Regulations

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…

Sandoz roadmap aims to up biosimilar use 30% by 2030

The 12 goals within Sandoz’ Act4Biosimilars initiative hope to increase the global adoption of biosimilars by at least 30% in over 30 countries by 2030. Novartis’s Sandoz division is a pioneer in the biosimilars space, being the first company to receive approval of a biosimilar in the US through Zarxio, a version of Amgen’s Neupogen(filgrastim), in 2015. It has also been an advocate of these medicines, which have the potential to reduce the cost of medicine for patients and payors.…

FDA AdCom endorses bluebird’s CALD gene therapy

An FDA Advisory Committee has unanimously agreed bluebird bio’s gene therapy elivaldogene autotemcel (eli-cel) outweigh the risks in treating early active cerebral adrenoleukodystrophy (CALD). The US Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) met yesterday to evaluate bluebird bio’s eli-cel as a treatment of early active cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC)…

Emergent claims J&J owes up to $420m in vaccine contract breach

J&J has terminated a COVID-19 vaccine contract because of breaches in GMP compliance at CDMO Emergent BioSolutions. J&J’s Janssen entered into an agreement with Emergent BioSolutions in 2020 for the manufacture of its viral vector-based COVID-19 vaccine. However, the contract development and manufacturing organization (CDMO) has been hit with numerous quality issues at its Bayview, Maryland facility causing an ingredient mix up and culminating in the destruction of nearly 400 million doses of vaccines, according to a recent US Government…

Time to competency must be reduced to get to market faster, says Virtuosi

Talent shortages and training issues were identified at Advanced Therapies 2022  as one of the challenges restricting ATMPs pathway to commercialization.   The difficulty to recruit highly-skilled individuals in the advanced therapy space has been heavily documented, and companies such as Lonza, SwedenBIO, and MilliporeSigma have expressed their talent shortage concerns to this publication at different times over the past couple of years. With its high cost and potential to affect time-to-market, it was no surprise that recruitment was at the forefront of…

FDA issues draft guide to tackle supply chain disruptions

The FDA says that implementing risk management principles should be applied throughout the drug supply chain to help mitigate the risk of shortages. “Today’s draft guidance is an important step in what the nation needs to achieve an increasingly secure and resilient drug supply,” the US Food and Drug Administration (FDA) said in a statement. “For Americans to have access to an uninterrupted supply of safe, effective and high-quality drugs, all entities involved in the manufacture of a drug should…

Emergent ditched 400m vaccines amid quality and staff issues, report finds

Emergent BioSolutions hid evidence of contamination and destroyed nearly 400 million doses of J&J and AstraZeneca vaccines, a US Government staff report has found. Emergent was one of the benefactors from the US Government’s efforts in countering COVID-19, winning a Trump Administration contract worth $628 million to make the Johnson & Johnson (J&J) and AstraZeneca vaccines. However, the contract development and manufacturing organization (CDMO) was hit with an FDA Form 483 and a directive to stop production at its Bayview,…

Regenxbio: ‘CDMO issue validates inhouse gene therapy investment’

Regenxbio will begin inhouse production of material for its Duchenne Muscular Dystrophy (DMD) gene therapy after a quality issue at a third-party manufacturer delayed clinical trial dosing. The initiation of dosing adeno-associated viral (AAV) based gene therapy candidate RGX-202 for a Phase I/II clinical trial has been delayed by between six and 12 months, Regenxbio announced last week during its first quarter 2022 financial results. According to CEO Ken Mills, the delay is due to an issue at an undisclosed contract…