentiviral vector (LVV) to add functional copies of the ABCD1 gene into a patient’s…

Biden pledges $2bn to launch US biomanufacturing initiative

The US government will invest over $2 billion to launch its biomanufacturing initiative, which aims to expand manufacturing capacity in the country and ensure pandemic preparedness. The Executive Order was signed by President Biden on Monday and outlines the key goals of the biomanufacturing initiative. Overall, the Order looks to strengthen the country’s supply chain in the biopharma space and to reduce reliance on foreign manufacturing for the drug industry. Biden said it is the policy of his Administration to…

Cali court’s stem cell clinic ruling ‘flawed’ and ‘dangerous,’ says CGT advocacy group

The International Society for Cell & Gene Therapy (ISCT) has slammed the decision of a federal judge in California who ruled surgical procedure using a patients’ stem cells does not fall under the FDA’s regulatory jurisdiction. In 2017, the US Food and Drug Administration (FDA) took a series of actions “to remove unproven, potentially harmful treatment used in ‘stem cell’ centers targeting vulnerable patients.” This included the issuing of Form 483s and warning letters, the seizing of vials of materials…

Takeda and Cryoport team to standardize cell therapy supply chain

A standardized approach to collecting, processing, and storing cell therapy starting materials can reduce variations in manufacturing outcomes and simplify supply chain management, says Cryoport’s CEO. Temperature-controlled supply chain services firm Cryoport has partnered with Takeda’s plasma donation and collection business BioLife Plasma Services to offer standardized cell therapy collection, processing, and storage services for cell therapy products across the US and Europe. The collaboration, of which no financial details have been divulged, aims to establish an integrated GMP-compliant supply…

Iceland plant lands Alvotech’s Humira biosimilar CRL

Alvotech’s Humira biosimilar has suffered a setback after the US FDA noted deficiencies relating to its Reykjavik, Iceland manufacturing facility in a complete response letter (CRL). Alvotech filed AVT02 – a version of AbbVie’s bestselling monoclonal antibody (mAb) Humira (adalimumab) – last year but this week announced the US Food and Drug Administration (FDA) has rejected the initial biosimilar Biologics License Application (BLA), due to issues at its manufacturing plant. After visiting the facility in Reykjavik, Iceland in March 2022,…

Bluebird wins US approval for world’s most expensive drug, Zynteglo

Zynteglo’s $2.8 million price tag marks a new high, but bluebird bio says the one-time gene therapy works out cheaper than current beta-thalassemia treatment options. Following a positive Advisory Committee outcome in June, the US Food and Drug Administration (FDA) granted full approval for Zynteglo (betibeglogene autotemcel, or beti-cel) this week for the treatment of beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. The gene therapy, which was approved in Europe in 2019, is…

What’s new in Baltimore? For Emergent, it’s an FDA warning

Aseptic processes and maintenance issues have landed CDMO Emergent BioSolutions a warning letter for its Camden plant – its second Baltimore, Maryland facility to fall foul of regulators in recent months. The 87,000 square-foot Camden facility offers Emergent’s customers clinical and commercial non-viral aseptic fill/finish services across four fill lines. Earlier this year, the plant was visited by the US Food and Drug Administration (FDA) resulting in a Form 483. During Emergent’s results call on August 1, CEO Bob Kramer…

BioMarin’s EU nod another lift for budding gene therapy sector

The European Medicines Agency (EMA) endorsement of BioMarin’s hemophilia A gene therapy comes days after the US recommended two bluebird bio gene therapy products. The Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Roctavian (valoctocogene roxaparvovec), laying the ground for the approval of the first gene therapy for the treatment of hemophilia A. Roctavian is based on an adeno-associated virus (AAV) containing the gene for factor VIII, which, once administered as a one-off therapy,…

Rentschler says FDA has closed out 483 at Germany plant

Observations made by an FDA inspection in February at Rentschler’s site in Laupheim have been fully resolved, says Rentschler Biopharma. This week, the US Food and Drug Administration (FDA) published results from an inspection at contract development and manufacturing organization (CDMO) Rentschler’s drug substance manufacturing site in Laupheim, Germany carried out in February. The heavily-redacted Form 483 noted nine observations at the facility, with issues including failures to assure compliance with established specifications and standards in laboratory procedures, problems with…